Orphanet: Wyszukaj badanie kliniczne

Wyszukaj badanie kliniczne

258 Wyniki

Krajowe badania kliniczne

CARDIO-TTRansform: A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) - AT Institution: Information not provided - AT

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) - AT Institution: Information not provided - AT

Ensayo ATTRIBUTE-CM: Estudio de fase III, aleatorizado, doble ciego y controlado con placebo de la eficacia y la seguridad de AG10 en pacientes con miocardiopatía amiloide por transtiretina sintomática Institution: Information not provided - ES

An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease (Phase 3) - AT Institution: Information not provided - AT

LEROS: External Natural History Controlled, Open-Label Intervention Study to Assess the Efficacy and Safety of Long-Term Treatment With Raxone® in Leber's Hereditary Optic Neuropathy (LHON) (Phase 4) - AT Institution: Information not provided - AT

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) - AT Allgemeines Krankenhaus der Stadt Wien Klinische Abteilung für Nephrologie und Dialyse

MODIFY: A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease (Phase 3) - AT Allgemeines Krankenhaus der Stadt Wien Klinische Abteilung für Nephrologie und Dialyse

Étude ouverte, multicentrique et multinationale portant sur l'innocuité et la pharmacocinétique à long terme de perfusions répétées bihebdomadaires de NeoGAA chez des patients atteints de maladie de Pompe (phase II-III) UZ Leuven - Campus Gasthuisberg UZ Leuven

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - BG - University Hospital Alexandrovska Clinic of Hematology

Enzyme Replacement Therapy for Fabry Disease: A Model for the Integration of Rare Disease Therapeutics Into the Canadian Health Care System (Phase IV) Dalhousie University Department of Medicine, Division of Nephrology

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - CZ Vseobecna fakultni nemocnice a 1. lekárska fakulta UK General Teaching Hospital and 1st Faculty of Medicine

MyFABT1 : Intérêt du suivi par IRM de patients atteints de la maladie de Fabry : impact du traitement par Agalsidase Alpha (Replagal®) CHU de Rouen Service d'Imagerie médicale Charles-Nicolle

RESCUE/REVERSE: Langfristiges Follow-up von ND4 LHON Probanden, die mit GS010 okulärer Gentherapie in der RESCUE oder REVERSE Phase III Klinischen Studien behandelt wurden Friedrich-Baur-Institut Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Eine multizentrische, offene, unkontrollierte, einarmige Verlängerungsstudie zur Bestimmung der langfristigen Sicherheit und Verträglichkeit von oralem Lucerastat bei Erwachsenen mit Fabry-Krankheit (Phase 3) Zentrum Innere Medizin (ZIM) Medizinische Klinik und Poliklinik I - Nephrologie

Mepolizumab Langzeitzugangsprogramm für Probanden, die an der Studie MEA115921 (Placebo-kontrollierte Studie von Mepolizumab zur Behandlung von eosinophiler Granulomatose mit Polyangiitis bei Probanden mit Standardtherapie) teilgenommen haben. - DE- Institution: Information not provided - DE

Eine offene, multizentrische multinationale Erweiterungsstudie zur langfristigen Sicherheit und Pharmakokinetik wiederholter zweiwöchentlicher Infusionen von NeoGAA bei Patienten mit Pompe-Erkrankungen (Phase II-III) - DE Institution: Information not provided - DE

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - IL - Rabin Medical Center - Beilinson Hospital Recanati Genetic Institute

Effetti della terapia cardioprotettiva, Carvedilol vs Ramipril, in pazienti affetti da Distrofia muscolare di Duchenne e Becker. Unione Italiana Lotta Distrofia Muscolare Centro Malattie Neuromuscolari

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - LB - Hôtel-Dieu de France, Université Saint-Joseph Oncology & Hematology

Prevention of Relapses in Proteinase 3 (PR3)-Anti-Neutrophil Cytoplasmic Antibodies (ANCA)-Associated Vasculitis UMCG - Universitair Medisch Centrum Groningen Afdeling Nefrologie

BALANCE: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta (Phase III) - NL Amsterdam UMC, locatie AMC Afdeling Erfelijke Stofwisselingsziekten

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) (Phase III) - NL Amsterdam UMC, locatie AMC Afdeling Erfelijke Stofwisselingsziekten

MODIFY: A Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Determine the EffIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (Phase III) - NL Amsterdam UMC, locatie AMC Afdeling Erfelijke Stofwisselingsziekten

