Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

NCT ID: NCT00526071

Last Updated: 2018-10-03

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-09-17
• Study Completion Date: 2012-09-08

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.

Conditions

Fabry Disease

Study Design

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

Study Groups

Migalastat

Migalastat was administered orally, 150 mg QOD, 250 mg QD for 3 days, 4 days off per week for 2 months, or 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. Participants received migalastat for up to 56 months.

Group Type: EXPERIMENTAL

migalastat HCl

Intervention Type: DRUG

Interventions

migalastat HCl

Intervention Type: DRUG

Other Intervention Names

AT1001; Galafold; migalastat

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

• Minimum Eligible Age: 18 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

Decatur, Georgia, United States

New York, New York, United States

Dallas, Texas, United States

Parkville, , Australia

Porto Alegre, , Brazil

Garches, , France

London, , United Kingdom

Salford, , United Kingdom

Countries

United States; Australia; Brazil; France; United Kingdom

Other Identifiers

FAB-CL-205

Identifier Type: -

Identifier Source: org_study_id