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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: |
NCT02324049 |
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Date of registration:
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15/12/2014 |
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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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Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
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Scientific title:
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A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously |
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Date of first enrolment:
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January 22, 2015 |
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Target sample size:
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11 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02324049 |
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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 1/Phase 2
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Countries of recruitment
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Spain
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United Kingdom
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United States
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Key inclusion & exclusion criteria
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Key Inclusion Criteria:
- A participant was greater than or equal to 2 years of age but less than 12 years of
age at the time of informed consent.
- Definitive diagnosis of MPS IIIB.
- Documented developmental delay.
Key Exclusion Criteria:
- Received treatment with gene therapy at any time.
- Previous hematopoietic stem cell or bone marrow transplant.
- Had any internal or non-removable external metal items that presented a safety risk
for study assessments that utilized magnetic fields, or any other medical condition or
circumstance in which magnetic resonance imaging was contraindicated according to
local institutional policy.
- Known hypersensitivity to eggs.
Age minimum:
2 Years
Age maximum:
12 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis IIIB
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Intervention(s)
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Drug: SBC-103
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Primary Outcome(s)
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Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
[Time Frame: Baseline to Week 142]
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Secondary ID(s)
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NGLU-CL02
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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