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An Open-Label Study Of The Safety, Pharmacokinetics, Pharmacodynamics, And Efficacy Of 12-Month Treatment With Migalastat In Pediatric Subjects (Aged 2 To < 12 Years) With Fabry Disease And Amenable GLA Variants

Status: Recruiting

The purpose of this study is to evaluate the safety and effectiveness of migalastat, an investigational treatment for children with Fabry disease. Researchers will also study how the medication is processed by the body and how it affects the disease. Participants will receive migalastat for up to 12 months and complete regular study visits and health assessments throughout the study.

I'm interested

Sex: Male or Female
Age Group: Up to 18 years old
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 2 to under 12 years of age
• diagnosis of Fabry disease
• see the ClinicalTrials.gov listing for complete inclusion criteria
Exclusion Criteria:

• moderate to severe kidney disease or requiring dialysis or a kidney transplant
• previous gene therapy or certain medications that may interfere with the study
• pregnant or breastfeeding
• see the ClinicalTrials.gov listing for complete exclusion criteria
Interventions:

Drug: Migalastat HCl 20 mg

Conditions:

Children's Health, Rare Diseases, Rare Diseases

Keywords:

Fabry disease

Study Contact: Brenda Diethelm-Okita - dieth001@umn.edu
Principal Investigator: Chester Whitley, MD, PhD
Phase: PHASE3
IRB Number: STUDY00025577
See this study on ClinicalTrials.gov

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