Zoek een klinische studie of trial
Andere zoekoptie(s)
226 Resultaat/Resultaten
Aanwerving proef
= ; Lopend onderzoek
=
; Terminated trial
=
; Gefinancierd door een IRDiRC-lid =
; Lid van een ERN =
Nationale klinische studie(s)

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

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: A Multi-arm, Adaptive, Group-sequential trial NETwork to evaluate drug efficacy in patients with Amyotrophic Lateral Sclerosis (ALS) - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology

VLAAMS BRABANT
LEUVEN

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 - 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

Baden-Württemberg
ULM

TUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS - DE-
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

KETO-ALS: Efficacy and Tolerability of Beta Hydroxybutyrate in Patients With Amyotrophic Lateral Sclerosis (ALS)
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

ADORE: A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis -DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM

COURAGE-ALS: 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) -DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM
COURAGE OLE: A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031) -DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Baden-Württemberg
ULM
MERIDIAN: A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS) -DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Bayern
MÜNCHEN

ROCK-ALS: Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS -DE-
Klinikum rechts der Isar der Technischen Universität München
Neurologische Klinik und Poliklinik

Berlin
BERLIN

PHOENIX: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS) - DE
Charité - Universitätsmedizin Berlin (CVK)
Klinik für Neurologie mit Experimenteller Neurologie (CVK)

Berlin
BERLIN

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 -DE
Charité - Universitätsmedizin Berlin (CVK)
Klinik für Neurologie mit Experimenteller Neurologie (CVK)

Hessen
HOCHHEIM

TransportNPC: A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol Cyclo (Hydroxypropyl beta cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1 -DE
SphinCS Lyso gemeinnützige UG (Haftungsbeschränkt)
SphinCS GmbH

Nordrhein-Westfalen
MÜNSTER

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study - DE
Universitätsklinikum Münster
Centrum für seltene Erkrankungen (ZSE) Münster

Sachsen
DRESDEN


HIMALAYA: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of SAR443820 in Adult Participants With Amyotrophic Lateral Sclerosis, Followed by an Open-label Extension -DE
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Klinik und Poliklinik für Neurologie

Sachsen
LEIPZIG
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 (Phase II-III) -DE-
Universitätsklinikum Leipzig AöR
Klinik und Poliklinik für Neurologie

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: 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 -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

TUDCA-ALS: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS - FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
LILLE

FAIR-ALS II: Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis: Multicentre, Parallel-group, Placebo-controlled, Randomized Clinical Trial of Deferiprone
CHRU de Lille - Hôpital Roger Salengro
Service de Neurologie B

ILE-DE-FRANCE
PARIS

DOSERALS : Study of Dopamine and Serotonin Transporters in Patients With Amyotrophic Lateral Sclerosis and Controls. Analysis With 123I-FP-CIT (Datscan) and 123 I-ADAM Brain SPECT (Phase III) - FR
AP-HP.Sorbonne Université - Hôpital de la Pitié-Salpêtrière
Groupement hospitalier universitaire Pitié-Salpétrière

OCCITANIE
MONTPELLIER
MD1003-ALS : Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study (Phase II)
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

OCCITANIE
MONTPELLIER

ROCK-ALS: Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS - FR
CHU de Montpellier - Hôpital Gui de Chauliac
Clinique du Motoneurone et Pathologies neuromusculaires

OCCITANIE
TOULOUSE

DOPS-AMS : Evaluate the Long-term (3 Months) Efficacy of L-threo DOPS (DroxiDopa) on Orthostatic Hypotension Symptoms and Other Non-motor Symptoms in Patients With Multiple System Atrophy (MSA). Comparative Study 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

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 -FR
CHU de Marseille - Hôpital de la Timone

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
Efficacy and safety of treatment with miglustat in patients with Niemann-Pick disease type c
Azienda Ospedaliera Universitaria "Federico II"
Laboratorio Dipartimento di Pediatria

CAMPANIA
NAPOLI
Efficacy safety and tolerability of growth hormone in patients with amyotrophic lateral sclerosis as add-on therapy to Riluzole-Phase II
Azienda Ospedaliera Universitaria "Federico II"
AOU Federico II

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

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) - IT
Institution: Information not provided - IT

LAZIO
ROMA

ALD-104 - A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects 0 to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
U.O. di Oncoematologia

LAZIO
ROMA

Efficacy of Transcranial Static Magnetic Field Stimulation (tSMS) in Amyotrophic Lateral Sclerosis (ALS)
Policlinico Universitario Campus Bio-Medico
U.O.C. Neurologia

LOMBARDIA
MILANO
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 -IT
Fondazione IRCCS Istituto Neurologico "Carlo Besta"

LOMBARDIA
MILANO

Tolerability, Safety and Efficacy of Sigh During Non-invasive Mechanical Ventilation in Patients With Motor Neuron Disease - A Pilot Study
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

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


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

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: 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 - ES
Hospital Universitari Vall d'Hebron
Servicio de Neurología

Cataluña
L'HOSPITALET DE LLOBREGAT

MAGNET: A Multi-arm, Adaptive, Group-sequential trial NETwork to evaluate drug efficacy in patients with Amyotrophic Lateral Sclerosis (ALS) -ES
Hospital Universitari de Bellvitge

Comunidad Valenciana
VALENCIA

ATLAS: 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 - ES
Hospital Universitario y Politécnico La Fe
Servicio de Neurología

Comunidad Valenciana
VALENCIA
IMCRELA: Impact of the combined treatment of the liposomated polyphenols curcula and resveratrol with dutasteride, in the clinical improvement of patients with ALS treated with riluzole -ES
Universidad Católica de Valencia San Vicente Mártir
Clínicas UCV

Comunidad Valenciana
VALENCIA

Impact of the Combined Treatment of Curcumin and Resveratrol Liposomed Polyphenols With Dutasteride on the Clinical Improvement of ALS Patients -ES
Universidad Católica de Valencia San Vicente Mártir
Facultad de Enfermería

Madrid
ADDRESS: NOT PROVIDED - ES
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 - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
MERIDIAN: A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

Phoenix: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

A prospective, multicenter, randomised, double-blind, placebo-controlled, parallel groups, phase 3 study to compare the efficacy and safety of masitinib in combination with Riluzole versus placebo in combination with Riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

ADORE: A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

A Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia) - ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
DAZALS: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients with Amyotrophic Lateral Sclerosis -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants with Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX) -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

TransportNPC: A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol Cyclo (Hydroxypropyl beta cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


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 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
ADOREXT: A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


HIMALAYA: A Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SAR443820 in adult participants with amyotrophic lateral sclerosis, followed by an open-label extension -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

CARDINALS: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis -ES
Institution: Information not provided - ES

Murcia
EL PALMAR

TCIM-ELAII: Phase II clinical trial of intramuscular infusion of autologous bone marrow stem cells in patients with amyotrophic lateral sclerosis -ES
Hospital Clínico Universitario Virgen de la Arrixaca
Servicio de Hematología

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

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

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

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

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

Baden-Württemberg
ULM


PIVOT-HD: A Phase 2a, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease -DE
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

Berlin
ADDRESS: NOT PROVIDED - DE

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) - DE
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE
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 -DE
Institution: Information not provided - DE

Berlin
ADDRESS: NOT PROVIDED - DE

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 -DE
Institution: Information not provided - DE

Hamburg
HAMBURG

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) -DE
UKE - Universitätsklinikum Hamburg-Eppendorf
Arbeitsgruppe Degenerative Gehirnkrankheiten

Niedersachsen
GÖTTINGEN

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) -DE
Universitätsmedizin Göttingen
Klinik für Kinder- und Jugendmedizin

Nordrhein-Westfalen
BOCHUM

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease -DE
Universitäts-Kinderklinik am St. Josef-Hospital
CeSER - Centrum für Seltene Erkrankungen (ZSE) der Ruhr-Universität Bochum

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

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

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
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 -FR
Institution: Information not provided - FR

HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR

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 -FR
Institution: Information not provided - FR

ILE-DE-FRANCE
CRÉTEIL
NEUROHD: Neuroleptics and Huntington Disease. Comparison of Olanzapine, Tetrabenazine and Tiapride. A multicentric, Randomised, Controlled Study (Phase III) - FR
Faculté de Médecine de Créteil
Equipe "NeuroPsychologie Interventionnelle"

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

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

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 - IT
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

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 - IT
Institution: Information not provided - IT

LIGURIA
GENOVA
Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA) Phase II - IT
Ente Ospedaliero 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

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

Cataluña
BARCELONA

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 -ES
Hospital Clínic de Barcelona
Servicio de Neurología

