Clinical trials: Anderson Fabry Disease
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
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, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
A 24-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) in participants with Fabry disease.
- Trial name
- NCT00283933
- Testing
- migalastat HCl
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Amicus 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
Fabry Disease
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry…
- Trial name
- NCT00140621
- Testing
- Agalsidase beta
- Where
- 7 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
Pharmacokinetic/Pharmacodynamic Study of 2 agalsidase beta Formulations in Single Dose Administered to Healthy Volunteers as intravenous infusion, at a concentration of 1 mg/kg
- Trial name
- NCT05343715
- Testing
- Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg
- Where
- Argentina
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Bio Sidus SA
Fabry Disease, Lysosomal Storage Diseases
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme.
- Trial name
- NCT04455230
- Testing
- FLT190
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
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
This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
- Trial name
- NCT01304277
- Testing
- agalsidase alfa
- Where
- 5 sites in Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
Safety Study of Replagal® Therapy in Children With Fabry Disease
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
- Trial name
- NCT01363492
- Testing
- Replagal (agalsidase alfa)
- Where
- 5 sites in United States
- Volunteers
- 15 people
- Sponsor
- Shire
Fabry Disease
Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease
This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease…
- Trial name
- NCT03454893
- Testing
- AVR-RD-01
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 15 people
- Sponsor
- AVROBIO
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
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
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
- Trial name
- NCT04049760
- Testing
- migalastat HCl 150 mg
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Amicus Therapeutics
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 Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
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
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A)…
- Trial name
- NCT01218659
- Testing
- migalastat hydrochloride, agalsidase
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 68 people
- Sponsor
- Amicus Therapeutics