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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: |
NCT01476163 |
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Date of registration:
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17/11/2011 |
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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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Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
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Scientific title:
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Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188) |
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Date of first enrolment:
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December 2011 |
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Target sample size:
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Recruitment status: |
Approved for marketing |
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URL:
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https://clinicaltrials.gov/show/NCT01476163 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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Countries of recruitment
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Australia
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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:
- Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic
kidney (HEK-293) cell-based assay
- 16-74 years of age
- Strong clinical indication for treatment of Fabry disease
- No other treatment option including either unsuitable for ERT or unable to access ERT
- Appropriate female and male contraception
- Willing to receive treatment with migalastat HCl via this program including having
signed an authorization for sharing clinical data
Exclusion Criteria:
- Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute
- Scheduled for renal or other organ transplant or replacement therapy
- Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy
FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)
- Contraindication to migalastat, i.e., sensitivity to other iminosugar such as
miglustat, miglitol
- Treated with another investigational drug within 30 days of start of migalastat HCl
treatment
- Unable to comply with study requirements or deemed otherwise unsuitable for study
entry in the opinion of the investigator.
Age minimum:
16 Years
Age maximum:
74 Years
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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Secondary ID(s)
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AT1001-188
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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