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Fabry Disease Clinical Trials

A listing of Fabry Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (5) clinical trials

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with ...

Phase

Study of the Safety Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients

This is an open-label switchover study to assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa treatment of 2 mg/kg every 4 weeks in patients previously treated with enzyme-replacement therapy (ERT): agalsidase alfa or agalsidase beta, for at least 3 years and on a stable dose (>80% labelled dose/kg) for ...

Phase

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.

Phase

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Phase

Safety Pharmacokinetics Pharmacodynamics and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

This is a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects 12 to <18 years of age and weighing 45 kg (99 pounds) with Fabry disease and with amenable GLA variants. Subjects must be either nave to ...

Phase