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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-11

Study Completion Date

2024-11-29

Brief Summary

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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

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Detailed Description

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Conditions

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Fabry Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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migalastat HCl 150 mg

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

Group Type EXPERIMENTAL

migalastat HCl 150 mg

Intervention Type DRUG

migalastat HCl 150 mg capsule

Interventions

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migalastat HCl 150 mg

migalastat HCl 150 mg capsule

Intervention Type DRUG

Other Intervention Names

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migalastat AT1001

Eligibility Criteria

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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
* If of reproductive potential, both male and female subjects agreed to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat

Exclusion Criteria

* Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2
* Subject had advanced kidney disease requiring dialysis or kidney transplantation
* Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
* Subject anticipated starting gene therapy during the study period
* Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
* Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
* Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
* Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
* Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
* In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements

Minimum Eligible Age

12 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No