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Studie přijímající pacienty
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Národní klinické studie

Victoria
ADDRESS: NOT PROVIDED - AU
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -AU
Institution: Information not provided - AU

TIROL
INNSBRUCK

REFALS: Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS (Amyotrophic Lateral Sclerosis) (Phase 3) - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

TIROL
INNSBRUCK

M-STAR: A Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy (Phase 3) - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

WIEN
ADDRESS: NOT PROVIDED - AT

REFALS-ES: Effects of oral Levosimendan (ODM-109) on respiratory function in patients with ALS (Amyotrophic Lateral Sclerosis): open label extension for patients completing study 3119002 (Phase 3) - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

Sequoia Study: A Phase 3, 4-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects With Primary Autonomic Failure - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

HORIZON: A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

Redwood Study: A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects With Primary Autonomic Failure - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT

OAK: A Phase 3, 182-week, Open-Label, Extension Study to Investigate the Safety and Tolerability of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension (Symptomatic nOH) in Subjects With Primary Autonomic Failure - AT
Institution: Information not provided - AT

WIEN
ADDRESS: NOT PROVIDED - AT


A Randomized, Double-blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous TAK-341 in Subjects With Multiple System Atrophy - AT
Institution: Information not provided - AT

VLAAMS BRABANT
LEUVEN
Een uitbreidingsonderzoek om de veiligheid, verdraagbaarheid, farmacokinetiek en effect op de ziekteprogressie op lange termijn te beoordelen van BIIB067, toegediend aan eerder behandelde volwassenen met amyotrofische laterale sclerose veroorzaakt door superoxide-dismutase 1-mutatie - BE
UZ Leuven - Campus Gasthuisberg
NMRC UZ Leuven (adults section)

VLAAMS BRABANT
LEUVEN

MAGNET: Un réseau d'essais multi-bras, adaptatifs et séquentiels en groupe pour évaluer l'efficacité des médicaments chez des patients atteints de sclérose latérale amyotrophique (SLA) - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology

VLAAMS BRABANT
LEUVEN

TUDCA-ALS OLE : Étude d'extension en ouvert visant à étudier la tolérance et l'efficacité à long terme de l'acide tauroursodésoxycholique chez des patients atteints de SLA ayant terminé l'étude TUDCA-ALS - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology

VLAAMS BRABANT
LEUVEN

TUDCA-ALS: Veiligheid en werkzaamheid van TUDCA als aanvullende behandeling bij patiënten met ALS - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology

Québec
ADDRESS: NOT PROVIDED - CA

A Phase 3b, Multicenter, Randomized, Double-blind Extension Study to Evaluate the Continued Efficacy and Safety of Oral Edaravone Administered for an Additional Period of up to 48 Weeks Following Study MT-1186-A02 in Subjects With Amyotrophic Lateral Sclerosis (ALS) -CA
Institution: Information not provided - CA

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Study NOG112264, a Phase II Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis - FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

TUDCA-ALS OLE : Étude d'extension en ouvert visant à étudier la tolérance et l'efficacité à long terme de l'acide tauroursodésoxycholique chez les patients atteints de SLA ayant terminé l'étude TUDCA-ALS -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

TUDCA-ALS: Tolérance et efficacité de TUDCA en tant que traitement complémentaire chez les patients atteints de SLA - FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
LILLE

FAIR-ALS II: Chélation conservatrice du fer comme stratégie de neuroprotection dans la sclérose latérale amyotrophique: Essai thérapeutique multicentrique randomisé en double aveugle de la deferiprone versus placebo en groupe parallèle
CHRU de Lille - Hôpital Roger Salengro
Service de Neurologie B

ILE-DE-FRANCE
PARIS

DOSERALS: Etude des transporteurs de la dopamine et de la sérotonine en imagerie TEMP utilisant les radiopharmaceutiques 123I-FP-CIT (datscan) and 123 I-ADAM dans la sclérose latérale amyotrophique et dans une population contrôle (Phase III) - FR
AP-HP.Sorbonne Université - Hôpital de la Pitié-Salpêtrière
Groupement hospitalier universitaire Pitié-Salpétrière

OCCITANIE
MONTPELLIER
MD1003-ALS : Etude randomisée, double aveugle, contre placebo pour évaluer la tolérance et l'efficacité du MD1003 dans la Sclérose latérale amyotrophique (Phase II)
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

OCCITANIE
MONTPELLIER

ROCK-ALS: étude clinique multicentrique, interventionnelle, randomisée et contrôlée en phase IIa évaluant l'innocuité, la tolérabilité et l'efficacité de Fasudil chez les patients atteints de sclérose latérale amyotrophique - FR
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

OCCITANIE
TOULOUSE

DOPS-AMS : Effets du L-Threo DOPS à long terme (3 mois) sur l'hypotension orthostatique et certains symptômes non moteurs dans l'atrophie multisystématisée (AMS) : étude comparative avec tirage au sort versus placebo (Phase II-III)
CHU de Toulouse - Hôpital Pierre-Paul Riquet
Unité d'explorations neurophysiologiques - Consultation spécialisée neuromusculaire

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Protocole TRIALS : Essai clinique randomisé exploratoire en double aveugle, contrôlé par placebo, visant à évaluer la tolérance et l'efficacité d'IFB-088 plus Riluzole 100 mg par rapport au placebo plus Riluzole 100 mg chez des patients atteints de sclérose latérale amyotrophique à début bulbaire -FR
CHU de Marseille - Hôpital de la Timone

Baden-Württemberg
ULM

TUDCA-ALS: Sicherheit und Wirksamkeit von TUDCA als Zusatztherapie bei ALS-Patienten
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

KETO-ALS: Wirksamkeit und Verträglichkeit von Beta-Hydroxybutyrat bei Patienten mit Amyotropher Lateralsklerose (ALS)
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

ADORE: Eine multizentrische, randomisierte, doppelblinde, placebokontrollierte Studie zur Untersuchung der Wirksamkeit und Sicherheit von FAB122 bei Patienten mit Amyotropher Lateralsklerose
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

COURAGE-ALS: Eine Phase 3, multizentrische, doppelblinde, randomisierte, Plazebo-kontrollierte Studie zur Beurteilung der Wirksamkeit und Sicherheit von Reldesemtiv bei Patienten mit Amyotropher Lateralsklerose (ALS)
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM
COURAGE OLE: Eine offene Phase-3-Verlängerung von COURAGE-ALS (CY 5031)
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM
MERIDIAN:Eine randomisierte, doppelblinde, placebokontrollierte, multizentrische Phase-2-Studie zur Bewertung der Wirksamkeit und Sicherheit von Pegcetacoplan bei Patienten mit amyotropher Lateralsklerose (ALS)
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Bayern
MÜNCHEN


