A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease
1 other identifier
interventional
4
2 countries
2
Brief Summary
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2006
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 27, 2006
CompletedFirst Posted
Study publicly available on registry
January 31, 2006
CompletedStudy Start
First participant enrolled
June 27, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 8, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
May 8, 2008
CompletedResults Posted
Study results publicly available
September 7, 2018
CompletedOctober 31, 2018
October 1, 2018
1.9 years
January 27, 2006
August 10, 2018
October 1, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
TEAEs were defined as any adverse event with a 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 adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Day 1 (after dosing) through Week 48 (end of extension period)
Secondary Outcomes (1)
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 12, And Week 48
Baseline, Week 12 (end of treatment period), Week 48 (end of extension period)
Study Arms (1)
Migalastat
EXPERIMENTALMigalastat 150 mg was administered orally QOD during the 12-week treatment period and then during the optional 36-week extension period.
Interventions
Eligibility Criteria
You may qualify if:
- Males between 18 and 65 years of age (inclusive)
- Hemizygous for Fabry disease
- Had a confirmed diagnosis of Fabry disease with a documented missense gene mutation (individual or familial)
- Had enhanceable enzyme activity based on in vitro tests
- Had documented evidence of cardiac and/or renal dysfunction (for example, abnormal electrocardiogram (ECG), left ventricular hypertrophy, renal insufficiency) and/or cerebral tissue dysfunction documented by evidence of stroke and/or peripheral nervous tissue dysfunction (for example, intolerance to heat/cold, decrease of perspiration)
- Must have been previously untreated by enzyme replacement therapy (ERT) or substrate depletion for Fabry disease or were able to stop ERT for at least 18 weeks or up to three months, and be willing to undergo two kidney and three skin biopsies
- Agreed to be sexually abstinent or use a condom with spermicide when engaging in sexual activity during the course of the study and for a period of 30 days following their completion of the study
- Were willing and able to sign an informed consent form
You may not qualify if:
- History of significant disease other than Fabry disease
- History of organ transplant
- Serum creatinine \>2 mg per deciliter on Day -2
- Screening 12-lead ECG demonstrating corrected QT interval \>450 milliseconds prior to dosing
- Pacemaker or other contraindication for magnetic resonance imaging (MRI) scanning
- Took any of the following prohibited medications: Fabrazyme® (agalsidase beta), Replagal™ (agalsidase alfa), Glyset® (miglitol), Zavesca® (miglustat), or any experimental therapy for any indication
- Participated in a previous clinical trial in the last 30 days
- Any other condition, which, in the opinion of the investigator, would jeopardize the safety of the participant or impact the validity of the study results
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Unknown Facility
Parkville, Australia
Unknown Facility
Porto Alegre, Brazil
Related Publications (2)
Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.
PMID: 27657681DERIVEDGermain DP, Giugliani R, Hughes DA, Mehta A, Nicholls K, Barisoni L, Jennette CJ, Bragat A, Castelli J, Sitaraman S, Lockhart DJ, Boudes PF. Safety and pharmacodynamic effects of a pharmacological chaperone on alpha-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis. 2012 Nov 24;7:91. doi: 10.1186/1750-1172-7-91.
PMID: 23176611DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Amicus Therapeutics
- Organization
- Medical Affairs
Study Officials
- STUDY DIRECTOR
Medical Monitor, Clinical Research
Amicus Therapeutics
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
January 27, 2006
First Posted
January 31, 2006
Study Start
June 27, 2006
Primary Completion
May 8, 2008
Study Completion
May 8, 2008
Last Updated
October 31, 2018
Results First Posted
September 7, 2018
Record last verified: 2018-10