Health TrialsbyHealthPanel

A Study of Patients With Fabry Disease (US Specific)

ConditionFabry Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorAmicus Therapeutics

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients with Fabry disease 18 years or older with amenable GLA variants who have commenced commercial migalastat treatment within 24 months preceding enrollment, who have an eGFR greater than or equal to 30 mL/min/1.73 m2 at the time of enrollment and are still taking migalastat at the time of enrollment, or who are starting migalastat at the time of enrollment, excluding those who participated in a prior migalastat clinical trial

a decrease in annualized rate of decline eGFRCKD-EPI of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment

microalbuminuria/macroalbuminuria (≥ 30 mg/24 h or ≥ 20 mg on first morning urine) or urine ACR of ≥ 30 mg/g (via spot urine collection) at any time prior to or at enrollment

proteinuria (> 0.5 g/g UPCR) any time prior to or at enrollment

Disqualifiers

None

Trial population

Globally, approximately 450 patients with classic and late onset/nonclassic phenotypes of Fabry disease will be enrolled, with approximately 250 patients in the migalastat-treated group, 100 in the ERT-treated group, and approximately 100 patients in the untreated group.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • migalastat HCl

    Drug

    Non-interventional study of participants receiving migalastat HCl 150 mg

  • ERT

    Drug

    Non-interventional study of participants receiving enzyme replacement therapy

Treatment groups

450 Participants
are divided into 3 treatment groups
Group A: Migalastat-treated1 intervention
Group B: ERT-treated1 intervention
Group C: Untreated0 interventions

Trial outcomes

Primary outcomes

1

Annualized rate of change in Estimated Glomerular Filtration Rate (eGFR)

Annualized rate of change in eGFR(CKD-EPI) over time from study enrollment for the comparison between migalastat-treated and untreated patients who have risk factors for eGFR decline

Time frame
Baseline and prospective up to 5 years

Secondary outcomes

1

Time to the first Fabry-associated clinical event (FACE)

Time to first FACE, which are cardiac, cerebrovascular, and renal events, and death due to FACEs, from enrollment in the study to compare between migalastat-treated and untreated patients.

Time frame
Baseline and prospective up to 5 years
2

Time to the first Fabry-associated clinical event (FACE)

Time to first FACE, which are cardiac, cerebrovascular, and renal events, and death due to FACEs, from start of treatment to compare between migalastat-treated and ERT-treated patients.

Time frame
Retrospective and prospective up to 5 years
3

Annualized rate of change in Estimated Glomerular Filtration Rate (eGFR)

Annualized rate of change in eGFR(CKD-EPI) from start of treatment over time for the comparison between migalastat-treated and ERT-treated patients

Time frame
Retrospective and prospective up to 5 years
4

Incidence and occurrence of FACE

Incidence and occurrence of FACE will be evaluated overall, and separately by cardiac, cerebrovascular, and renal clinical events (including death in these categories)

Time frame
Retrospective and prospective up to 5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Amicus Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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