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

NCT00526071 | Terminated Phase 2 Results

• 1 other identifier: FAB-CL-205
• Study Type: interventional
• Target: 23
• Locations: 5 countries
• Sites: 8

Brief Summary

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

Conditions

Fabry Disease

Keywords

Amicus Therapeutics; AT1001; Galafold; Migalastat; Substrate

Outcome Measures

Primary Outcomes (1)

• Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)

  A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through End of Study (EOS) or follow-up (for participants who did not enroll in Study AT1001-041) are presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.

  Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)

Secondary Outcomes (2)

• Absolute Change From Baseline In α-Galactosidase A (α-Gal A) Activity In Leukocytes To Month 42

  Baseline, Month 42

• Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration

  0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])

Study Arms (1)

Migalastat

EXPERIMENTAL

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.

Drug: migalastat HCl

Interventions

migalastat HCl DRUG

Also known as: AT1001, Galafold, migalastat

Migalastat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (8)

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Unknown Facility

New York, New York, 10016, United States

Location

Unknown Facility

Dallas, Texas, 78226, United States

Location

Unknown Facility

Parkville, Australia

Location

Unknown Facility

Porto Alegre, Brazil

Location

Unknown Facility

Garches, France

Location

Unknown Facility

London, United Kingdom

Location

Unknown Facility

Salford, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat; larazotide acetate

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Amicus Therapeutics
• Organization: Medical Affairs

MedInfoUSA@amicusrx.com

+1-877-426-4287 (877-4-AMICUS)

Study Officials

• Medical Monitor, Clinical Research

  Amicus Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 5, 2007
• First Posted: September 6, 2007
• Study Start: September 17, 2007
• Primary Completion: September 8, 2012
• Study Completion: September 8, 2012
• Last Updated: October 3, 2018
• Results First Posted: October 3, 2018

Record last verified: 2018-10

Locations

US (3); GB (2); BR (1); AU (1); FR (1)