ROCK-ALS: Hemmung der Rho Kinase (ROCK) mit Fasudil als krankheitsmodifizierende Behandlung bei ALS
Klinikum rechts der Isar der Technischen Universität München
Neurologische Klinik und Poliklinik

Berlin
BERLIN

PHOENIX: Eine randomisierte, doppelblinde, placebokontrollierte, multizentrische Phase-III-Studie zur Untersuchung der Sicherheit und Wirksamkeit von AMX0035 gegenüber Placebo bei der 48-wöchigen Behandlung erwachsener Patienten mit amyotropher Lateralsklerose (ALS)
Charité - Universitätsmedizin Berlin (CVK)
Klinik für Neurologie mit Experimenteller Neurologie (CVK)

Nordrhein-Westfalen
MÜNSTER

Auswirkungen von N-Acetyl-L-Leucin auf die Niemann-Pick-Krankheit Typ C (NPC): Eine randomisierte, plazebokontrollierte, doppelblinde Crossover-Studie der Phase III
Universitätsklinikum Münster
Centrum für seltene Erkrankungen (ZSE) Münster

Sachsen
DRESDEN


HIMALAYA: Eine multizentrische, randomisierte, doppelblinde, placebokontrollierte Phase-2-Studie zur Untersuchung der Wirksamkeit und Sicherheit von SAR443820 bei erwachsenen Teilnehmern mit amyotropher Lateralsklerose, gefolgt von einer offenen Verlängerung
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Klinik und Poliklinik für Neurologie

Sachsen
LEIPZIG
Eine randomisierte, doppelblinde, placebokontrollierte, multinationale, multizentrische Studie mit offener Behandlungserweiterung, um die Wirkung von MIN-102 auf das Fortschreiten der Adrenomyeloneuropathie bei männlichen Patienten mit X-chromosomaler Adrenoleukodystrophie (Phase II-III) zu untersuchen.
Universitätsklinikum Leipzig AöR
Klinik und Poliklinik für Neurologie

County Dublin
ADDRESS: NOT PROVIDED - IE

Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS - IE
Institution: Information not provided - IE

County Dublin
ADDRESS: NOT PROVIDED - IE
Safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS)-IE
Institution: Information not provided - IE

County Dublin
ADDRESS: NOT PROVIDED - IE
Effects of oral Levosimendan (ODM-109) on respiratory function in patients with ALS: open label extension for patients completing study 3119002-IE
Institution: Information not provided - IE

County Dublin
DUBLIN


TUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS - IE
Trinity College Dublin
Research Motor Neurone - Trinity Biomedical Sciences Institute

ISRAEL
ADDRESS: NOT PROVIDED - IL

Phase 1/2a Randomized Controlled Study for Treatment of Early- to Moderate Stage Multiple System Atrophy Patients With the Investigational Allogeneic Cell Therapy Product, hOMSC300
Institution: Information not provided - IL

CAMPANIA
NAPOLI
Valutazione dell'efficacia e del trattamento con Miglustat in pazienti affetti da malattia di Niemann-Pick di tipo c
Azienda Ospedaliera Universitaria "Federico II"
Laboratorio Dipartimento di Pediatria

CAMPANIA
NAPOLI
Studio monocentrico randomizzato controllato con placebo per confrontare l'efficacia sicurezza e tollerabilità dell'ormone della crescita in pazienti affetti da Sclerosi Laterale Amiotrofica-Phase II
Azienda Ospedaliera Universitaria "Federico II"
AOU Federico II

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

Studio di estensione di fase IIIb, multicentrico, randomizzato, in doppio cieco per valutare il mantenimento dell'efficacia e della sicurezza di edaravone orale somministrato per un periodo aggiuntivo fino a 48 settimane dopo lo studio MT-1186-A02 a soggetti con sclerosi laterale amiotrofica (SLA)
Institution: Information not provided - IT

LAZIO
ROMA

ALD-104 - Studio di fase 3 sul farmaco Lenti-D dopo condizionamento mieloablativo con busulfan e fludarabina in soggetti di età compresa fra 0 e 17 anni affetti da adrenoleucodistrofia cerebrale (CALD) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
U.O. di Oncoematologia

LAZIO
ROMA

Studio pilota sull'efficacia della stimolazione cerebrale transcranica con campo magnetico statico (tSMS) in soggetti affetti da sclerosi laterale amiotrofica (SLA)
Policlinico Universitario Campus Bio-Medico
U.O.C. Neurologia

LOMBARDIA
MILANO
Protocollo TRIALS: Studio clinico esplorativo randomizzato, controllato con placebo, in doppio cieco, per valutare la sicurezza e l'efficacia di IFB-088 più Riluzolo 100 mg in confronto a placebo più Riluzolo 100 mg in pazienti con sclerosi laterale amiotrofica ad esordio bulbare -IT
Fondazione IRCCS Istituto Neurologico "Carlo Besta"

LOMBARDIA
MILANO

Tollerabilità, sicurezza ed efficacia del sospiro durante la ventilazione meccanica non invasiva in pazienti con malattia del motoneurone - Uno studio pilota
IRCCS Ospedale San Raffaele
U.O. di Neurologia

JAPAN
ADDRESS : NOT PROVIDED - JP


A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)
Institution: Information not provided - JP

Utrecht
UTRECHT
Targeting Metabolic Flexibility in ALS (MetFlex); Safety and Tolerability of Trimetazidine for the Treatment of ALS - NL
UMC Utrecht - Universitair Medisch Centrum Utrecht
Polikliniek Neuromusculaire Ziekten

Szczecin
SZCZECIN

Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells
Zaklad Patologii Ogolnej

Bratislavsky kraj
ADDRESS: NOT PROVIDED - SK
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -SK
Institution: Information not provided - SK

Cataluña
BARCELONA
ADVANCE: Estudio multinacional, multicéntrico, aleatorizado, doble ciego y controlado con placebo con una extensión del tratamiento abierto para evaluar el efecto de MIN-102 en la progresión de la adrenomieloneuropatía en varones con adrenoleucodistrofia ligada al cromosoma X
Hospital Universitari Vall d'Hebron
Servicio de Neurología

Cataluña
L'HOSPITALET DE LLOBREGAT

MAGNET: Red de ensayo secuencial de grupo, adaptativa y de multi-rama para evaluar la eficacia de fármacos en pacientes con esclerosis lateral amiotrófica (ELA) -ES
Hospital Universitari de Bellvitge

