14 Participants Needed

Migalastat for Fabry Disease

Recruiting at 17 trial locations
AT
Overseen ByAmicus Therapeutics Patient Advocacy
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 8 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you do not take certain medications like Glyset, Replagal, Fabrazyme, or Zavesca while participating. If you are on these, you would need to stop them to join the trial.

What data supports the effectiveness of the drug Migalastat for Fabry Disease?

Migalastat has been shown to be effective in treating Fabry disease by increasing the activity of a specific enzyme that is deficient in patients with this condition. Clinical trials have demonstrated that it can reduce heart size and stabilize kidney function in patients with certain genetic mutations, making it a valuable treatment option for those with amenable mutations.12345

Is Migalastat safe for humans?

Research shows that Migalastat has been used in patients with Fabry disease for over 8 years, and it generally maintains kidney function without significant safety concerns. This suggests that it is generally safe for human use in this context.678910

How is the drug migalastat unique in treating Fabry disease?

Migalastat is unique because it is an oral drug that acts as a pharmacological chaperone, stabilizing specific mutant forms of the enzyme α-galactosidase A, which helps it function properly in patients with certain genetic mutations. Unlike traditional enzyme replacement therapies that require intravenous infusions, migalastat offers a more convenient oral administration for those with amenable mutations.1241112

What is the purpose of this trial?

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)

Research Team

CR

Clinical Research

Principal Investigator

Amicus Therapeutics

Eligibility Criteria

This trial is for adults with Fabry disease and severe kidney impairment, including those on stable hemodialysis. Participants must have a specific GLA gene variant treatable by migalastat and agree to use contraception if of reproductive potential. Exclusions include pregnancy, breastfeeding, unstable heart conditions, recent other investigational drugs or gene therapy, allergy to migalastat or similar drugs.

Inclusion Criteria

If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception
My records show a GLA variant treatable with migalastat.
I have end-stage renal disease and will complete all my dialysis sessions as prescribed.
See 5 more

Exclusion Criteria

I am allergic to migalastat or similar medications.
I do not have any health conditions that would stop me from following the study's requirements.
Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
See 10 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Baseline

Baseline visit to confirm enrollment eligibility and conduct initial assessments

Within 30 days of screening
1 visit (in-person)

Treatment

Participants receive migalastat based on their cohort assignment and eGFRMDRD result

12 months
Regular visits as per dosing schedule

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Migalastat HCl
Trial Overview The study tests the safety and how the body processes Migalastat HCl in individuals with Fabry disease who also have severe renal impairment (SRI) or are at the end stage of renal disease (ESRD). It's an open-label study where all participants know they're receiving Migalastat.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: Cohort 2: End-Stage Renal DiseaseExperimental Treatment1 Intervention
All hemodialysis subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat). Subjects will take 1 migalastat capsule orally with water every other week.
Group II: Cohort 1: Severe Renal ImpairmentExperimental Treatment1 Intervention
All subjects will receive migalastat 123 mg, equivalent to 150 mg migalastat HCl (hereafter, migalastat) at a dose regimen based on their eGFRMDRD result at Visit 1. Subjects will take 1 migalastat capsule orally with water either every 4 or 7 days.

Migalastat HCl is already approved in European Union, United States, Canada, Japan, Switzerland for the following indications:

🇪🇺
Approved in European Union as Galafold for:
  • Fabry disease
🇺🇸
Approved in United States as Galafold for:
  • Fabry disease
🇨🇦
Approved in Canada as Galafold for:
  • Fabry disease
🇯🇵
Approved in Japan as Galafold for:
  • Fabry disease
🇨🇭
Approved in Switzerland as Galafold for:
  • Fabry disease

Find a Clinic Near You

Who Is Running the Clinical Trial?

Amicus Therapeutics

Lead Sponsor

Trials
55
Recruited
2,700+

Findings from Research

Migalastat (Galafold™) has been approved in the EU for treating Fabry disease in patients with specific mutations that allow the drug to restore α-galactosidase activity.
Fabry disease is caused by a deficiency of α-galactosidase, leading to harmful accumulation of certain substances in cells, and migalastat offers a targeted treatment option for this rare disorder.
Migalastat: First Global Approval.Markham, A.[2018]
Migalastat (Galafold®) is an effective oral treatment for Fabry disease that can improve enzyme activity in patients with specific mutations, showing benefits such as reduced left ventricular mass and stabilized kidney function.
Switching from traditional enzyme replacement therapy to migalastat has been shown to be safe and effective, with positive outcomes observed in both treatment-naive patients and those previously on enzyme replacement therapy.
Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?Perretta, F., Jaurretche, S.[2023]
Migalastat is a novel oral treatment for Fabry disease that works by stabilizing mutant forms of the enzyme alpha-galactosidase A, allowing them to function better and reduce harmful substance accumulation in cells.
In pivotal Phase III studies, migalastat showed promising results, being more effective than enzyme replacement therapy in reducing left ventricular mass in patients with cardiac hypertrophy, while also demonstrating comparable effects on kidney function.
[Chaperone molecules: The example of Fabry disease].Barbey, F., Monney, P., Dormond, O.[2021]

References

Migalastat: First Global Approval. [2018]
Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today? [2023]
[Chaperone molecules: The example of Fabry disease]. [2021]
Migalastat: A Review in Fabry Disease. [2020]
Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study. [2022]
Safety and Efficacy of Intermittent High-Dose Liposomal Amphotericin B Antifungal Prophylaxis in Haemato-Oncology: An Eight-Year Single-Centre Experience and Review of the Literature. [2021]
In Vitro and In Vivo Amenability to Migalastat in Fabry Disease. [2020]
Long-term follow-up of renal function in patients treated with migalastat for Fabry disease. [2023]
Comparative efficacy, toxicity and biodistribution of the liposomal amphotericin B formulations Fungisome® and AmBisome® in murine cutaneous leishmaniasis. [2021]
10.United Statespubmed.ncbi.nlm.nih.gov
Comparison of in vitro antifungal activities of free and liposome-encapsulated nystatin with those of four amphotericin B formulations. [2020]
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. [2021]
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. [2022]
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