Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00925301
• Date of registration: 19/06/2009
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
• Scientific title: A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations
• Date of first enrolment: October 23, 2009
• Target sample size: 67
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00925301
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Brazil, Canada, Chile, Denmark, Egypt, France, Germany, Israel, Italy, Netherlands, Poland, South Africa, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Medical Monitor, Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female between the ages of 16 and 74 diagnosed with Fabry disease.

- Confirmed mutant form of a-galactosidase A shown to be responsive to migalastat in
vitro.

- Participant has never been treated with enzyme replacement therapy (ERT) or has not
received ERT for 6 consecutive months or longer before the screening visit for the
study.

- Urine GL-3 =4 times the upper limit of normal at screening.

- Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor
blockers must be on a stable dose for a minimum of 4 weeks before the baseline visit.

- Females who can become pregnant and all males agree to be sexually abstinent or use
medically accepted methods of birth control during the study and for 30 days after
study completion.

- Participant is willing and able to provide written informed consent and assent, if
applicable.

Exclusion Criteria:

- Participant has undergone or is scheduled to undergo kidney transplantation, or is
currently on dialysis.

- Estimated glomerular filtration rate <30 milliliters per minute per 1.73 meters
squared (chronic kidney disease Stage 4 or 5) based on the Modification of Diet in
Renal Disease equation at screening.

- Pregnant or breast-feeding.

- History of allergy or sensitivity to study medication (including excipients) or other
iminosugars (for example, miglustat, miglitol).

- Participant is treated or has been treated with any investigational drug within 30
days of study start.

- Participant is currently treated or has ever been treated with migalastat.



Age minimum: 16 Years
Age maximum: 74 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Placebo

Drug: migalastat hydrochloride

Primary Outcome(s)

Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions [Time Frame: Baseline, Month 6]

Secondary Outcome(s)

Change From Baseline Through Month 24 In Urine GL-3 Levels [Time Frame: Baseline, Months 6, 12, and 24]

Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 [Time Frame: Baseline, Month 6]

Secondary ID(s)

AT1001-011

2009-013459-31

FACETS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/10/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00925301