Comunidad Valenciana
VALENCIA

ATLAS: Ensayo en fase III, aleatorizado, controlado con placebo, con una preinclusión de evolución natural longitudinal y extensión abierta para evaluar BIIB067 iniciado en adultos clínicamente presintomáticos con una mutación de superóxido dismutasa 1 confirmada
Hospital Universitario y Politécnico La Fe
Servicio de Neurología

Comunidad Valenciana
VALENCIA
IMCRELA: Impacto del tratamiento combinado de los polifenoles liposomados cúrcula y resveratrol con dutasterida, en la mejora clínica de pacientes con ELA tratados con riluzol -ES
Universidad Católica de Valencia San Vicente Mártir
Clínicas UCV

Comunidad Valenciana
VALENCIA

Impacto del tratamiento combinado de polifenoles liposomados de curcumina y resveratrol con dutasterida en la mejora clínica de los pacientes con ELA -ES
Universidad Católica de Valencia San Vicente Mártir
Facultad de Enfermería

Madrid
ADDRESS: NOT PROVIDED - ES
OAK: Estudio en fase III, de 182 semanas de duración y abierto sobre la seguridad y la tolerabilidad de TD 9855 en el tratamiento de la hipotensión ortostática neurogénica sintomática (HOns) en sujetos con fallo autonómico primario
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
MERIDIAN: Estudio de fase 2, aleatorizado, doble ciego, controlado con placebo, multicéntrico, para evaluar la eficacia y la seguridad de pegcetacoplán en sujetos con esclerosis lateral amiotrófica (ELA) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio de extensión para evaluar la seguridad, la tolerabilidad, la farmacocinética y el efecto sobre la progresión de la enfermedad a largo plazo de BIIB067 administrado a adultos tratados previamente con esclerosis lateral amiotrófica causada por una mutación de la superóxido dismutasa 1
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

COURAGE-ALS: Ensayo de fase 3, multicéntrico, doble ciego, aleatorizado y controlado con placebo para evaluar la eficacia y la seguridad de Reldesemtiv en pacientes con Esclerosis Lateral Amiotrófica (ELA) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Phoenix: Un ensayo de fase III, aleatorizado, doble ciego, controlado por placebo y multicéntrico para evaluar la seguridad y eficacia de AMX0035 frente al placebo durante un tratamiento de 48 semanas en pacientes adultos con esclerosis lateral amiotrófica (ELA) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio Fase III, prospectivo, multicéntrico, randomizado, doble ciego, controlado con placebo y de grupos paralelos para comparar la eficacia y seguridad de masitinib en combinación con riluzol frente a placebo en combinación con riluzol para el tratamiento de pacientes con Esclerosis Lateral Amiotrófica (ELA) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

ADORE: Estudio multicéntrico, aleatorizado, doble ciego y controlado con placebo para investigar la eficacia y seguridad de FAB122 en pacientes con esclerosis lateral amiotrófica
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio de fase 2a de TPN-101 en pacientes con ELA/DFT C9ORF72 (esclerosis lateral amiotrófica y/o demencia frontotemporal)
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
DAZALS: Estudio multicéntrico, aleatorizado, con enmascaramiento doble, controlado con placebo y de fase 2 en el que se evalúan la seguridad y la eficacia de CORT113176 (Dazucorilant) en pacientes con esclerosis lateral amiotrófica -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de extensión de etiqueta abierta de Fase IIIb para evaluar la seguridad y tolerabilidad de AMX0035 hasta 108 semanas en participantes adultos con esclerosis lateral amiotrófica (ELA) previamente inscritos en el estudio A35-004 (PHOENIX) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

TransportNPC: Estudio fase 3, multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo, para evaluar la seguridad, la tolerabilidad y la eficacia de 2000 mg/kg de Trappsol Cyclo (hidroxipropil-beta-ciclodextrina) y tratamiento estándar comparado con placebo y tratamiento estándar en pacientes con la enfermedad de Niemann-Pick de tipo C1 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


Estudio de fase II, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad, la tolerabilidad, la farmacocinética y la farmacodinámica de TAK-341 intravenoso en sujetos con atrofia multisistémica -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
ADOREXT: Un estudio de extensión, multicéntrico, abierto para investigar la seguridad a largo plazo de FAB122 en pacientes con esclerosis lateral amiotrófica -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

COURAGE OLE: Estudio de fase 3, extensión abierta del estudio COURAGE-ALS (CY5031) -ES
Institution: Information not provided - ES

Murcia
EL PALMAR

TCIM-ELAII: Ensayo clínico en fase II de infusión intramuscular de células madre de médula ósea autólogas en pacientes con Esclerosis lateral amiotrófica -ES
Hospital Clínico Universitario Virgen de la Arrixaca
Servicio de Hematología

Region Stockholm
ADDRESS: NOT PROVIDED - SE

MAGNET: A Multi-arm, Adaptive, Group-sequential trial NETwork to evaluate drug efficacy in patients with Amyotrophic Lateral Sclerosis (ALS) - SE
Institution: Information not provided - SE

Region Stockholm
SOLNA

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Amyotrophic Lateral Sclerosis - SE
Karolinska Institutet
Department of Clinical Neuroscience (CNS)

Suisse Alémanique
BERN
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study - CH
Universitätsspital Inselspital
University Hospital Inselspital

Suisse Alémanique
ST. GALLEN

ROCK-ALS : Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS - CH
Kantonsspital St. Gallen
Muskelzentrum/ ALS clinic

Greater London
ADDRESS: NOT PROVIDED - GB
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy - GB
Institution: Information not provided - GB

Greater London
ADDRESS: NOT PROVIDED - GB

Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial
Institution: Information not provided - GB

Greater London
ADDRESS: NOT PROVIDED - GB
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study -GB
Institution: Information not provided - GB

West Midlands
BIRMINGHAM
A Phase II Pilot Single-arm Safety and Tolerability Study of ILB in Patients With Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)-GB
University of Birmingham
Institute for cancer studies

Kansas
KANSAS CITY


Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis - US
University of Kansas Medical Center Research Institute

Minnesota
ROCHESTER

Intrathecal Autologous Mesenchymal Stem Cell Therapy in Multiple System Atrophy (MSA) - Effect of Dose and Natural History (Phase I) - US
Mayo Clinic Rochester

Washington
ADDRESS: NOT PROVIDED - US


Protocol CC100B. CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US


Phase 2 Norepinephrine Transporter Blockade, Autonomic Failure
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US

