Open-Label Phase 3 Long-Term Safety Study of Migalastat

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01458119
• Date of registration: 20/10/2011
• Prospective Registration: No
• Primary sponsor: Amicus Therapeutics
• Public title: Open-Label Phase 3 Long-Term Safety Study of Migalastat AT1001-041
• Scientific title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
• Date of first enrolment: October 14, 2011
• Target sample size: 85
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01458119
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey, United Kingdom, United States

Contacts

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

Key inclusion & exclusion criteria

Inclusion Criteria:

- Completed migalastat treatment in a previous Fabry disease protocol

- Both male and female participants were enrolled

- Age 16 years or older

- Male and female participants had to agree to use protocol-identified acceptable
contraception

Exclusion Criteria:

- Estimated glomerular filtration rate (eGFR) in the previous study was <30
milliliters/minute/1.73 square meters (mL/min/1.73 m^2) unless there was a measured
GFR available within 3 m of the Baseline Visit that was >30 mL/min/1.73 m^2

- Had undergone, or was scheduled to undergo, kidney transplantation or was currently on
dialysis

- Pregnant or breast feeding

- Treated with another investigational drug (except migalastat) within 30 days of study
start

- Unable to comply with study requirements, or deemed otherwise unsuitable for study
entry, in the opinion of the investigator

- Had documented transient ischemic attack, stroke, unstable angina, or myocardial
infarction within the 12 m before the Baseline Visit

- Had clinically significant, unstable cardiac disease in the opinion of the
investigator

- Had a history of allergy or sensitivity to migalastat (including excipients) or to
other iminosugars

- Required treatment with Glyset (miglitol) or Zavesca (miglustat)

- Had any intercurrent illness or condition that may have precluded the participant from
fulfilling the protocol requirements

- Had a severe or unsuitable concomitant medical condition

- Had a clinically significant abnormal laboratory value and a clinically significant
electrocardiogram finding at the Baseline Visit.



Age minimum: 16 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: migalastat hydrochloride

Primary Outcome(s)

Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) [Time Frame: Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months.]

Secondary Outcome(s)

Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) [Time Frame: Baseline, Every 6 m until the End of Study (42 m)]

Secondary ID(s)

2011-004800-40

AT1001-041

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 02/10/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01458119