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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00304512 |
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Date of registration:
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17/03/2006 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
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Scientific title:
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A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease |
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Date of first enrolment:
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September 7, 2006 |
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Target sample size:
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9 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00304512 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Australia
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Brazil
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Canada
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France
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United Kingdom
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United States
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Contacts
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Name:
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Medical Monitor, Clinical Research |
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Address:
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Telephone:
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Email:
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Affiliation:
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Amicus Therapeutics |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- 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 Criteria:
- 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.
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Drug: migalastat HCl
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Primary Outcome(s)
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Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
[Time Frame: Day 1 (after dosing) through Week 48]
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Secondary Outcome(s)
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a-Gal A Activity In Leukocytes At Baseline, Week 12, And Week 48
[Time Frame: Baseline, Week 12 (end of treatment period), Week 48 (end of extension period)]
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PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat
[Time Frame: 0 (predose), 0.5, 1, 2, 3, 4, 5, 6, 8, and 10 hours (postdose)]
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Secondary ID(s)
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FAB-CL-204
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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