NCT06906367 A Study of Patients With Fabry Disease (US Specific)
| NCT ID | NCT06906367 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Amicus Therapeutics |
| Condition | Fabry Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 450 participants |
| Start Date | 2026-02-13 |
| Primary Completion | 2032-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 450 participants in total. It began in 2026-02-13 with a primary completion date of 2032-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
I. Migalastat-treated patients (Commercial only participants) 1. 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 2. Patients who show a decline in their Fabry disease symptomatology based on any of the following: 1. a decrease in annualized rate of decline eGFRCKD-EPI of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment 2. 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 3. proteinuria (\> 0.5 g/g UPCR) any time prior to or at enrollment 4. males with classic Fabry disease phenotype II. Migalastat-treated patients who are not considered to be in renal decline (Commercial migalastat users only) 1\. Patients with Fabry disease with amenable GLA variants who have been on commercial migalastat regardless of the duration of treatment III. Migalastat-treated patients (Prior clinical trial participants) 1. Patients with Fabry disease 18 years or older who had commenced treatment with migalastat while in a clinical trial and were exposed to treatment for at least 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 who are still taking migalastat at the time of enrollment, having switched to commercial product IV. Untreated patients 1. Patients with Fabry disease 18 years or older with amenable GLA variants, who have never been on treatment for Fabry disease, who have an eGFR greater than or equal to 30 mL/min/1.73 m2 at the time of enrollment, and who meet local treatment guidelines for Fabry disease 2. Patients who show a decline in their Fabry disease symptomatology based on any of the following: 1. a decrease in annualized rate of decline eGFRCKD-EPI of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment 2. 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 3. proteinuria (\> 0.5 g/g UPCR) any time prior to or at enrollment 4. males with classic Fabry disease phenotype V. ERT-treated patients 1. Patients with Fabry disease 18 years or older who have commenced ERT 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 being treated with ERT at the time of enrollment, and who have amenable GLA variants 2. Patients who show a decline in their Fabry disease symptomatology based on any of the following: 1. a decrease in eGFRCKD-EPI annualized rate of decline of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment 2. 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 3. proteinuria (\> 0.5 g/g UPCR) any time prior to or at enrollment 4. males with classic Fabry disease phenotype All patients 1. All treated and untreated patients with Fabry disease who are enrolled in the study must be able to understand and provide written informed consent or assent. Exclusion Criteria 1\. Patients who currently are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment
Contact & Investigator
Clinical Research
STUDY DIRECTOR
Amicus Therapeutics
Frequently Asked Questions
Who can join the NCT06906367 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Fabry Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06906367 currently recruiting?
Yes, NCT06906367 is actively recruiting participants. Contact the research team at patientadvocacy@amicusrx.com for enrollment information.
Where is the NCT06906367 trial being conducted?
This trial is being conducted at Birmingham, United States, Little Rock, United States, Atlanta, United States, St Louis, United States and 4 additional locations.
Who is sponsoring the NCT06906367 clinical trial?
NCT06906367 is sponsored by Amicus Therapeutics. The principal investigator is Clinical Research at Amicus Therapeutics. The trial plans to enroll 450 participants.