Clinical trials: Fabry Disease, open to volunteers
Fabry Disease
A Study of Patients With Fabry Disease (US Specific)
This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.
- Trial name
- NCT06906367
- Testing
- migalastat HCl, ERT
- Where
- 8 sites in United States
- Volunteers
- 450 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
- Trial name
- NCT06270316
- Testing
- AMT-191
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- UniQure Biopharma B.V.
Fabry Disesase
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD.
- Trial name
- NCT06858397
- Testing
- HM15421/GC1134A
- Where
- 10 sites in United States, Argentina and 1 more
- Volunteers
- 18 people
- Sponsor
- GC Biopharma Corp
Fabry Disease
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
- Trial name
- MODERN (NCT06663358)
- Testing
- Pegunigalsidase-alfa
- Where
- 10 sites in United States, Slovenia and 1 more
- Volunteers
- 100 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Fabry Disease Registry & Pregnancy Sub-registry
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status.
- Trial name
- NCT00196742
- Where
- 284 sites in United States, Argentina and 42 more
- Volunteers
- 9,000 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
- Trial name
- NCT06819514
- Testing
- EXG110 Injection
- Where
- 3 sites in China
- Volunteers
- 16 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Trial name
- FLY (NCT06328608)
- Testing
- PRX-102 1 mg/kg every two weeks
- Where
- 12 sites in United States, Austria and 4 more
- Volunteers
- 22 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world…
- Trial name
- PEGASO (NCT07109375)
- Testing
- pegunigalsidase alfa
- Where
- 7 sites in Italy
- Volunteers
- 75 people
- Sponsor
- Chiesi Italia
Fabry Disease
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Fabry Disease is a rare blood disorder that some people are born with. People with Fabry disease have low levels of an enzyme called alpha-galactosidase A. This enzyme helps to cut down fat-like substances.
- Trial name
- NCT07187440
- Testing
- No intervention
- Where
- 18 sites in China
- Volunteers
- 200 people
- Sponsor
- Takeda
Fabry Disease
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease.
- Trial name
- RISE (NCT05710692)
- Testing
- PRX-102 1 mg/kg every 2 weeks, PRX-102 2 mg/kg every 4 weeks
- Where
- 10 sites in Japan
- Volunteers
- 16 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
A Study of Replagal in Children and Adults With Fabry Disease in India
The main aim of this study is to learn more about the safety profile of Replagal. Participants will receive Replagal every 2 weeks at the clinic for about 1 year.
- Trial name
- NCT05067868
- Testing
- Replagal
- Where
- 3 sites in India
- Volunteers
- 5 people
- Sponsor
- Shire
Fabry Disease
4D-310 in Adults With Fabry Disease and Cardiac Involvement
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration.
- Trial name
- NCT05629559
- Testing
- 4D-310
- Where
- 4 sites in Australia, Taiwan
- Volunteers
- 18 people
- Sponsor
- 4D Molecular Therapeutics
Fabry Disease
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to < 12 years of age with Fabry disease and with amenable GLA variants.
- Trial name
- NCT06904261
- Testing
- Migalastat HCl 20 mg
- Where
- 12 sites in United States, Belgium and 4 more
- Volunteers
- 8 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
The purpose of this clinical trial is to learn how well lucerastat works and how safe it is in untreated adult male participants with Fabry disease.
- Trial name
- Fab-Klear (NCT07778667)
- Testing
- Lucerastat
- Where
- 16 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Fabry Disease
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding.
- Trial name
- NCT04252066
- Testing
- migalastat
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Amicus Therapeutics
Fabry Disease, Pregnancy
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during…
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Trial name
- MOS (NCT06941025)
- Testing
- Not applicable- observational study
- Where
- 5 sites in United States, Germany and 3 more
- Volunteers
- 10 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on…
- Trial name
- FAVORIT (NCT06880250)
- Where
- 23 sites in Russia
- Volunteers
- 100 people
- Sponsor
- NPO Petrovax