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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: |
NCT00526071 |
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Date of registration:
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05/09/2007 |
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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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Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
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Scientific title:
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Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease |
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Date of first enrolment:
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September 17, 2007 |
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Target sample size:
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23 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT00526071 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group 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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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:
- 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
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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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 EOS (up to 56 months) or follow-up (28 days after EOS)]
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Secondary Outcome(s)
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Absolute Change From Baseline In a-Galactosidase A (a-Gal A) Activity In Leukocytes To Month 42
[Time Frame: Baseline, Month 42]
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Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration
[Time Frame: 0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])]
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Secondary ID(s)
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FAB-CL-205
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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