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NL Amsterdam UMC, locatie AMC Polikliniek Kindermetabole Ziekten

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NO Haukeland University Hospital Paediatric Department

FIELD: Badanie dwóch schematów dawkowanie Fabrazyme (agalazydazy beta) u wcześniej nieleczonych chłopców bez ciężkich objawów (faza IIIb) Instytut "Pomnik-Centrum Zdrowia Dziecka" Klinika Pediatrii, Zywienia i Chorob Metabolicznych

Estudio de extensión abierto para evaluar la seguridad y la eficacia a largo plazo de pegunigalsidasa alfa (PRX-102) en pacientes con la enfermedad de Fabry (Fase III) Hospital Quirónsalud Zaragoza Servicio de Hematología

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III) Hospital Quirónsalud Zaragoza Servicio de Hematología

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III) Hospital Universitari Vall d'Hebron Unidad de Enfermedades Minoritarias

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III) Hospital Universitari de Bellvitge Servicio de Nefrología

MODIFY: Estudio multicéntrico, doble ciego, aleatorizado, controlado con placebo y de grupos paralelos para determinar la eficacia y la seguridad de lucerastat en monoterapia oral en adultos con enfermedad de Fabry - ES Institution: Information not provided - ES

Setmelanotide (RM-493): Ensayo de tratamiento en fase 2 de Setmelanotida (RM-493) en pacientes con trastornos genéticos raros de obesidad Hospital Infantil Universitario Niño Jesús Servicio de Endocrinología, Crecimiento y Metabolismo

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III) Hospital Universitario Ramón y Cajal Servicio de Medicina Interna

LEROS: Estudio de intervención abierto controlado por grupo de evolución natural externo para evaluar la eficacia y la seguridad del tratamiento prolongado con Raxone® en la neuropatía óptica hereditaria de Leber (NOHL) (Fase IV) Hospital Universitario Ramón y Cajal Servicio de Oftalmología

REFLECT: Ensayo aleatorizado, con doble enmascaramiento y controlado con placebo en sujetos afectados con Neuropatía óptica hereditaria de Leber G11778A ND4 desde hace un año (Fase III) Hospital Universitario Ramón y Cajal Servicio de Oftalmología

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - TN - Hôpital La Rabta Service de Pédiatrie

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo-controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service Bristol Royal Infirmary University Hospitals Bristol NHS Foundation Trust HQ

CCRN 2438: A Double-blind, Randomised, Placebo-controlled Study to Investigate the Efficacy and Safety of Mepolizumab in the Treatment of Eosinophilic Granulomatosis with Polyangiitis in Subjects Receiving Standard of Care Therapy (Phase III) - UK Addenbrooke's Hospital Addenbrookes Hospital

A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Actelion Registration Ltd.

RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials-GB Moorfield's Eye Hospital

PB-102-F02 - An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients - UK Royal Free London Hospital Lysosomal Disorders Unit

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease (Phase III) - UK Royal Free London Hospital Lysosomal Disorders Unit

A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study (Phase II) - UK Institution: Information not provided - UK

An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe DiseasE - GB Institution: Information not provided - UK

A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants (Phase IV) Salford Royal NHS Foundation Trust

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB University Hospital of Wales Division of Psychological Medicine and Clinical Neurosciences

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB Queen Elizabeth Hospital

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity University Hospital Birmingham Marshfield Clinic

COMET: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment-naïve Patients With Late-onset Pompe Disease - GB Institution: Information not provided - US

A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene -GB Institution: Information not provided - US

PROPEL Study: A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo - AT Institution: Information not provided - AT

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease - AT Institution: Information not provided - AT

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT Institution: Information not provided - AT

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

Etude ouverte sur l'innocuité, la tolérabilité et la pharmacocinétique de l'Eteplirsen chez de jeunes patients atteints de dystrophie musculaire de Duchenne par thérapie du saut de l'exon 51 - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

Langdurig, open-label extensieonderzoek voor patiënten met Duchenne spierdystrofie die deelnamen aan klinische onderzoeken ter evaluatie van Casimersen of Golodirsen - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

MOMENTUM: Een fase 2, tweedelige, meervoudige oplopende dosis studie van SRP-5051 voor dosisbepaling en vervolgens dosisuitbreiding bij patiënten met Duchenne spierdystrofie die vatbaar zijn voor Exon 51-overslaande behandeling - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

