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

Recruiting in Palo Alto (17 mi)

+18 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Amicus Therapeutics

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.

Eligibility Criteria

Inclusion Criteria

Key Inclusion Criteria

Willing and able to provide written consent or assent (participant and parent/legal guardian, as applicable)

Male or female between 12 and <18 years of age diagnosed with Fabry disease

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Treatment Details

Interventions

Migalastat HCl 150 mg (Chaperone Therapy)

Participant Groups

1Treatment groups

Experimental Treatment

Group I: migalastat HCl 150 mgExperimental Treatment1 Intervention

One migalastat 123 milligrams (mg) capsule equivalent to 150 mg migalastat hydrochloride (HCl) (herein referred to as "migalastat") was administered every other day for 12 months.