Phase 3
Completed N=84
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Source: ClinicalTrials.gov NCT02194985 ↗Enrolled (actual)
84
Serious AEs
31.0%
Results posted
Dec 2020
Primary outcomePrimary: Number Of Participants Experiencing Adverse Events (AEs) — 80; 26; 1; 0 Participants
◆ Published Evidence
Emerging
13citations · ~2 / year
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
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.
Linked Publications
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Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Experiencing Adverse Events (AEs) |
80; 26; 1; 0; 24; 56 | — |
| SECONDARY Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) |
-1.6107; -1.3528 | — |
| SECONDARY Change From Baseline In eGFR At End Of Study |
79.0; 75.8; -1.4; 84.70; 82.02; -0.93 | — |
| SECONDARY Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study |
13.317; 11.758; -1.054 | — |
| SECONDARY Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study |
6.882; 5.290; -1.375 | — |
| SECONDARY Change From Baseline In 24-hour Urine Protein To End Of Study |
478.9; 394.0; 5.4 | — |
| SECONDARY Change From Baseline In Left Ventricular Mass (LVM) To End Of Study |
178.879; 157.896; -0.803 | — |
| SECONDARY Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study |
96.513; 83.912; -0.809 | — |
| SECONDARY Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire |
48.313; 46.415; -1.276; 46.197; 45.929; 0.390 | — |
Eligibility Criteria
Inclusion Criteria
- 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)
Exclusion Criteria
- Participant's last available estimated glomerular filtration rate (eGFR) in the previous study 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
Data sourced from ClinicalTrials.gov (NCT02194985) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.