Back to resultsSafety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Primary Purpose
Mucopolysaccharidosis IIIB
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SBC-103
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis IIIB focused on measuring MPS IIIB, Mucopolysaccharidosis, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome, Metabolism, Inborn Errors, Metabolic Diseases, Genetic Diseases, Inborn, Carbohydrate Metabolism, Inborn Errors, Connective Tissue Diseases, Lysosomal Storage Diseases, Mucinoses, Mucopolysaccharidoses, Pathologic Processes
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
SBC-103
Arm Description
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.
Outcomes
Primary Outcome Measures
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02324049
Brief Title
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Official Title
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 Type
Interventional
2. Study Status
Record Verification Date
July 2018
Overall Recruitment Status
Completed
Study Start Date
January 22, 2015 (Actual)
Primary Completion Date
October 16, 2017 (Actual)
Study Completion Date
October 16, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.
Detailed Description
This study was designed as a 3-part study to evaluate the safety and tolerability of IV administration of SBC-103. Participants enrolled in Part A (0.3, 1.0, or 3.0 milligrams [mg] per kilogram [kg] of SBC-103 administered every other week [QOW] for 24 consecutive weeks). Participants who completed Part A were eligible for Part B (an increase to 1.0 or 3.0 mg/kg QOW). Participants who completed Part B were eligible for Part C (5.0 and/or 10.0 mg/kg to continue through Week156; no participants received both 5.0 and 10.0 mg/kg). Due to the early termination of the SBC-103 development program, including this study, all participants withdrew from Part C at the sponsor's decision. As a result of the early termination of this program, this report provides only safety data.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis IIIB
Keywords
MPS IIIB, Mucopolysaccharidosis, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome, Metabolism, Inborn Errors, Metabolic Diseases, Genetic Diseases, Inborn, Carbohydrate Metabolism, Inborn Errors, Connective Tissue Diseases, Lysosomal Storage Diseases, Mucinoses, Mucopolysaccharidoses, Pathologic Processes
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
SBC-103
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
SBC-103
Other Intervention Name(s)
recombinant human alpha-N-acetylglucosaminidase
Primary Outcome Measure Information:
Title
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
Description
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.
Time Frame
Baseline to Week 142
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55414
Country
United States
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
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