Cataluña
ESPLUGUES DE LLOBREGAT

NEXUS: An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions -ES
Hospital Sant Joan de Déu Barcelona

Madrid
ADDRESS: NOT PROVIDED - ES

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) [...] glucosylceramide-based sphingolipid pathway -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

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

Madrid
ADDRESS: NOT PROVIDED - ES


Randomized, double-blind, placebo-controlled study to evaluate the effect of metformin, an activator of AMPK, on cognitive measures of progression in Huntington's disease patients -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
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 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
VIBRANT-HD: A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington's Disease -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES
Huntiam: Multi-technical test on the use of combined therapy of themin and biotine in patients with Huntington's disease - ES
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 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES


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 -ES
Institution: Information not provided - ES

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

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

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

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

Baden-Württemberg
ULM

Bayern
HAUSHAM

A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy -DE
Krankenhaus Agatharied GmbH
Abteilung für Neurologie

Bayern
MÜNCHEN

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 - DE
Klinikum rechts der Isar der Technischen Universität München

Hamburg
HAMBURG

Clinical, kinematic and electrophysiological characterization of patients with Progressive Supranuclear Palsy and pharmacological modulation with Venlafaxine
UKE - Universitätsklinikum Hamburg-Eppendorf
Klinik und Poliklinik für Neurologie

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

Madrid
ADDRESS: NOT PROVIDED - ES

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 -ES
Institution: Information not provided - ES

Madrid
ADDRESS: NOT PROVIDED - ES

A Phase 1/2, Multicenter, Randomized, Placebo-Controlled, Double-Blind Single Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia Followed by an Open-Label Extension -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 : Efficacy of Rivastigmine on Motor, Cognitive and Behavioural Impairment in Progressive Supranuclear Palsy: A Randomised Double Blind Placebo-controlled Clinical Trial (Phase III)
CHU de Marseille - Hôpital de la Timone
Service de Neurologie, pathologie du mouvement

LAZIO
ROMA
Effect of riluzole as a symptomatic approach in patients with chronic cerebellar ataxia
A.O. S. Andrea
U.O.C. di Neurologia

VENETO
TREVISO

Fatal familial insomnia: preventive treatment with doxycycline of at risk individuals
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
An Open-Label Extension Study to Evaluate the Safety and Tolerability of Long-Term UCB0107 Administration in Study Participants With Progressive Supranuclear Palsy - ES
Institution: Information not provided - ES

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

Baden-Württemberg
TÜBINGEN


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 -DE
DZNE-Standort Tübingen
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V.

Baden-Württemberg
TÜBINGEN

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 -DE
Hertie-Institut für klinische Hirnforschung (HIH)
Abteilung Neurologie mit Schwerpunkt neurodegenerative Erkrankungen

Hessen
FRANKFURT AM MAIN


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 -DE-
Universitätsklinikum Frankfurt
Schwerpunkt Allergologie, Pneumologie und Mukoviszidose

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 (Phase II) -DE-
Universitätsklinikum Aachen
Klinik für Neurologie

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
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 -FR
Institution: Information not provided - FR

ILE-DE-FRANCE
PARIS

AOA1 : Evolution of Albumin on AOA1 Patients Supplemented With 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

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

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

Cataluña
ESPLUGUES DE LLOBREGAT

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 -ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares

Cataluña
ESPLUGUES DE LLOBREGAT

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia -ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares

Cataluña
SALT
Calcitriol-FA: Pilot Trial About the Effects of Calcitriol's Treatment in the Neurological Function and Frataxin's Level in Friedreich's Ataxia Patients -ES
IDIBGI - Institut d'Investigació Biomèdica de Girona Dr. Josep Trueta
Grupo de investigación Neurodegenerativas y Neuroinflamación

Madrid
ADDRESS: NOT PROVIDED - ES

STRIDES: A Double-blind, Randomized, Placebo Controlled, Trial to Assess Safety and Efficacy of SLS-005 (Trehalose Injection, 90.5 mg/mL for Intravenous Infusion) for the Treatment of Adults With Spinocerebellar Ataxia -ES
Institution: Information not provided - ES

Madrid
MADRID

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study -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
Multinationale klinische studie(s)

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

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

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

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

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

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

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

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

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

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

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

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

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

Berkshire
MAIDENHEAD

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

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

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

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

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
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.
Institution: Information not provided - US

Suisse Romande
ADDRESS: NOT PROVIDED - CH