Migalastat for Fabry Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the long-term effects of treatments for Fabry disease, focusing mainly on the drug migalastat. Researchers aim to evaluate the effectiveness and safety of these treatments and their impact on patients' quality of life. The trial includes different groups: those currently treated with migalastat or enzyme replacement therapy (ERT), and those who have not yet received any treatment. Individuals with Fabry disease who have certain genetic characteristics and experience worsening symptoms, such as kidney issues or protein in urine, may be suitable for this study. As a Phase 3 trial, this study serves as the final step before FDA approval, offering participants a chance to contribute to the validation of a promising treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, it seems that patients already on migalastat or ERT (enzyme replacement therapy) can continue their treatment while participating in the study.
What is the safety track record for migalastat and ERT treatments?
A previous study found that migalastat was well tolerated by teenagers with Fabry disease, meaning it didn't cause any unexpected problems. Research also shows that in women, using migalastat for about five years did not lead to any new safety concerns. Other studies confirm that migalastat is generally well tolerated. Overall, existing research suggests that migalastat is safe.12345
Why are researchers excited about this trial?
Researchers are excited about migalastat for Fabry disease because it works differently from the standard enzyme replacement therapies (ERT) like agalsidase beta. Unlike ERT, which directly replaces the deficient enzyme, migalastat is a small molecule that stabilizes the patient's own dysfunctional enzyme, helping it function better. This approach can potentially offer a more convenient oral treatment instead of regular infusions. Additionally, migalastat may improve the body's ability to target and treat the underlying cause of the disease at a cellular level.
What evidence suggests that this trial's treatments could be effective for Fabry disease?
Research has shown that migalastat effectively treats Fabry disease. In this trial, one group of participants will receive migalastat, which studies have found helps maintain stable kidney function by preserving healthy eGFR levels, a measure of kidney health. Migalastat is generally well tolerated over time, with few serious side effects. It also helps manage symptoms in various parts of the body for people with Fabry disease. This suggests that migalastat could be a promising option for those seeking to manage their condition effectively.12367
Who Is on the Research Team?
Clinical Research
Principal Investigator
Amicus Therapeutics
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Enrollment
Participants are enrolled in the study and assigned to treatment groups
Observation
Participants are observed for long-term effectiveness, safety, and health-related quality of life
Follow-up
Participants are monitored for safety and effectiveness after the main observation period
What Are the Treatments Tested in This Trial?
Interventions
- ERT
- Migalastat
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
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.
Migalastat-treated patients at the time of enrollment who started the treatment within the 24 months prior to enrollment.
Patients receiving ERT at the time of enrollment who started the treatment within the 24 months prior to enrollment.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Amicus Therapeutics
Lead Sponsor
Citations
Safety and efficacy of migalastat in adolescent patients with ...
Treatment with migalastat was well tolerated in this adolescent population with no new or unexpected safety findings observed.
Long-term efficacy of migalastat in females with Fabry ...
Migalastat treatment preserved renal function in females with Fabry disease, with the rate of eGFRCKD-EPI decline per year similar to that ...
Migalastat as oral monotherapy for Fabry disease
Conclusions: Migalastat was generally well tolerated in both trials. Subsequent extension publications showed similar results, confirming the ...
Renal and multisystem effectiveness of 3.9 years ...
Renal and multisystem effectiveness of 3.9 years of migalastat in a global real-world cohort: Results from the followME Fabry Pathfinders registry
Long-term multisystemic efficacy of migalastat on Fabry ...
Results: During long-term migalastat treatment, 17 patients (17.5%) experienced 22 FACEs and there were no deaths. The incidence rate of FACEs ...
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs ...
This is a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, PK, PD, and efficacy of 12 months of migalastat treatment ...
Treatment of Fabry's Disease with the Pharmacologic ...
Migalastat, an oral pharmacologic chaperone, stabilizes specific mutant forms of α-galactosidase, increasing enzyme trafficking to lysosomes.
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