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

NCT ID: NCT01458119

Last Updated: 2018-10-02

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 85 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-10-14
• Study Completion Date: 2016-02-17

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.

Detailed Description

Study AT1001-041 was an open-label, noncomparative, multicenter, long-term extension study for participants with Fabry disease who completed treatment in one of three previous trials of migalastat (AT1001-011 [NCT00925301], AT1001-012 [NCT01218659], or FAB-CL-205 [NCT00526071]). In these trials, migalastat was given as monotherapy. This was an extension study designed to evaluate the long-term safety and efficacy of migalastat for the treatment of Fabry disease. Study visits occurred every 6 months (m). Visit evaluations included physical examinations, clinical laboratory parameters, adverse events, and participant reported outcomes.

The study consisted of a Baseline Visit, which was performed at the time of the final visit of the previous study, followed by clinic visits every 6 m for each year of the study. Study assessments included a physical examination, echocardiography, laboratory parameters, and participant-reported outcomes. Since participants enrolled in the study at varying time points based on the completion of the preceding migalastat study, treatment duration varied among participants. No maximum treatment duration was defined. There were no control groups in this study; all participants received migalastat as a 150-mg capsule taken orally once every other day (QOD) and inactive reminder capsules on alternate days.

The sponsor (Amicus Therapeutics) discontinued Study AT1001-041 for logistical reasons and not due to either safety concerns or lack of efficacy. For participants who were ongoing in Study AT1001-041 at the time of discontinuation, the investigators were offered participation in a similar open-label, long-term migalastat treatment study (AT1001-042 [NCT02194985]) for participants ongoing at discontinuation.

Conditions

Fabry Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Migalastat

Migalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.

Group Type: EXPERIMENTAL

migalastat hydrochloride

Intervention Type: DRUG

Oral capsule QOD

Interventions

migalastat hydrochloride

Oral capsule QOD

Intervention Type: DRUG

Other Intervention Names

AT1001; Galafold; Migalastat

Eligibility 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.

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amicus Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor, Clinical Research

Role: STUDY_DIRECTOR

Amicus Therapeutics

Locations

Atlanta, Georgia, United States

Chicago, Illinois, United States

Kansas City, Kansas, United States

Boston, Massachusetts, United States

Grand Rapids, Michigan, United States

New York, New York, United States

Portland, Oregon, United States

Pittsburgh, Pennsylvania, United States

Dallas, Texas, United States

Fairfax, Virginia, United States

Seattle, Washington, United States

Pilar, , Argentina

Adelaide, , Australia

Parkville, , Australia

Edegem, , Belgium

Porto Alegre, , Brazil

Montreal, , Canada

Copenhagen, , Denmark

Cairo, , Egypt

Garches, , France

Roma, , Italy

Barcelona, , Spain

Ankara, , Turkey (Türkiye)

London, , United Kingdom

Salford, , United Kingdom

Countries

United States; Argentina; Australia; Belgium; Brazil; Canada; Denmark; Egypt; France; Italy; Spain; Turkey (Türkiye); United Kingdom

References

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.

Reference Type: DERIVED

PMID: 27509102 (View on PubMed)

Other Identifiers

2011-004800-40

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AT1001-041

Identifier Type: -

Identifier Source: org_study_id