Birmingham, ALNCT06906367Now EnrollingIRB Ready

Fabry Disease Clinical Trial in Birmingham, AL

Access cutting-edge fabry disease treatment through this clinical trial at a research site in Birmingham. Study-provided care at no cost to qualified participants.

Sponsored by Amicus Therapeutics

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Expert Care in Birmingham

Access fabry disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related fabry disease treatment provided free

Apply for This Birmingham Location

Check if you qualify for this fabry disease clinical trial in Birmingham, AL

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Why Participate?

  • No-Cost Study Care

  • Local to Birmingham

    Convenient for AL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Birmingham site if eligible
  4. 4Begin participation

About This Fabry Disease Study in Birmingham

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.

Sponsor: Amicus Therapeutics

Who Can Participate

Inclusion Criteria

I. Migalastat-treated patients (Commercial only participants)
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
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
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
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)
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
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
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
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
males with classic Fabry disease phenotype V. ERT-treated patients
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
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
a decrease in eGFRCKD-EPI annualized rate of decline 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
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

Exclusion Criteria

1\. Patients who currently are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Birmingham?

Yes, this clinical trial (NCT06906367) has an active research site in Birmingham, AL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Fabry Disease Treatment Options in Birmingham, AL

If you're searching for fabry disease treatment options in Birmingham, AL, this clinical trial (NCT06906367) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Birmingham research site is actively enrolling participants for this clinical trial. You'll receive care from experienced fabry disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all fabry disease clinical trials near you to find additional studies recruiting in your area.

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