Multi-centered Double Blind, Placebo Controlled Study Evaluating the Safety and Efficacy of Memantine at 20 mg BID in Patients With ALS Currently Taking Riluzole
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US


A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US


HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS)
Institution: Information not provided - US

Victoria
ADDRESS: NOT PROVIDED - AU

A Phase 2a, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease -AU
Institution: Information not provided - AU

Victoria
ADDRESS: NOT PROVIDED - AU

A Phase 2a, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease -GB
Institution: Information not provided - AU

SALZBURG
SALZBURG


A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease - AT
Christian-Doppler-Klinik - Universitätsklinikum der PMU
Universitätsklinik für Neurologie, neurologische Intensivmedizin und Neurorehabilitation

TIROL
INNSBRUCK


A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

TIROL
INNSBRUCK


An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Intrathecally Administered RO7234292 (RG6042) in Patients With Huntington's Disease (Phase 3) - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

TIROL
INNSBRUCK

PROOF- HD: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients With Early Stage of Huntington Disease - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

TIROL
INNSBRUCK

Abdominal Binders to Treat Orthostatic Hypotension in Parkinsonian Syndromes: a Randomized, Placebo-controlled, Double-blind, Crossover Phase II Trial
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

WIEN
ADDRESS: NOT PROVIDED - AT

AMETHIST: A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) - AT
Institution: Information not provided - AT

VLAAMS BRABANT
LEUVEN

iMagemHTT: Eerste adaptieve studie bij mensen om de bindende en kinetische eigenschappen van twee nieuwe PET-liganden te onderzoeken en hun geschiktheid voor kwantificering van geaggregeerd mutant Huntingtine in de hersenen van de genuitbreidingsdragers van de ziekte van Huntington
UZ Leuven - Campus Gasthuisberg
Huntington Clinic

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

Etude de phase 2a, randomisée, contrôlée par placebo et à dose variable évaluant la tolérance et l'efficacité de PTC518 chez les sujets atteints de la maladie de Huntington -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Étude de phase IIb, randomisée, en double aveugle, contrôlée par placebo en groupes parallèles, évaluant l'efficacité et la tolérance de deux doses de SOM3355 chez des patients atteints de la maladie de Huntington et présentant des mouvements choréiques -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

DIAN-TU : Etude de plateforme de phase II/III, multicentrique, randomisée, en double aveugle, contrôlée par placebo, en deux parties et de conception adaptative, de traitements expérimentaux pour la prévention primaire de la progression de la maladie d'Alzheimer à transmission dominante -FR
Institution: Information not provided - FR

ILE-DE-FRANCE
CRÉTEIL
NEUROHD: Neuroleptiques et maladie de Huntington. Une étude multicentrique, randomisée, contrôlée pour comparer Olanzapine, Tetrabenazine et Tiapride (Phase III)
Faculté de Médecine de Créteil
Equipe "NeuroPsychologie Interventionnelle"

ILE-DE-FRANCE
PARIS
TRIHEP3: Etude comparative de phase 2, évaluant l'efficacité de la triheptanoine, thérapie anaplérotique de la maladie de Huntington - FR
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

ILE-DE-FRANCE
PARIS

REVHD : Etude de phase IIa dans la maladie de Huntington visant à évaluer le potentiel thérapeutique du resvératrol
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

Baden-Württemberg
ULM


Eine offene Verlängerungsstudie zur Bewertung der langfristigen Sicherheit und Verträglichkeit von intrathekal verabreichtem RO7234292 (RG6042) bei Patienten mit Huntington-Krankheit.
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM


PIVOT-HD: Eine randomisierte, placebokontrollierte Phase-2a-Studie zur Untersuchung der Sicherheit und Wirksamkeit von PTC518 bei Patienten mit Huntington-Krankheit
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Berlin
ADDRESS: NOT PROVIDED - DE

AMETHIST: Eine multizentrische, multinationale, randomisierte, doppelblinde, placebokontrollierte Studie zur Beurteilung der Wirksamkeit, Pharmakodynamik, Pharmakokinetik, Sicherheit und Verträglichkeit von Venglustat bei spät einsetzender GM2-Gangliosidose (Tay-Sachs-Krankheit und Sandhoff-Krankheit)
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
Randomisierte, doppelblinde, placebokontrollierte Phase-IIb-Parallelgruppenstudie zur Beurteilung der Wirksamkeit und Sicherheit von zwei Dosen SOM3355 bei Patienten mit Chorea Huntington mit choreatischen Bewegungen.
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE

DIAN-TU: Eine multizentrische, randomisierte, doppelblinde, placebokontrollierte, zweiteilige, adaptive Phase-II/III-Studie mit Prüfpräparaten zur primären Prävention des Fortschreitens der Krankheit bei dominant vererbter Alzheimer-Krankheit
Institution: Information not provided - DE

Berlin
BERLIN


Eine offene Verlängerungsstudie zur Bewertung der langfristigen Sicherheit und Verträglichkeit von intrathekal verabreichtem RO7234292 (RG6042) bei Patienten mit Huntington-Krankheit.
Charité - Universitätsmedizin Berlin (CCM)
Klinik für Psychiatrie und Psychotherapie

Hamburg
HAMBURG

Eine offene, multizentrische Studie bei männlichen pädiatrischen Patienten mit zerebraler X-chromosomaler Adrenoleukodystrophie (Cald) zur Beurteilung der Auswirkungen einer MIN-102 Behandlung auf das Fortschreiten der Krankheit vor einer humanen Stammzelltransplantation (HSCT)
UKE - Universitätsklinikum Hamburg-Eppendorf
Arbeitsgruppe Degenerative Gehirnkrankheiten

Niedersachsen
GÖTTINGEN

Eine offene, multizentrische Studie bei männlichen pädiatrischen Patienten mit zerebraler X-chromosomaler Adrenoleukodystrophie (Cald) zur Beurteilung der Auswirkungen einer MIN-102 Behandlung auf das Fortschreiten der Krankheit vor einer humanen Stammzelltransplantation (HSCT)
Universitätsmedizin Göttingen
Klinik für Kinder- und Jugendmedizin

Nordrhein-Westfalen
BOCHUM

Eine multizentrische, randomisierte, doppelblinde, placebokontrollierte Phase-1b/2a-Studie mit WVE-003, das bei Patienten mit Huntington-Krankheit intrathekal verabreicht wird
Universitäts-Kinderklinik am St. Josef-Hospital
CeSER - Centrum für Seltene Erkrankungen (ZSE) der Ruhr-Universität Bochum