Een open-label onderzoek naar veiligheid, verdraagbaarheid en werkzaamheid van Eteplirsen bij patiënten met Duchenne spierdystrofie die studie 4658-102 hebben voltooid - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

SIDEROS-E: een fase III open-label extensiestudie om de veiligheid en werkzaamheid op lange termijn van idebenone te beoordelen bij patiënten met Duchenne spierdystrofie die de SIDEROS-studie hebben voltooid - BE UZ Leuven - Campus Gasthuisberg Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

Etude ouverte visant à évaluer l'innocuité, la tolérance et l'efficacité à long terme de GIVINOSTAT chez tous les patients DMD ayant déjà été traités dans l'une des études GIVINOSTAT- BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II) London Health Sciences Centre Children's Hospital

An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of MLN9708 Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain (AL) Amyloidosis Who Require Further Treatment - FR Institution: Information not provided - FR

Extension de l'étude de phase III de l'Ataluren (PTC124) chez des patients présentant des mutations de non-sens dans les dystrophinopathie Institution: Information not provided - FR

AmyDara: Etude Multicentrique En Ouvert de Phase 2 du Daratumumab chez les patients avec une amylose AL n'ayant pas atteint au moins une très bonne réponse partielle après traitement. CHU de Limoges - Hôpital Dupuytren Service d'hématologie clinique et de thérapie cellulaire

FOR-DMD: Doppelblinde randomisierte Phase III-Studie zur Steroid-Therapie optimierung bei Muskeldystrophie Typ Duchenne Zentrum für Kinder- und Jugendmedizin Freiburg Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene Phase-1/2-Studie von Melflufen und Dexamethason für Patienten mit Al-Amyloidose nach mindestens einer vorherigen Therapielinie Zentrum für Innere Medizin (Krehl-Klinik) Abteilung Innere Medizin V - Hämatologie, Onkologie und Rheumatologie

Eine Offene Langzeitsicherheits-, Verträglichkeits- und Wirksamkeitsstudie von GIVINOSTAT bei allen DMD-Patienten, die zuvor in einer der GIVINOSTAT-Studien behandelt wurden (Phase III) Institution: Information not provided - DE

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen DRK Kliniken Berlin Westend Klinik für Kinder- und Jugendmedizin

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS-Studie: Randomisierte, doppelblinde, placebokontrollierte, multizentrische Studie zur Beurteilung der Wirksamkeit und Sicherheit von Givinostat bei gehfähigen Patienten mit Duchenne-Muskeldystrophie Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

Eine randomisierte, doppelblinde, Placebo-kontrollierte Studie zur Beurteilung der Wirksamkeit, Sicherheit und Verträglichkeit von BMS-986089 bei ambulant behandelten Jungen mit Duchenne-Muskeldystrophie Universitätsklinikum Essen

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)-IE Children's Health Ireland @ Temple Street Department of Neurology

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE Children's Health Ireland @ Temple Street Department of Neurology

(DMD-FOR): Studio mirato alla ricerca del regime steroideo ottimale per la Distrofia Muscolare di Duchenne AOU Università degli Studi della Campania "Luigi Vanvitelli" - Centro storico Servizio di Cardiomiologia e Genetica Medica

Studio di fase IIb randomizzato, in doppio cieco, a gruppi paralleli, placebo e controllo-attivo con estensione in doppio cieco per valutare l'efficacia e la sicurezza del vamorolone in ragazzi deambulanti con distrofia muscolare di duchenne (DMD) Institution: Information not provided - IT

CARDIO-TTRansform: A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) - AT
Institution: Information not provided - AT

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) - AT
Institution: Information not provided - AT

Ensayo ATTRIBUTE-CM: Estudio de fase III, aleatorizado, doble ciego y controlado con placebo de la eficacia y la seguridad de AG10 en pacientes con miocardiopatía amiloide por transtiretina sintomática
Institution: Information not provided - ES

An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease (Phase 3) - AT
Institution: Information not provided - AT

LEROS: External Natural History Controlled, Open-Label Intervention Study to Assess the Efficacy and Safety of Long-Term Treatment With Raxone® in Leber's Hereditary Optic Neuropathy (LHON) (Phase 4) - AT
Institution: Information not provided - AT

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease (Phase 3) - AT
Allgemeines Krankenhaus der Stadt Wien

Klinische Abteilung für Nephrologie und Dialyse

MODIFY: A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease (Phase 3) - AT
Allgemeines Krankenhaus der Stadt Wien

