Status:
COMPLETED
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
FEMALE
18-65 years
Phase:
PHASE2
Brief Summary
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
Detailed Description
This was a Phase 2, open-label study in female participants with Fabry disease. The study consisted of a 4-week screening period, during which participants' galactosidase (GLA) genotype was assessed f...
Eligibility Criteria
Inclusion
- Females between 18 and 65 years of age (inclusive)
- Heterozygous 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
- Were naïve to enzyme replacement therapy (ERT) and other therapies, except for palliative therapies for the signs and symptoms of Fabry disease, or stopped ERT for at least 18 weeks
- Had end organ dysfunction, even minimal, demonstrated by abnormal electrocardiogram (ECG) or evidence of left ventricular hypertrophy documented by echocardiogram or by cardiac biopsy; or renal insufficiency documented by common clinical assessments such as creatinine and glomerular filtration rate or by renal biopsy; or brain tissue dysfunction as documented by evidence of stroke (clinically or imaging); or peripheral nervous tissue dysfunction documented by complaints of intolerance to heat or cold, decreased vibratory sense and proprioception, decreased ability to perspire, or acroparesthesia.
- Were willing to undergo 2 renal and 3 skin biopsies
- Agreed to be sexually abstinent or practice an effective method of contraception when engaging in sexual activity during the course of the study and for a period of 30 days following their completion of the study for women of childbearing potential.
- Were willing and able to provide written informed consent
Exclusion
- Pregnant or lactating
- History of organ transplant
- History of significant disease other than Fabry disease (for example, end-stage renal disease; Class III or IV heart disease \[per the New York Heart Association classification\]; current diagnosis of cancer, except for basal cell carcinoma of the skin; diabetes \[unless hemoglobin A1c ≤8\]; or neurological disease that would have impaired the participant's ability to participate in the study)
- Serum creatinine \>176 micromoles/liter on Day -2
- Screening 12-lead ECG demonstrating corrected QT interval \>450 milliseconds
- Pacemaker or other contraindication for magnetic resonance imaging scanning
- Taking a medication prohibited by the protocol: 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.
Key Trial Info
Start Date :
September 7 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 9 2008
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00304512
Start Date
September 7 2006
End Date
May 9 2008
Last Update
October 3 2018
Active Locations (6)
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1
Decatur, Georgia, United States, 30033
2
Parkville, Victoria, Australia, 3050
3
Porto Alegre, Brazil, RS, 90035-903
4
Montreal, Canada, H4J 1C5