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT


AMETHIST: A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) - IT
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

Studio di Fase IIb, randomizzato, in doppio cieco, controllato con placebo e a gruppi paralleli, volto a valutare l'efficacia e la sicurezza di due dosi di SOM3355 in pazienti affetti da malattia di Huntington con movimenti coreici
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

DIAN-TU: Studio multicentrico di fase II/III multicentrico, randomizzato, in doppio cieco, controllato con placebo, in due parti, con disegno adattivo, di trattamenti sperimentali per la prevenzione primaria della progressione della malattia nella malattia di Alzheimer ereditaria dominante
Institution: Information not provided - IT

LIGURIA
GENOVA
Trattamento ferrochelante nei pazienti affetti da Neurodegenerazione con accumulo cerebrale di ferro (NBIA).Studio in apero multicentrico a braccio singolo (Fase II).
Ospedali Galliera
S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro

Utrecht
ADDRESS: NOT PROVIDED - NL
GALax-C: A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) - NL
Institution: Information not provided - NL

Cataluña
BARCELONA

DIAN-TU: Ensayo de fase II/III multicéntrico aleatorizado, doble ciego, controlado con placebo, de de diseño adaptativo en dos partes, ensayo de plataforma de investigación de tratamientos para la prevención primaria de la progresión de la enfermedad de Alzheimer hereditaria dominante -ES
Hospital Clínic de Barcelona
Servicio de Neurología

Cataluña
ESPLUGUES DE LLOBREGAT

NEXUS: Estudio multicéntrico, exploratorio, abierto, en pacientes pediátricos varones con adrenoleucodistrofia cerebral ligada a x (cALD) para evaluar los efectos del tratamiento con min 102 sobre la progresión de las lesiones cerebrales -ES
Hospital Sant Joan de Déu Barcelona

Cataluña
L'HOSPITALET DE LLOBREGAT
Efectos de N-acetil-L-leucina en Gangliosidosis GM2 (enfermedad de Tay-Sachs y Sandhoff): un ensayo en fase II multinacional, multicéntrico, sin enmascaramiento para los pacientes y con enmascaramiento para los evaluadores -ES
Hospital Universitari de Bellvitge
Servicio de Neurología

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio clínico aleatorizado, multicéntrico, doble ciego, controlado con placebo, de fase III para evaluar la eficacia y seguridad de RO7234292 administrado por vía intratecal en pacientes con enfermedad de Huntington manifiesta -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

AMETHIST: Estudio multicéntrico, multinacional, aleatorizado, con doble ciego, controlado con placebo para evaluar la eficacia, farmacodinamia, farmacocinética, seguridad y tolerabilidad de venglustat en gangliosidosis GM2 de inicio tardío (enfermedad de Tay-Sachs y enfermedad de Sandhoff) [...] esfingolípidos con base de glucosilceramida -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio de extensión abierto para evaluar la seguridad y la tolerabilidad a largo plazo de RO7234292 (RG6042) administrado por vía intratecal en pacientes con enfermedad de Huntington - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


Estudio aleatorizado, doble ciego, controlado con placebo para evaluar el efecto de la metformina, un activador de la AMPK, sobre medidas cognitivas de progresión en pacientes de la enfermedad de Huntington -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

PROOF- HD: Estudio en fase III, aleatorizado, con doble enmascaramiento, controlado con placebo, con grupos paralelos y multicéntrico para evaluar la eficacia y la seguridad de pridopidina en pacientes con enfermedad de Huntington en etapa temprana -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
VIBRANT-HD: Ensayo de búsqueda de dosis, aleatorizado, doble ciego, controlado con placebo y con una extensión abierta para evaluar la seguridad, tolerabilidad, farmacocinética y farmacodinámica de LMI070/branaplam administrado en dosis orales semanales a participantes en etapas tempranas de la enfermedad de Huntington -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio de fase 1b/2a, multicéntrico, aleatorizado, doble ciego y controlado con placebo de WVE- 003 administrado por vía intratecal en pacientes con enfermedad de Huntington -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
Huntiam: Ensayo multicéntrico sobre el uso de terapia combinada de tiamina y biotina en pacientes con enfermedad de Huntington
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de fase IIb, aleatorizado, doble ciego y controlado con placebo en grupos paralelos para evaluar la eficacia y la seguridad de dos dosis de SOM3355 en pacientes con enfermedad de Huntington con movimientos coreicos -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


GENERATION HD2: Estudio de fase II, aleatorizado, doble ciego, controlado con placebo y de búsqueda de dosis para evaluar la seguridad, los biomarcadores y la eficacia de tominersen en pacientes con enfermedad de Huntington prodrómica y de aparición precoz -ES
Institution: Information not provided - ES

Suisse Alémanique
BERN
Phase IIb, Randomized, Double-blind, Placebo-controlled Study in Parallel Groups Assessing the Efficacy and Safety of Two Doses of SOM3355 in Patients Suffering From Huntington's Disease With Choreic Movements -CH
Universitätsspital Inselspital
University Hospital Inselspital

Greater Manchester
ADDRESS: NOT PROVIDED - UK

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease
Institution: Information not provided - UK

West Midlands
BIRMINGHAM


An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Intrathecally Administered RO7234292 (RG6042) in Patients With Huntington's Disease -GB
University of Birmingham
University of Birmingham HQ

Washington
ADDRESS: NOT PROVIDED - US


Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US


Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients With Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant.
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US


An Open-Label Extension Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Huntington's Disease Patients Who Participated in Prior Investigational Studies of ISIS 443139
Institution: Information not provided - US

VLAAMS BRABANT
LEUVEN

INFRONT-3: Een fase 3, multicenter, gerandomiseerde, dubbelblinde, placebo-gecontroleerde studie om de werkzaamheid en veiligheid van AL001 te evalueren bij personen met een risico op of met frontotemporale dementie als gevolg van heterozygote mutaties in het progranuline-gen - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene -FR
Institution: Information not provided - FR

Baden-Württemberg
ULM

Bayern
HAUSHAM

Eine randomisierte, doppelblinde, kontrollierte Phase-2-Studie zur Bewertung der Wirksamkeit, langfristigen Sicherheit und Verträglichkeit von RT001 bei Patienten mit progressiver supranukleärer Lähmung
Krankenhaus Agatharied GmbH
Abteilung für Neurologie