Klinische Abteilung für Nephrologie und Dialyse

Étude ouverte, multicentrique et multinationale portant sur l'innocuité et la pharmacocinétique à long terme de perfusions répétées bihebdomadaires de NeoGAA chez des patients atteints de maladie de Pompe (phase II-III)
UZ Leuven - Campus Gasthuisberg

UZ Leuven

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - BG -
University Hospital Alexandrovska

Clinic of Hematology

Enzyme Replacement Therapy for Fabry Disease: A Model for the Integration of Rare Disease Therapeutics Into the Canadian Health Care System (Phase IV)
Dalhousie University

Department of Medicine, Division of Nephrology

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - CZ
Vseobecna fakultni nemocnice a 1. lekárska fakulta UK

General Teaching Hospital and 1st Faculty of Medicine

MyFABT1 : Intérêt du suivi par IRM de patients atteints de la maladie de Fabry : impact du traitement par Agalsidase Alpha (Replagal®)
CHU de Rouen

Service d'Imagerie médicale Charles-Nicolle

RESCUE/REVERSE: Langfristiges Follow-up von ND4 LHON Probanden, die mit GS010 okulärer Gentherapie in der RESCUE oder REVERSE Phase III Klinischen Studien behandelt wurden
Friedrich-Baur-Institut

Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Eine multizentrische, offene, unkontrollierte, einarmige Verlängerungsstudie zur Bestimmung der langfristigen Sicherheit und Verträglichkeit von oralem Lucerastat bei Erwachsenen mit Fabry-Krankheit (Phase 3)
Zentrum Innere Medizin (ZIM)

Medizinische Klinik und Poliklinik I - Nephrologie

Mepolizumab Langzeitzugangsprogramm für Probanden, die an der Studie MEA115921 (Placebo-kontrollierte Studie von Mepolizumab zur Behandlung von eosinophiler Granulomatose mit Polyangiitis bei Probanden mit Standardtherapie) teilgenommen haben. - DE-
Institution: Information not provided - DE

Eine offene, multizentrische multinationale Erweiterungsstudie zur langfristigen Sicherheit und Pharmakokinetik wiederholter zweiwöchentlicher Infusionen von NeoGAA bei Patienten mit Pompe-Erkrankungen (Phase II-III) - DE
Institution: Information not provided - DE

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - IL -
Rabin Medical Center - Beilinson Hospital

Recanati Genetic Institute

Effetti della terapia cardioprotettiva, Carvedilol vs Ramipril, in pazienti affetti da Distrofia muscolare di Duchenne e Becker.
Unione Italiana Lotta Distrofia Muscolare

Centro Malattie Neuromuscolari

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - LB -
Hôtel-Dieu de France, Université Saint-Joseph

Oncology & Hematology

Prevention of Relapses in Proteinase 3 (PR3)-Anti-Neutrophil Cytoplasmic Antibodies (ANCA)-Associated Vasculitis
UMCG - Universitair Medisch Centrum Groningen

Afdeling Nefrologie

BALANCE: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta (Phase III) - NL
Amsterdam UMC, locatie AMC

Afdeling Erfelijke Stofwisselingsziekten

An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) (Phase III) - NL
Amsterdam UMC, locatie AMC

Afdeling Erfelijke Stofwisselingsziekten

MODIFY: A Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Determine the EffIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (Phase III) - NL
Amsterdam UMC, locatie AMC

Afdeling Erfelijke Stofwisselingsziekten

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NL
Amsterdam UMC, locatie AMC

Polikliniek Kindermetabole Ziekten

FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - NO
Haukeland University Hospital

Paediatric Department

FIELD: Badanie dwóch schematów dawkowanie Fabrazyme (agalazydazy beta) u wcześniej nieleczonych chłopców bez ciężkich objawów (faza IIIb)
Instytut "Pomnik-Centrum Zdrowia Dziecka"

Klinika Pediatrii, Zywienia i Chorob Metabolicznych

Estudio de extensión abierto para evaluar la seguridad y la eficacia a largo plazo de pegunigalsidasa alfa (PRX-102) en pacientes con la enfermedad de Fabry (Fase III)
Hospital Quirónsalud Zaragoza

Servicio de Hematología

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III)
Hospital Quirónsalud Zaragoza

Servicio de Hematología

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III)
Hospital Universitari Vall d'Hebron

