- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02324049
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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Barcelona, Spain, 08950
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Birmingham, United Kingdom, B4 6NH
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Minnesota
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Minneapolis, Minnesota, United States, 55414
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: SBC-103
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first. Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C. Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg. |
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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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TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A).
A severe AE was defined as an AE that was incapacitating and required medical intervention.
TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented.
A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
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Baseline to Week 142
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- NGLU-CL02
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Alexion PharmaceuticalsTerminatedMucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo BUnited Kingdom
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Alexion PharmaceuticalsCompletedMPS IIIB (Sanfilippo Syndrome)Spain, Netherlands, United Kingdom, United States, Brazil
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Nationwide Children's HospitalSanfilippo Children's Research Foundation; The Sanfilippo Research Foundation; The Children's Medical Research FoundationCompletedMucopolysaccharidosis Type IIIA | Mucopolysaccharidosis Type IIIBUnited States
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Alexion PharmaceuticalsTerminatedMPS IIIB (Sanfilippo B Syndrome)Spain, United States, Brazil, United Kingdom, Portugal, Italy
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Allievex CorporationCompletedMucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 BUnited States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIBUnited States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
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Allievex CorporationCompletedMucopolysaccharidosis Type IIIB | MPS III BUnited States, Taiwan, Spain, Colombia, Germany, United Kingdom, Turkey
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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIB | MPS III BGermany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
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Alexion PharmaceuticalsCompletedMPS IIIB (Sanfilippo B Syndrome)United Kingdom
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