Bayern
MÜNCHEN
Wirksamkeit, Sicherheit und Verträglichkeit von Fosmetpantotenat (RE-024), einer Phosphopantothenatersatztherapie, bei Patienten mit Pantothenat-Kinase-assoziierter Neurodegeneration (PKAN): Eine randomisierte, doppelblinde, Placebo-kontrollierte Studie mit offener Verlängerung.
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Bayern
MÜNCHEN

INFRONT-3: Eine multizentrische, randomisierte, doppelblinde, placebokontrollierte Phase-3-Studie zur Bewertung der Wirksamkeit und Sicherheit von AL001 bei Personen mit einem Risiko für oder mit Frontotemporaler Demenz aufgrund von heterozygoten Mutationen im Progranulin-Gen
Klinikum rechts der Isar der Technischen Universität München

Hamburg
HAMBURG
Eine multizentrische, multinationale Phase I/II-Verlängerungsstudie zur Beurteilung der Langzeit-Wirksamkeit und Langzeit-Sicherheit von BMN 190 bei Patienten mit CLN2-Krankheit
UKE - Universitätsklinikum Hamburg-Eppendorf
Arbeitsgruppe Degenerative Gehirnkrankheiten

Hamburg
HAMBURG

Eine offene Phase 2-Studie zur Beurteilung der Sicherheit, Verträglichkeit und Wirksamkeit von intra-zerebro-ventrikulärem BMN 190 bei Patienten mit CLN2-Krankheit
UKE - Universitätsklinikum Hamburg-Eppendorf
Arbeitsgruppe Degenerative Gehirnkrankheiten

Hamburg
HAMBURG

Klinische, kinematische und elektrophysiologische Charakterisierung von Patienten mit Progressiver Supranukleärer Paralyse und pharmakologische Modulation durch Venlafaxin
UKE - Universitätsklinikum Hamburg-Eppendorf
Klinik und Poliklinik für Neurologie

Madrid
ADDRESS: NOT PROVIDED - ES

INFRONT-3: Estudio de fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de AL001 en pacientes con riesgo o con demencia frontotemporal causada por mutaciones heterocigotas en el gen de la progranulina -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Estudio de fase I/II, multicéntrico, aleatorizado, controlado con placebo, doble ciego, de dosis única y dosis múltiples para evaluar la seguridad, la tolerabilidad, la farmacocinética y la farmacodinámica de DNL593 en participantes sanos y participantes con demencia, seguido de una extensión sin enmascaramiento -ES
Institution: Information not provided - ES

Québec
ADDRESS: NOT PROVIDED - CA

An Extension Study of ABBV-8E12 in Progressive Supranuclear Palsy (PSP)-CA
Institution: Information not provided - CA

Québec
ADDRESS: NOT PROVIDED - CA

INFRONT: A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation-CA
Institution: Information not provided - CA

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

RIVA-PSP : Etude randomisée, en double aveugle vs placebo de l'efficacité de la RIVAstigmine sur les troubles moteurs et cognitivo-comportementaux dans la Paralysie Supranucléaire Progressive (Phase III)
CHU de Marseille - Hôpital de la Timone
Service de Neurologie, pathologie du mouvement

LAZIO
ROMA
Studio degli effetti del riluzolo su pazienti affetti da atassia cerebellare cronica
A.O. S. Andrea
U.O.C. di Neurologia

VENETO
TREVISO

Insonnia fatale familiare: trattamento preventivo con doxiciclina di soggetti a rischio
Azienda ULSS 9 - Ospedale di Treviso
Associazione Familiari Insonnia Familiare Fatale - Malattie da Prioni

Zuid-Holland
ROTTERDAM
The Triac Trial II: Effects of the Thyroid Hormone Analog Triac on the Neurocognitive Phenotype in Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter (Phase II) - NL
Erasmus MC - Erasmus Medisch Centrum
Polikliniek Endocrinologie

Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de extensión de etiqueta abierta para evaluar la seguridad y la tolerabilidad de la administración a largo plazo de UCB0107 en participantes del estudio con parálisis supranuclear progresiva
Institution: Information not provided - ES

TIROL
INNSBRUCK
MOXIe: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

TIROL
INNSBRUCK

NICOFA: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia - AT
Medizinische Universität Innsbruck
Universitätsklinik für Neurologie

WIEN
ADDRESS: NOT PROVIDED - AT


TALAPRO-1: A phase 2, open-label, response rate study of talazoparib in men with DNA repair defects metastatic castration-resistant prostate cancer who previously received taxane-based chemotherapy and progressed on at least 1 novel hormonal agent (enzalutamide and/or abiraterone acetate/prednisone) - AT
Institution: Information not provided - AT

VLAAMS BRABANT
LEUVEN


OLE-IEDAT: Open-label, langdurige extensie-studie van intra-erytrocyten Dexamethason natriumfosfaat bij patiënten met Ataxia Telangiectasia die deelnamen aan de IEDAT-02-2015-studie - BE
UZ Leuven - Campus Gasthuisberg
Laboratory of Pediatric Immunology

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR


A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Etude de phase 1, en aveugle, randomisée, contrôlée par placebo, évaluant l'innocuité, la tolérance et les propriétés pharmacocinétiques de doses multiples croissantes de BIIB132 administrées par voie intrathécale à des adultes atteints d'ataxie spinocérébelleuse 3 -FR.
Institution: Information not provided - FR

ILE-DE-FRANCE
PARIS

AOA1 : Evolution du taux d'albumine chez des patients atteints du syndrome d'ataxie-apraxie oculo-motrice de type 1 (AOA1) supplémentés en Coenzyme Q10 (Phase III)
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

Baden-Württemberg
TÜBINGEN


MOVE-FA: Eine randomisierte, parallele, doppelblinde, placebokontrollierte Studie mit offener Verlängerung zur Bewertung der Wirksamkeit und Sicherheit von Vatiquinon bei der Behandlung von Friedreich-Ataxie
DZNE-Standort Tübingen
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V.

