Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

Recruiting Phase 2 Phase 3 Interventional Study
Fabry Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
13 – 70
Sex
Any
Trial phase
Phase 2/3
Study type
Interventional
Purpose
Treatment
Participants needed
16 (estimated)
Sponsor
Chiesi Farmaceutici S.p.A. · Industry
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About This Trial

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. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.

Trial Locations
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Eligibility Criteria
Inclusion criteria (all subjects) 1. Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent 2. A documented diagnosis of Fabry disease, as determined by the following: * Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity…
Contacts

Chiesi Clinical Trial

+3905212791

clinicaltrials_info@chiesi.com

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