A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Recruiting 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.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
2 – 11
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
8 (estimated)
Sponsor
Amicus Therapeutics · Industry
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial

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 Locations
Loading…

Loading trial locations…

Eligibility Criteria
Inclusion Criteria * Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \< 12 years at randomization (subjects aged 11 years must have birthdays \> 30 days after randomization) * Subject's parent or legally authorized representative is willing and able to provide writt…
Contacts

Amicus Therapeutics Patient Advocacy

609-662-2000

patientadvocacy@amicusrx.com

CONTACT