Baden-Württemberg
TÜBINGEN

Eine verblindete, randomisierte, placebokontrollierte Phase-1-Studie zur Untersuchung der Sicherheit, Verträglichkeit und Pharmakokinetik mehrerer aufsteigender Dosen von BIIB132, die Erwachsenen mit spinozerebellärer Ataxie intrathekal verabreicht werden 3
Hertie-Institut für klinische Hirnforschung (HIH)
Abteilung Neurologie mit Schwerpunkt neurodegenerative Erkrankungen

Berlin
ADDRESS: NOT PROVIDED - DE

Offene, langfristige Extensionsbehandlung mit Intra-Erythrozyten-Dexamethason-Natriumphosphat bei Patienten mit Ataxia Telangiectasia, die an der IEDAT-02-2015-Studie teilgenommen haben -DE-
Institution: Information not provided - DE

Hessen
FRANKFURT AM MAIN


OLE-IEDAT: Offene, langfristige, verlängerte Behandlung mit Intra-Erythrozyten-Dexamethason-Natriumphosphat bei Patienten mit Ataxia Telangiectasia, die an der Studie IEDAT-02-2015 teilgenommen haben
Universitätsklinikum Frankfurt
Schwerpunkt Allergologie, Pneumologie und Mukoviszidose

Nordrhein-Westfalen
AACHEN

NICOFA: Eine randomisierte, doppel-verblindete, Placebo-kontrollierte, zweiarmige, multizentrische Studie zur Effizienz und Sicherheit von Nicotinamid in Patienten mit einer Friedreich-Ataxie
Universitätsklinikum Aachen
Klinik für Neurologie

ISRAEL
ADDRESS: NOT PROVIDED - IL
A Phase 1, Blinded, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of BIIB132 Administered Intrathecally to Adults With Spinocerebellar Ataxia 3 -IL
Institution: Information not provided - IL

Gelderland
NIJMEGEN
SCA3-tDCS: Elektrische stimulatie van de kleine hersenen als behandeling voor SCA3
Radboudumc - Radboud universitair medisch centrum
Afdeling Neurologie

Cataluña
ESPLUGUES DE LLOBREGAT

MOVE-FA: Estudio aleatorizado, de grupos paralelos, doble ciego, controlado con placebo, con extensión abierta, para evaluar la eficacia y la seguridad del vatiquinona para el tratamiento de la ataxia de Friedreich -ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares

Cataluña
ESPLUGUES DE LLOBREGAT

Estudio abierto a largo plazo para evaluar la seguridad y la eficacia de vatiquinona en pacientes con ataxia de Friedreich -ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares

Cataluña
SALT
Calcitriol-FA: Estudio piloto sobre los efectos del tratamiento con Calcitriol en la función neurológica y los niveles de frataxina en pacientes con ataxia de Friedreich -ES
IDIBGI - Institut d'Investigació Biomèdica de Girona Dr. Josep Trueta
Grupo de investigación Neurodegenerativas y Neuroinflamación

Madrid
ADDRESS: NOT PROVIDED - ES

STRIDES: Ensayo doble ciego, aleatorizado, controlado con placebo, para evaluar la seguridad y la eficacia de SLS-005 (inyección de trehalosa, 90,5 mg/ml para infusión intravenosa) para el tratamiento de adultos con ataxia espinocerebelosa -ES
Institution: Information not provided - ES

Madrid
MADRID

NICOFA: Estudio multicéntrico aleatorizado, doble ciego, controlado con placebo, de grupos paralelos, sobre la eficacia y seguridad de la nicotinamida en pacientes con ataxia de Friedreich
Hospital Carlos III
Unidad de ELA

Madrid
MADRID


OLE-IEDAT: Tratamiento de extensión a largo plazo y sin enmascaramiento usando fosfato sódico de dexametasona intraeritrocitaria en pacientes con ataxia telangiectasia que participaron en el estudio IEDAT-02-2015
Hospital Universitario La Paz
Servicio de Neurología

Madrid
MADRID

Efectos de N-acetil-L-leucina en la Ataxia-Telangiectasia; un ensayo en fase II multinacional, multicéntrico, sin enmascaramiento para los pacientes y con enmascaramiento para los evaluadores -ES
Hospital Universitario La Paz
Servicio de Neurología infantil

Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 1, Blinded, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of BIIB132 Administered Intrathecally to Adults With Spinocerebellar Ataxia 3 -GB
Institution: Information not provided - GB

Greater London
LONDON


NICOFA: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia-GB
University College London - UCL Darwin Building
University College London
Nadnárodní klinické studie

Queensland
QUEENSLAND
Targeting Metabolic Flexibility in ALS (MetFlex); Safety and Tolerability of Trimetazidine for the Treatment of ALS
Royal Brisbane & Women's Hospital

Finland
ESPOO

Finland
ESPOO

Finland
ESPOO
REFALS: Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS (Amyotrophic Lateral Sclerosis)
Orion Corporation

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
An open-label safety extension study of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) patients treated with Riluzole (Phase II/III)
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
TRIALS Protocol: A Double-blind, Placebo-controlled, Exploratory Randomised Clinical Trial to Assess the Safety and Efficacy of IFB-088 Plus Riluzole 100 mg vs Placebo Plus Riluzole 100 mg in Patients With Bulbar-onset Amyotrophic Lateral Sclerosis.
Institution: Information not provided - FR

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
Phase II/III, Multicenter, Randomized, Parallel Group, Double-Blind, Placebo Controlled Study to Assess Safety and Efficacy of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) Patients Treated With Riluzole (MITOTARGET) (coordination) COMPLETED
Parc scientifique et technologiques de Luminy
Trophos SA

Bayern
MÜNCHEN
ROCK-ALS: Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS
Klinikum rechts der Isar der Technischen Universität München
Neurologische Klinik und Poliklinik

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
A Multi-centre, Open-label, follow-on Study to Assess Long-term Safety and Tolerability of Intracerebroventricular Administration of sNN0029 in Patients With Amyotrophic Lateral Sclerosis
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
A Phase I, Randomised, Double-blind, Placebo-controlled Study in Patients With Amyotrophic Lateral Sclerosis to Further Assess the Safety and Tolerability of Intracerebroventricular Administration of sNN0029 Infusion Solution
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
TUDCA-ALS OLE: Open Label Extension Study to Investigate Long Term Safety, Tolerability and Efficacy of Tauroursodeoxycholic Acid in Patients With ALS Who Completed the TUDCA-ALS Study.
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
TUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS.
Institution: Information not provided - IT

Madrid
ADDRESS: NOT PROVIDED - ES
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy
Institution: Information not provided - ES

Region Stockholm
ADDRESS: NOT PROVIDED - SE
A Phase IIa, Multi-Centre, Double-Blind, Randomised, Placebo-Controlled, Parallel Group 12-Month Treatment, Adaptive Design Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of AZD3241 in Patients with Multiple System Atrophy
Institution: Information not provided - SE

Greater London
ADDRESS: NOT PROVIDED - GB
Study NOG112264: A phase II study of Ozanezumab (GSK1223249) versus placebo in the treatment of Amyotrophic Lateral Sclerosis
Institution: Information not provided - GB

Greater London
ADDRESS: NOT PROVIDED - GB
A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation - ATLAS
Institution: Information not provided - GB

Greater London
ADDRESS: NOT PROVIDED - GB
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
Institution: Information not provided - GB

Greater London
LONDON
MAGNET: A Multi-arm, Adaptive, Group-sequential trial NETwork to evaluate drug efficacy in patients with Amyotrophic Lateral Sclerosis (ALS).
King's College London
King's College London Headquarters

Oxfordshire
BEGBROKE

California
SOUTH SAN FRANCISCO

Washington
ADDRESS: NOT PROVIDED - US
ENVISION - An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis (Phase III) (Coordination)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
EMPOWER - A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis (Phase III)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
BENEFIT-ALS: A Phase IIb, Multi-National, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 3, Multi-center, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Reldesemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS). - COURAGE-ALS
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients With Alexander Disease
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 3b, Multicenter, Randomized, Double-blind Extension Study to Evaluate the Continued Efficacy and Safety of Oral Edaravone Administered for an Additional Period of up to 48 Weeks Following Study MT-1186-A02 in Subjects With Amyotrophic Lateral Sclerosis (ALS).
Institution: Information not provided - US

Sjælland
BALLERUP

ILE-DE-FRANCE
PARIS
TRIHEP3: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

ISRAEL
ADDRESS: NOT PROVIDED - IL
A Phase 2, Dose-Finding, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled Study, Evaluating the Safety and Efficacy of Pridopidine 45 mg, 67.5 mg, 90 mg, and 112.5 mg Twice-Daily versus Placebo for Symptomatic Treatment in Patients with Huntington's Disease
Institution: Information not provided - IL

ISRAEL
ADDRESS: NOT PROVIDED - IL
LEGATO-HD: A Multicenter, Multinational, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Laquinimod (0.5, 1.0 and 1.5 mg/day) as Treatment in Patients with Huntington's Disease (Phase II)
Institution: Information not provided - IL

ISRAEL
PETAH TIKVA
A Multi-centered, Randomized, Double-blind, Placebo-controlled Clinical Trial to Assess the Efficacy, Safety, and Tolerability of Rasagiline Mesylate 1 mg in Patients with Multiple System Atrophy of the Parkinsonian Subtype (MSA-P) (Phase IIb) (coordination)
Teva Pharmaceutical Industries Ltd.

Madrid
ADDRESS: NOT PROVIDED - ES
NEXUS: An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
Phase IIb, Randomized, Double-blind, Placebo-controlled Study in Parallel Groups Assessing the Efficacy and Safety of Two Doses of SOM3355 in Patients Suffering From Huntington's Disease With Choreic Movements.
Institution: Information not provided - ES

Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Intrathecally Administered RO7234292 (RG6042) in Patients With Huntington's Disease
Institution: Information not provided - CH

Suisse Romande
ADDRESS: NOT PROVIDED - CH
GENERATION HD2: A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's Disease.
Institution: Information not provided - CH

Greater Manchester
ADDRESS: NOT PROVIDED - UK
SAVVY CHILD: Phase I/II open-label trial to determine the safety and tolerability of intracranial gene therapy in GM2 gangliosidosis using recombinant adeno-associated viral vectors
Institution: Information not provided - UK

Oxfordshire
BEGBROKE

Massachusetts
CAMBRIDGE

Missouri
ST. LOUIS
DIAN-TU: A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled, Two-part Adaptive Design, Platform Trial of Investigational Treatments for Primary Prevention of Disease Progression in Dominantly Inherited Alzheimer's Disease.
Washington University School of Medicine

Washington
ADDRESS: NOT PROVIDED - US
A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 443139 in Patients With Early Manifest Huntington's Disease
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
An Open Label Extension Study To Investigate The Long Term Safety, Tolerability And Efficacy Of Pf-02545920 In Subjects With Huntington's Disease Who Previously Completed Study A8241021
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
An Open Label, Long Term Safety Study of SD-809 ER in Patients With Chorea Associated With Huntington Disease
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Randomized, Double Blind Placebo Controlled Trial To Evaluate The Safety, Tolerability, Pharmacokinetics And Efficacy Of Pf-04360365 (Ponezumab) In Adult Subjects With Probable Cerebral Amyloid Angiopathy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
GALax-C: A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 2a, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease.
Institution: Information not provided - US

Québec
ADDRESS: NOT PROVIDED - CA
TIRCON - A phase III randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN)
Institution: Information not provided - CA

Québec
ADDRESS: NOT PROVIDED - CA
TIRCON: Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN)
Institution: Information not provided - CA

Berkshire
MAIDENHEAD

California
SAN FRANCISCO
Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Davunetide for the Treatment of Progressive Supranuclear Palsy - coordination - terminated
University of California, San Francisco
Memory and Aging Center

Washington
ADDRESS: NOT PROVIDED - US
A phase 1/2 open-label dose-escalation study to evaluate safety, tolerability, pharmacokinetics, and efficacy of intracerebroventricular BMN 190 in patients with CLN2 disease (190-201)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the Cisterna Magna of Adult Subjects With Frontotemporal Dementia and Mutations in the Progranulin Gene
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
INFRONT-3: A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene
Institution: Information not provided - US

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

Greater London
ADDRESS: NOT PROVIDED - GB
DeNDRoN: A Double-Blind, Placebo-Controlled, Randomized, Parallel Group, 12-Month Safety and Efficacy Trial of Leuco-methylthioninium bis(hydromethanesulfonate) in Subjects with Behavioral Variant Frontotemporal Dementia (bvFTD) (phase III)
Institution: Information not provided - GB

Washington
ADDRESS: NOT PROVIDED - US
INFRONT: A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin Mutation
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
An Extension Study of ABBV-8E12 in Progressive Supranuclear Palsy (PSP)
Institution: Information not provided - US

Ontario
WESTON
An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia (Phase II) (coordination)
Apotex Inc.
ApoPharma Inc.

Ontario
WESTON
A Six-Month Double-Blind, Randomized, Placebo-Controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia (Phase I-II) (coordination)
Apotex Inc.
ApoPharma Inc.

Nordrhein-Westfalen
AACHEN
NICOFA: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia
Universitätsklinikum Aachen

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
Institution: Information not provided - IT

Suisse Romande
ADDRESS: NOT PROVIDED - CH
MICONOS Extension : A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients (coordination)
Institution: Information not provided - CH

Washington
ADDRESS: NOT PROVIDED - US
A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
MOVE-FA: A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multi-center, open-label, rater-blinded Phase II study - IB1001-203
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US