Clinical trial NCT00526071: Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Official title: Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Fabry Disease
Keywords: Amicus Therapeutics
AT1001
Galafold
Migalastat
Substrate
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2007-09-06
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease

Detailed description: This was a long-term open-label study of migalastat in participants with Fabry disease who were previously enrolled in a Phase 2 study of migalastat (FAB-CL-201 \[NCT00214500\], FAB-CL-202 \[NCT00283959\], FAB-CL-203 \[NCT00283933\], or FAB-CL-204 \[NCT00304512\]). Participants could enter this extension study immediately upon completion of participation in their previous study of migalastat, or at a later time point. Thus, some participants did not necessarily have continuous treatment with migalastat from the end of the original study to the time of enrollment into this extension study. Participants who enrolled before Protocol Amendment 2 received migalastat 150 milligrams (mg) orally once every other day (QOD). After the amendment, these participants entered a dose escalation period (DEP) at their next scheduled visit. During the DEP, participants received migalastat 250 mg (once daily \[QD\] for 3 days and 4 days off per week) for the first 2 months. If there were no safety concerns, the dose was then increased to 500 mg QD for 3 days and 4 days off per week). Participants received 500 mg (QD for 3 days and 4 days off per week) for up to 10 months, depending on the approval date of the protocol amendments at each site. An interim review of safety and PD data was performed after all enrolled participants had completed at least 4 months of treatment in the DEP. After the review, the dose and regimen of migalastat was returned to 150 mg QOD for all participants, except those who were on another dose as previously agreed by the investigator and medical monitor.

The sponsor (Amicus Therapeutics) discontinued Study FAB-CL-205 for logistical reasons and not due to either safety concerns or lack of efficacy. Participants who were ongoing in Study FAB-CL-205 at the time of discontinuation were offered participation in another open-label, long-term treatment study of migalastat (AT1001-041 \[NCT01458119\]). Participants who did not enroll in Study AT1001-041 were contacted by telephone or another suitable method approximately 1 month after the End of Study (EOS) visit to inquire about adverse events and concomitant medications.

Eligibility criteria: Inclusion Criteria:

* Must have completed another Phase 2 study of migalastat in Fabry Disease
* Women of childbearing potential must have had a negative result on their pregnancy test
* Male and female participants agreed to use a reliable method of contraception during study treatment and for 4 weeks after study treatment termination
* Were willing and able to provide written informed consent

Exclusion Criteria:

* Had not completed a Phase 2 study of migalastat in Fabry Disease
* Had a major protocol violation in the preceding migalastat trial and was discontinued
* Had undergone or was scheduled to undergo kidney transplantation or was currently on dialysis
* Had been treated with another investigational drug (except migalastat) within 30 days of study start
* Had been treated with Fabrazyme® (agalsidase beta), Replagal™ (agalsidase alfa), Glyset® (miglitol), or Zavesca® (miglustat) within 2 weeks prior to enrollment

Primary outcomes:
measure: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
timeFrame: Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)
Secondary outcomes:
measure: Absolute Change From Baseline In α-Galactosidase A (α-Gal A) Activity In Leukocytes To Month 42
timeFrame: Baseline, Month 42
measure: Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration
timeFrame: 0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])
Other outcomes:
Locations:
city: Decatur
country: United States
state: Georgia
city: New York
country: United States
state: New York
city: Dallas
country: United States
state: Texas
city: Parkville
country: Australia
city: Porto Alegre
country: Brazil
city: Garches
country: France
city: London
country: United Kingdom
city: Salford
country: United Kingdom
Study officials:
affiliation: Amicus Therapeutics
name: Medical Monitor, Clinical Research
role: STUDY_DIRECTOR
References: