Open-Label Phase 3 Long-Term Safety Study of Migalastat (AT1001-041)
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|ClinicalTrials.gov Identifier: NCT01458119|
Recruitment Status : Terminated (Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons.)
First Posted : October 24, 2011
Results First Posted : October 2, 2018
Last Update Posted : October 2, 2018
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|Study Design||Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment|
Drug: migalastat hydrochloride
|Recruitment Details||Eighty-five eligible participants with Fabry disease who completed treatment with migalastat in one of three previous studies (AT1001-011 [NCT00925301], AT1001-012 [NCT01218659], or FAB-CL-205 [NCT00526071]) were enrolled in this open-label extension study to enable the collection of long-term safety and efficacy data.|
|Arm/Group Description||Participants received migalastat hydrochloride (migalastat) 150-milligram (mg) capsule (equivalent to 123 mg of migalastat) given orally once every other day (QOD) for a median duration of 23.5 months (m). Participants received an inactive reminder capsule on alternate days during the study.|
|Period Title: Overall Study|
|Received at Least 1 Dose of Study Drug||85|
|Intent to Treat (ITT) Population ||85|
|ITT-Amenable Population ||68|
|Reason Not Completed|
|Withdrawal Due to Nonamenable Mutation||1|
|Consent Withdrawn by Participant||8|
|Non-compliance With Study Drug||3|
Received at least 1 dose of study drug.
Amenable mutations based on Good Laboratory Practice (GLP) Human Embryonic Kidney (HEK) assay.
|Arm/Group Description||Participants received migalastat 150-mg capsule given orally QOD for a median duration of 23.5 m. Participants received an inactive reminder capsule on alternate days during the study.|
|Overall Number of Baseline Participants||85|
|Baseline Analysis Population Description||
ITT Population: All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study.
Mean (Standard Deviation)
Unit of measure: Years
|Number Analyzed||85 participants|
Sex: Female, Male
Measure Type: Count of Participants
Unit of measure: Participants
|Number Analyzed||85 participants|
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.
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
Results Point of Contact
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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.
|Responsible Party:||Amicus Therapeutics|
|Other Study ID Numbers:||
2011-004800-40 ( EudraCT Number )
|First Submitted:||October 20, 2011|
|First Posted:||October 24, 2011|
|Results First Submitted:||August 10, 2018|
|Results First Posted:||October 2, 2018|
|Last Update Posted:||October 2, 2018|