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Ensayo clínico en reclutamiento
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Ensayo(s) clínico(s) nacional(es)
ALEMANIA
Bayern
WÜRZBURG
MARVEL1: A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease -DE
Zentrum Innere Medizin (ZIM)
Medizinische Klinik und Poliklinik I - Fabry-Zentrum Würzburg
ALEMANIA
Bayern
WÜRZBURG
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) -DE-
Zentrum Innere Medizin (ZIM)
Medizinische Klinik und Poliklinik I - Nephrologie
ALEMANIA
Berlin
BERLIN
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) -DE-
Charité - Universitätsmedizin Berlin (CVK)
Medizinische Klinik mit Schwerpunkt Nephrologie und Internistische Intensivmedizin
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
PERIDOT: A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
A Multicentre, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease - AT
Institution: Information not provided - AT
AUSTRIA
WIEN
WIEN
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
AUSTRIA
WIEN
WIEN
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
CANADA
Nouvelle-Écosse
HALIFAX
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
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
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-CA
Institution: Information not provided - CA
ESPAÑA
Aragón
ZARAGOZA
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 -ES
Hospital Quirónsalud Zaragoza
Servicio de Hematología
ESPAÑA
Madrid
ADDRESS: NOT PROVIDED - ES
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 -ES
Institution: Information not provided - ES
ESPAÑA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio abierto de fase III para evaluar la eficacia y la seguridad de Replagal® en sujetos con enfermedad de Fabry que no han recibido tratamiento previo -ES
Institution: Information not provided - ES
ESPAÑA
Madrid
ADDRESS: NOT PROVIDED - ES
Estudio de fase 3 aleatorizado, abierto, de grupos paralelos y 18 meses de duración para evaluar el efecto de venglustat sobre el índice de masa ventricular izquierda, en comparación con el estándar de tratamiento en pacientes con enfermedad de Fabry e hipertrofia ventricular izquierda -ES
Institution: Information not provided - ES
FRANCIA
NORMANDIE
ROUEN
MyFABT1 : Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)
CHU de Rouen
Service d'Imagerie médicale Charles-Nicolle
IRLANDA
County Dublin
ADDRESS: NOT PROVIDED - IE
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-IE
Institution: Information not provided - IE
IRLANDA
County Dublin
ADDRESS: NOT PROVIDED - IE
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.-IE
Institution: Information not provided - IE
NORUEGA
Vestlandet
BERGEN
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
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
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
Afdeling Erfelijke Stofwisselingsziekten
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
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
Afdeling Erfelijke Stofwisselingsziekten
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
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
Afdeling Erfelijke Stofwisselingsziekten
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
CARAT: A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease - NL
Amsterdam UMC
Afdeling Erfelijke Stofwisselingsziekten
PAISES BAJOS (LOS)
Noord-Holland
AMSTERDAM
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
POLONIA
Warszawa
WARSAW
FIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - PL
Instytut "Pomnik-Centrum Zdrowia Dziecka"
Klinika Pediatrii, Zywienia i Chorob Metabolicznych
REINO UNIDO (EL)
Greater London
LONDON
A Multicentre, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease - GB
Royal Free London Hospital
Royal Free London
REINO UNIDO (EL)
Greater Manchester
SALFORD
A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants (Phase IV)
Salford Royal NHS Foundation Trust
REINO UNIDO (EL)
South Glamorgan
CARDIFF
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
REINO UNIDO (EL)
West Midlands
BIRMINGHAM
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
REPUBLICA CHECA (LA)
Capital City Prague
PRAHA
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. lekarska fakulta UK
General Teaching Hospital and 1st Faculty of Medicine
SUIZA
Suisse Alémanique
ZÜRICH
Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study
Universitätsspital Zürich
Klinik für Endokrinologie, Diabetologie und Klinische Ernährung
REINO UNIDO (EL)
Greater Manchester
MANCHESTER
The efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy
Wythenshawe Hospital
Cardiology
Ensayo(s) clínico(s) multinacional(es)
ESTADOS UNIDOS
New Jersey
CRANBURY
A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease (Phase II) (coordination)
Amicus Therapeutics Inc.
ESTADOS UNIDOS
New Jersey
CRANBURY
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations (Phase III) (coordination)
Amicus Therapeutics Inc.
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease (phase III)
Institution: Information not provided - US
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
ATTRACT: A randomized, open-label study to compare the efficacy and safety of AT1001 and Enzyme Replacement Therapy (ERT) in patients with Fabry disease and AT1001-responsive GLA mutations (Phase III)
Institution: Information not provided - US
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease
Institution: Information not provided - US
ISRAEL
ISRAEL
CARMIEL
ISRAEL
ISRAEL
CARMIEL
PAISES BAJOS (LOS)
Utrecht
ADDRESS: NOT PROVIDED - NL
A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients
Institution: Information not provided - NL
PAISES BAJOS (LOS)
Utrecht
ADDRESS: NOT PROVIDED - NL
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 (Phase I/II)
Institution: Information not provided - NL
REINO UNIDO (EL)
Greater London
ADDRESS: NOT PROVIDED - GB
An Open-Label Extension Study to Evaluate the LongTerm Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease (Phase III) (Coordination)
Institution: Information not provided - GB
REINO UNIDO (EL)
Greater London
ADDRESS: NOT PROVIDED - GB
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
Institution: Information not provided - GB
SUIZA
Suisse Alémanique
ALLSCHWIL