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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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16 December 2017 |
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Main ID: |
NCT01473875 |
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Date of registration:
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10/11/2011 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
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Scientific title:
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An Open Label Multicenter Extension Study to Evaluate the Long-term Efficacy and Safety of SBC-102 in Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 |
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Date of first enrolment:
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November 2011 |
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Target sample size:
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10 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01473875 |
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Study type:
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Interventional |
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Study design:
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Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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France
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United Kingdom
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Subject's parent or legal guardian provides written consent/permission prior to any
study procedures
- Subject completed treatment in study LAL-CL03 or Subject received treatment with
SBC-102 for at least 4 months under an expanded access treatment regimen
- Subject had no life-threatening or unmanageable study drug toxicity during treatment
with SBC-102 under LAL-CL03 or expanded access treatment regimen.
Exclusion Criteria:
- Clinically important concurrent disease
- Myeloablative preparation, or other systemic pre-transplant conditioning, for
hematopoietic stem cell or liver transplantation
- Previous hematopoietic stem cell transplant.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Lysosomal Acid Lipase Deficiency
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Wolman Disease
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Intervention(s)
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Drug: SBC-102
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Primary Outcome(s)
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Overall survival
[Time Frame: 12 months]
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Secondary Outcome(s)
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Survival rates at periodic intervals and median survival time.
[Time Frame: 3 years]
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Long-term safety of SBC-102 in children with growth failure due to LAL Deficiency
[Time Frame: 3 years]
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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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