Study Stopped
Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons.
Open-Label Phase 3 Long-Term Safety Study of Migalastat
AT1001-041
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
2 other identifiers
interventional
85
13 countries
25
Brief Summary
This was a long-term, open-label study of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) in participants with Fabry disease who completed treatment in a previous monotherapy trial with migalastat.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Oct 2011
Typical duration for phase_3
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 14, 2011
CompletedFirst Submitted
Initial submission to the registry
October 20, 2011
CompletedFirst Posted
Study publicly available on registry
October 24, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 17, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
February 17, 2016
CompletedResults Posted
Study results publicly available
October 2, 2018
CompletedOctober 2, 2018
October 1, 2018
4.3 years
October 20, 2011
August 10, 2018
October 1, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at the time of reporting; visits typically occurred every 6 months. A TEAE was defined as an AE starting on or after the first study drug administration date. Serious AEs were life-threatening or resulted in death, persistent or significant incapacitation, inpatient or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: minimal discomfort, does not interfere with normal everyday activities; Moderate: sufficiently discomforting, interferes with normal everyday activities; Severe: prevents normal everyday activities. The number of participants experiencing TEAEs is presented for those who received migalastat treatment. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
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 Outcomes (1)
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Baseline, Every 6 m until the End of Study (42 m)
Study Arms (1)
Migalastat
EXPERIMENTALMigalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (25)
Unknown Facility
Atlanta, Georgia, 30322, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
Kansas City, Kansas, 66160, United States
Unknown Facility
Boston, Massachusetts, 02114, United States
Unknown Facility
Grand Rapids, Michigan, 49525, United States
Unknown Facility
New York, New York, 10016, United States
Unknown Facility
Portland, Oregon, 97239, United States
Unknown Facility
Pittsburgh, Pennsylvania, 15213, United States
Unknown Facility
Dallas, Texas, 75246, United States
Unknown Facility
Fairfax, Virginia, 22030, United States
Unknown Facility
Seattle, Washington, 98195, United States
Unknown Facility
Pilar, B1629ODT, Argentina
Unknown Facility
Adelaide, 5000, Australia
Unknown Facility
Parkville, 3050, Australia
Unknown Facility
Edegem, 2650, Belgium
Unknown Facility
Porto Alegre, 90035-903, Brazil
Unknown Facility
Montreal, H4J 1C5, Canada
Unknown Facility
Copenhagen, 2100, Denmark
Unknown Facility
Cairo, 11451, Egypt
Unknown Facility
Garches, 92380, France
Unknown Facility
Roma, 00168, Italy
Unknown Facility
Barcelona, 08025, Spain
Unknown Facility
Ankara, 06500, Turkey (Türkiye)
Unknown Facility
London, NW3 2QG, United Kingdom
Unknown Facility
Salford, M6 8HD, United Kingdom
Related Publications (1)
Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.
PMID: 27509102DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
The study was discontinued for logistical reasons and not due to either safety concerns or lack of efficacy. The investigators were offered participation in a similar long-term migalastat study for participants ongoing at discontinuation.
Results Point of Contact
- Title
- Medical Affairs
- Organization
- Amicus Therapeutics
Study Officials
- STUDY DIRECTOR
Medical Monitor, Clinical Research
Amicus Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 20, 2011
First Posted
October 24, 2011
Study Start
October 14, 2011
Primary Completion
February 17, 2016
Study Completion
February 17, 2016
Last Updated
October 2, 2018
Results First Posted
October 2, 2018
Record last verified: 2018-10