Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Amicus Therapeutics

Status and phase: Active, not recruiting; Phase 3

Conditions: Fabry Disease

Treatments: Drug: migalastat HCl 150 mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT04049760

2019-000222-21 (EudraCT Number)

AT1001-036

Details and patient eligibility

About

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

Enrollment: 15 patients

Sex: All

Ages: 12 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable

Exclusion criteria

• Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
• Has advanced kidney disease requiring dialysis or kidney transplantation
• History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
• Has received any gene therapy at any time or anticipates starting gene therapy during the study period
• Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
• Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
• Subject is treated or has been treated with any investigational/experimental drug, biologic or device within 30 days before screening
• Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
• Pregnant or breast-feeding
• Otherwise unsuitable for the study in the opinion of the investigator

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

migalastat HCl 150 mg

Experimental group

Description:

One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.

Treatment:

Drug: migalastat HCl 150 mg