Unidad de Enfermedades Minoritarias

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III)
Hospital Universitari de Bellvitge

Servicio de Nefrología

MODIFY: Estudio multicéntrico, doble ciego, aleatorizado, controlado con placebo y de grupos paralelos para determinar la eficacia y la seguridad de lucerastat en monoterapia oral en adultos con enfermedad de Fabry - ES
Institution: Information not provided - ES

Setmelanotide (RM-493): Ensayo de tratamiento en fase 2 de Setmelanotida (RM-493) en pacientes con trastornos genéticos raros de obesidad
Hospital Infantil Universitario Niño Jesús

Servicio de Endocrinología, Crecimiento y Metabolismo

Estudio de extensión multicéntrico, abierto, no controlado y de un solo grupo para determinar la seguridad y tolerabilidad a largo plazo de lucerastat oral en adultos con enfermedad de Fabry (Fase III)
Hospital Universitario Ramón y Cajal

Servicio de Medicina Interna

LEROS: Estudio de intervención abierto controlado por grupo de evolución natural externo para evaluar la eficacia y la seguridad del tratamiento prolongado con Raxone® en la neuropatía óptica hereditaria de Leber (NOHL) (Fase IV)
Hospital Universitario Ramón y Cajal

Servicio de Oftalmología

REFLECT: Ensayo aleatorizado, con doble enmascaramiento y controlado con placebo en sujetos afectados con Neuropatía óptica hereditaria de Leber G11778A ND4 desde hace un año (Fase III)
Hospital Universitario Ramón y Cajal

Servicio de Oftalmología

ENGAGE - A phase 3, randomized, double-blind, placebo-controlled, multi-center study confirming the efficacy and safety of Genz-112638 in patients with Gaucher disease type 1 - TN -
Hôpital La Rabta

Service de Pédiatrie

Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo-controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service
Bristol Royal Infirmary

University Hospitals Bristol NHS Foundation Trust HQ

CCRN 2438: A Double-blind, Randomised, Placebo-controlled Study to Investigate the Efficacy and Safety of Mepolizumab in the Treatment of Eosinophilic Granulomatosis with Polyangiitis in Subjects Receiving Standard of Care Therapy (Phase III) - UK
Addenbrooke's Hospital

Addenbrookes Hospital

A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease
Actelion Registration Ltd.

RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials-GB
Moorfield's Eye Hospital

PB-102-F02 - An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients - UK
Royal Free London Hospital

Lysosomal Disorders Unit

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease (Phase III) - UK
Royal Free London Hospital

Lysosomal Disorders Unit

A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study (Phase II) - UK
Institution: Information not provided - UK

An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe DiseasE - GB
Institution: Information not provided - UK

A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants (Phase IV)
Salford Royal NHS Foundation Trust

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB
University Hospital of Wales

Division of Psychological Medicine and Clinical Neurosciences

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease-GB
Queen Elizabeth Hospital

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham

Marshfield Clinic

COMET: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment-naïve Patients With Late-onset Pompe Disease - GB
Institution: Information not provided - US

A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene -GB
Institution: Information not provided - US

PROPEL Study: A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo - AT
Institution: Information not provided - AT

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease - AT
Institution: Information not provided - AT

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT
Institution: Information not provided - AT

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT
Gottfried von Preyer'sches Kinderspital

Abteilung für Kinder- und Jugendheilkunde

Etude ouverte sur l'innocuité, la tolérabilité et la pharmacocinétique de l'Eteplirsen chez de jeunes patients atteints de dystrophie musculaire de Duchenne par thérapie du saut de l'exon 51 - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

Langdurig, open-label extensieonderzoek voor patiënten met Duchenne spierdystrofie die deelnamen aan klinische onderzoeken ter evaluatie van Casimersen of Golodirsen - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

MOMENTUM: Een fase 2, tweedelige, meervoudige oplopende dosis studie van SRP-5051 voor dosisbepaling en vervolgens dosisuitbreiding bij patiënten met Duchenne spierdystrofie die vatbaar zijn voor Exon 51-overslaande behandeling - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

Een open-label onderzoek naar veiligheid, verdraagbaarheid en werkzaamheid van Eteplirsen bij patiënten met Duchenne spierdystrofie die studie 4658-102 hebben voltooid - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

SIDEROS-E: een fase III open-label extensiestudie om de veiligheid en werkzaamheid op lange termijn van idebenone te beoordelen bij patiënten met Duchenne spierdystrofie die de SIDEROS-studie hebben voltooid - BE
UZ Leuven - Campus Gasthuisberg

Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

Etude ouverte visant à évaluer l'innocuité, la tolérance et l'efficacité à long terme de GIVINOSTAT chez tous les patients DMD ayant déjà été traités dans l'une des études GIVINOSTAT- BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II)
London Health Sciences Centre

Children's Hospital

An Open-Label, Dose-Escalation, Phase 1 Study of the Oral Formulation of MLN9708 Administered Weekly in Adult Patients With Relapsed or Refractory Light-Chain (AL) Amyloidosis Who Require Further Treatment - FR
Institution: Information not provided - FR

Extension de l'étude de phase III de l'Ataluren (PTC124) chez des patients présentant des mutations de non-sens dans les dystrophinopathie
Institution: Information not provided - FR

AmyDara: Etude Multicentrique En Ouvert de Phase 2 du Daratumumab chez les patients avec une amylose AL n'ayant pas atteint au moins une très bonne réponse partielle après traitement.
CHU de Limoges - Hôpital Dupuytren

Service d'hématologie clinique et de thérapie cellulaire

FOR-DMD: Doppelblinde randomisierte Phase III-Studie zur Steroid-Therapie optimierung bei Muskeldystrophie Typ Duchenne
Zentrum für Kinder- und Jugendmedizin Freiburg

Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene Phase-1/2-Studie von Melflufen und Dexamethason für Patienten mit Al-Amyloidose nach mindestens einer vorherigen Therapielinie
Zentrum für Innere Medizin (Krehl-Klinik)

Abteilung Innere Medizin V - Hämatologie, Onkologie und Rheumatologie

Eine Offene Langzeitsicherheits-, Verträglichkeits- und Wirksamkeitsstudie von GIVINOSTAT bei allen DMD-Patienten, die zuvor in einer der GIVINOSTAT-Studien behandelt wurden (Phase III)
Institution: Information not provided - DE

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen
DRK Kliniken Berlin Westend

Klinik für Kinder- und Jugendmedizin

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS-Studie: Randomisierte, doppelblinde, placebokontrollierte, multizentrische Studie zur Beurteilung der Wirksamkeit und Sicherheit von Givinostat bei gehfähigen Patienten mit Duchenne-Muskeldystrophie
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

Eine randomisierte, doppelblinde, Placebo-kontrollierte Studie zur Beurteilung der Wirksamkeit, Sicherheit und Verträglichkeit von BMS-986089 bei ambulant behandelten Jungen mit Duchenne-Muskeldystrophie
Universitätsklinikum Essen

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)-IE
Children's Health Ireland @ Temple Street

Department of Neurology

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE
Children's Health Ireland @ Temple Street

Department of Neurology

(DMD-FOR): Studio mirato alla ricerca del regime steroideo ottimale per la Distrofia Muscolare di Duchenne
AOU Università degli Studi della Campania "Luigi Vanvitelli" - Centro storico

Servizio di Cardiomiologia e Genetica Medica

Studio di fase IIb randomizzato, in doppio cieco, a gruppi paralleli, placebo e controllo-attivo con estensione in doppio cieco per valutare l'efficacia e la sicurezza del vamorolone in ragazzi deambulanti con distrofia muscolare di duchenne (DMD)
Institution: Information not provided - IT

Studio di fase 1/2 in aperto su Melflufen e Desametasone in pazienti con Amiloidosi AL che seguono almeno una precedente linea di terapia - IT
Institution: Information not provided - IT

Studio multicentrico randomizzato di fase III del Melphalan e Dexamethasone (MDex) vs Bortezomib, Melphalan e Dexamethasone (BMDex) nel trattamento di pazienti con Amiloidosi.
Fondazione IRCCS Policlinico San Matteo

Laboratorio di Biotecnologie e Tecnologie Biomediche

Studio randomizzato di fase II/III di doxiciclina rispetto alla terapia di supporto standard nei pazienti con amiloidosi cardiaca AL di nuova diagnosi sottoposti a terapia a base di bortezomib - IT
Fondazione IRCCS Policlinico San Matteo

Dipartimento di Medicina Molecolare

(DMD-FOR): Studio mirato alla ricerca del regime steroideo ottimale per la Distrofia Muscolare di Duchenne
Azienda Ospedaliera Universitaria di Padova - Campus Biomedico Pietro d'Abano

Neuromuscular Laboratory