Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
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
84
14 countries
27
Brief Summary
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl.
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 Mar 2015
Longer than P75 for phase_3
27 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
First Submitted
Initial submission to the registry
July 17, 2014
CompletedFirst Posted
Study publicly available on registry
July 21, 2014
CompletedStudy Start
First participant enrolled
March 14, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 23, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
October 23, 2019
CompletedResults Posted
Study results publicly available
December 21, 2020
CompletedDecember 21, 2020
November 1, 2020
4.6 years
July 17, 2014
October 21, 2020
November 25, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number Of Participants Experiencing Adverse Events (AEs)
An 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 time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
Secondary Outcomes (8)
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In eGFR At End Of Study
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Change From Baseline In 24-hour Urine Protein To End Of Study
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
- +3 more secondary outcomes
Study Arms (1)
Migalastat HCl 150 mg
EXPERIMENTALMigalastat HCl 150 milligram (mg).
Interventions
Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.
Eligibility Criteria
You may qualify if:
- Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
- Male and female participant agreed to use protocol-identified acceptable contraception
- Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
You may not qualify if:
- Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was \<30 milliliter (mL)/minute (min)/1.73 meters squared (m\^2); unless there was measured GFR available within 3 months of Baseline Visit, which was \>30 mL/min/1.73 m\^2
- Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
- Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
- Participant had clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
- Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
- Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- Participants with severe or unsuitable concomitant medical condition
- Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
Clinical Study Site
Atlanta, Georgia, 30322, United States
Clinical Study Site
Kansas City, Kansas, 66160, United States
Clinical Study Site
Grand Rapids, Michigan, 49525, United States
Clinical Study Site
New York, New York, 10016, United States
Clinical Study Site
Portland, Oregon, 97239, United States
Clinical Study Site
Dallas, Texas, 75226, United States
Clinical Study Site
Fairfax, Virginia, 22030, United States
Clinical Study Site
Pilar, B1629ODT, Argentina
Clinical Study Site
Adelaide, 5000, Australia
Clinical Study Site
Parkville, 3050, Australia
Clinical Study Site
Vienna, 1090, Austria
Clinical Study Site
Edegem, 2650, Belgium
Clinical Study Site
Porto Alegre, 90035-903, Brazil
Clinical Study Site
Montreal, Quebec, H4J 1C5, Canada
Clinical Study Site
Copenhagen, 2100, Denmark
Clinical Study Site
Cairo, 11451, Egypt
Clinical Study Site
Garches, 92380, France
Clinical Study Site
Lille, 59037, France
Clinical Study Site
Florence, 50134, Italy
Clinical Study Site
Roma, 00168, Italy
Clinical Study Site
Suita, Osaka, 565-0871, Japan
Clinical Study Site
Niigata, 951-8520, Japan
Clinical Study Site
Osaka, 545-8586, Japan
Clinical Study Site
Tokyo, 105-8471, Japan
Clinical Study Site
Barcelona, 08025, Spain
Clinical Study Site
Ankara, 06500, Turkey (Türkiye)
Clinical Study Site
London, NW3 2QG, United Kingdom
Clinical Study Site
London, WC1N3BG, United Kingdom
Related Publications (1)
Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.
PMID: 31889231DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
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
- Expanded Access
- Yes
Study Record Dates
First Submitted
July 17, 2014
First Posted
July 21, 2014
Study Start
March 14, 2015
Primary Completion
October 23, 2019
Study Completion
October 23, 2019
Last Updated
December 21, 2020
Results First Posted
December 21, 2020
Record last verified: 2020-11