Your session is about to expire
← Back to Search
Migalastat for Fabry Disease
Study Summary
This trial will test the safety and how well the drug migalastat HCl works in people with Fabry disease and severe kidney impairment.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2019 Phase 3 trial • 84 Patients • NCT02194985Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- 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.My records show a GLA variant treatable with migalastat.I am currently taking or have taken Zavesca® (miglustat).I have end-stage renal disease and will complete all my dialysis sessions as prescribed.I have never had gene therapy and don't plan to during the study.I have had a stroke, heart attack, or severe chest pain.I have a serious heart condition that is not stable.Your kidney function, measured by eGFR, is very low (less than 30 mL/min/1.73 m2) on at least one occasion in the last 3 months and at the start of the study.I am currently undergoing peritoneal dialysis.I have been on a stable dialysis schedule 2 or 3 times a week for at least 2 months.I need treatment with Glyset, Replagal, or Fabrazyme.I have had a kidney transplant.I am 18 or older and have been diagnosed with Fabry disease.I am currently pregnant or breastfeeding.I have end-stage renal disease and can do 4 dialysis sessions every 2 weeks.
- Group 1: Cohort 1: Severe Renal Impairment
- Group 2: Cohort 2: End-Stage Renal Disease
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Why was this research project undertaken?
"This study, which will be evaluated over a Baseline through Month 12 time frame, aims to assess the pharmacodynamics of migalastat in subjects with Fabry disease and severe renal impairment. Secondary outcomes include change from baseline in plasma globotriaosylsphingosine (lyso-Gb3), number of subjects with abnormal electrocardiogram (ECG) findings as a measure of safety and tolerability, and change from baseline eGFR based on the Chronic Kidney Disease Epidemiology Collaboration equation (eGFRCKD-EPI)."
Are investigators still looking for more test subjects?
"Unfortunately, this particular clinical trial is no longer recruiting patients. The original posting was on December 1st, 2022 and the most recent update was on November 7th, 2022. If you are looking for similar studies, there are 517 trials for fabry disease and 2 trials concerning migalastat HCl 150 mg that are still enrolling participants."
Are there other experiments that have been done using migalastat HCl 150 mg?
"At the moment, there are two ongoing clinical trials investigating migalastat HCl 150 mg. 2 of these active studies have progressed to Phase 3 testing. Although most of the research is conducted in Minneapolis, Minnesota, 23 different locations are running similar investigations."
Have these treatments been used before in other clinical trials?
"Amicus Therapeutics sponsored the first study for migalastat HCl 150 mg in 2019. This initial research was followed by Phase 3 drug approval in 2019. Currently, there are 2 active studies being conducted across 11 cities and 8 countries."
Are there any know risks associated with taking migalastat HCl 150 mg?
"There is some evidence, from both Phase 3 data and other rounds of testing, that migalastat HCl 150 mg is safe."
How many people are participating in this clinical trial?
"This particular trial is not presently admitting patients. The study was first posted on December 1st, 2022 and last updated on November 7th, 2022. However, there are 517 other trials for fabry disease and 2 studies involving migalastat HCl 150 mg that are still recruiting patients."
In how many different medical clinics is this study being run?
"Currently, there are six centres recruiting patients for this trial. These include the Lysosomal and Rare Disorders Research and Treatment Centre, Inc in Fairfax, Emory University in Atlanta, and Emory SOM in Pittsburgh; as well as 6 other locations."
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger