A Study of Patients With Fabry Disease (US Specific)

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Amicus Therapeutics
Study ID
NCT06906367
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • migalastat HCl — DRUG
    Non-interventional study of participants receiving migalastat HCl 150 mg
  • ERT — DRUG
    Non-interventional study of participants receiving enzyme replacement therapy

Study Details

This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.

Key Dates

Start date
Feb 13, 2026
Status verified
Apr 2026
Primary completion
Jun 30, 2032
Completion
Jun 30, 2032

Study Design

Enrollment
450 participants (estimated)

Arms

  • Arm: Migalastat-treated
    Migalastat-treated patients at the time of enrollment who started the treatment within the 24 months prior to enrollment.
  • Arm: ERT-treated
    Patients receiving ERT at the time of enrollment who started the treatment within the 24 months prior to enrollment.
  • Arm: Untreated
    Untreated patients at the time of enrollment; these patients must never have been on therapy for Fabry disease prior to enrollment into the study and must meet criteria for receiving treatment with migalastat.

Primary Outcome Measure

Annualized rate of change in Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline and prospective up to 5 years ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
UAB Nephrology Research Clinic at Paula BuildingBirminghamAlabama35233-
Arkansas Children's HospitalLittle RockArkansas72202-
Emory GeneticsAtlantaGeorgia30322-
Washington University School of MedicineSt LouisMissouri63110-
New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical CenterNew YorkNew York10032-
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224-
Renal Disease Research InstituteDallasTexas75204-
Lysosomal and Rare Disorders Research and Treatment Center, Inc.FairfaxVirginia22030-

Find similar trials in Birmingham, AL

By research site
UAB Nephrology Research Clinic at Paula Building· Birmingham, ALArkansas Children's Hospital· Little Rock, AREmory Genetics· Atlanta, GAWashington University School of Medicine· St Louis, MONew York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center· New York, NYUPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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