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.

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

Fabry Disease

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.

migalastat hydrochloride

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

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.

The annualized rate of change of the eGFR was assessed per participant by the slope of
the simple linear regression between the observed values and the assessment times. It was
calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
equation (eGFR [CKD-EPI]) and the Modification of Diet in Renal Disease (MDRD) equation
(eGFR [MDRD]). The equations are as follows:

eGFR [MDRD] = 175 * (Serum Creatinine)^-1.154 * (Age)^-0.203 * 1.212 (if black or African
American) * 0.742 (if female); eGFR [CKD-EPI] = 141 * min(serum creatinine/kappa,1)^alpha
* max(serum creatinine/kappa, 1)^-1.209 * 0.993^age * 1.1018(if female) * 1.159(if black
or African American), where kappa is 0.7 for females and 0.9 for males, alpha is -0.329
for females and -0.411 for males, min is minimum of serum creatinine/kappa or 1, and max
is the maximum of serum creatinine/kappa or 1. The number of participants with at least a
Baseline and a post-Baseline value are presented.

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

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

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status