AT1001-025
Research type
Research Study
Full title
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment
IRAS ID
265104
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Amicus Therapeutics, Inc
Eudract number
2018-001133-40
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
068456, US IND number
Duration of Study in the UK
2 years, 3 months, 30 days
Research summary
The purpose of this study is to find out whether a study drug called migalastat HCl (migalastat) can help people with Fabry disease, who also have severe kidney disease, and how safe it is.
Migalastat (Galafold™) is approved in the European Union, Switzerland, Israel, Japan, Australia, Canada, South Korea, Japan, and the United States for the long-term treatment of adults and adolescents aged 16 years and older with Fabry disease. However, it has not been approved for use in adults with Fabry disease causing severe kidney disease.
This study will be conducted in people who may have previously taken part in or are discontinuing from another Migalastat study. Subjects discontinuing from a current (ongoing) study will enter this study immediately to maintain stable treatment.
About 10 to 12 adult men and women will take part in this study at about 13 sites worldwide.
This study involves up to 6 visits to the hospital in which the study doctor will provide the patient with the study drug in either 4 days or every 7 days for 4 to 8 weeks depending on which treatment group the patient has been allocated too. Whether the patient takes Migalastat every 4 days or every 7 days will depend on the results of kidney function tests performed during the screening period of the study.
Blood an urine samples will be taken at most visits to assess the effectiveness of the study drug and observe any changes in the patients condition.
The objective of this study is to assess if Migalastat is safe and effective for use in adults with Fabry disease and severe kidney disease.
REC name
London - Brent Research Ethics Committee
REC reference
19/LO/1725
Date of REC Opinion
6 Jan 2020
REC opinion
Further Information Favourable Opinion