Clinical trials: Fabry's Disease
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire
Fabry Disease
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease
- Trial name
- NCT00526071
- Testing
- migalastat HCl
- Where
- 8 sites in United States, Australia and 3 more
- Volunteers
- 23 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks.
- Trial name
- NCT04974749
- Testing
- REPLAGAL
- Where
- 6 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Fabry Disease
Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients
This study aimed to evaluate blood oxygenation at the optic nerve head in relation with visual field losses observed in many Fabry patients. Data collected allowed to evaluate if there is a link between these two entities.
- Trial name
- NCT02023086
- Testing
- Contrast sensitivity measurement, Slit Lamp assessment and intra-ocular pressure measurement, Visual field testing
- Where
- Canada
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Université de Montréal
Fabry Disease
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and…
- Trial name
- SMILE (NCT05843916)
- Testing
- Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta)
- Where
- 5 sites in Argentina
- Volunteers
- 20 people
- Sponsor
- Bio Sidus SA
Fabry Disease
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl) (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of agalsidase and the effects of agalsidase on…
- Trial name
- NCT01196871
- Testing
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme.
- Trial name
- NCT00081497
- Testing
- agalsidase beta
- Where
- 25 sites in United States, Canada and 4 more
- Volunteers
- 67 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
- Trial name
- MODIFY (NCT03425539)
- Testing
- Lucerastat
- Where
- 49 sites in United States, Australia and 12 more
- Volunteers
- 118 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Fabry Disease
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of…
- Trial name
- NCT04840667
- Testing
- REPLAGAL
- Where
- 28 sites in Canada, Finland and 6 more
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix
Fabry Disease
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase…
- Trial name
- BRIGHT (NCT03180840)
- Testing
- Pegunigalsidase alfa
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Protalix
Fabry's Disease
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume.
- Trial name
- SHORTEN (NCT06019728)
- Testing
- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sanofi
Fabry Disease
Dosing Study of Replagal in Patients With Fabry Disease
This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks…
- Trial name
- NCT00068107
- Testing
- Replagal
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Baylor Research Institute
Fabry Disease
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2…
- Trial name
- NCT01650779
- Testing
- Agalsidase beta
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease, Lysosomal Storage Diseases
A Fabry Disease Gene Therapy Study
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
- Trial name
- MARVEL1 (NCT04040049)
- Testing
- FLT190
- Where
- 13 sites in United States, Austria and 5 more
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire