Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Study Identifier:
NGLU-CL02
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: SBC-103
Date
Jan 2015 - Oct 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 2 - 12 Years
Requirements Information
Inclusion and Exclusion Criteria
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.
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.
Sex
Female & Male
Age
2 - 12 Years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: SBC-103
Date
Jan 2015 - Oct 2017
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 2 - 12 Years years
Requirements Information
Inclusion and Exclusion Criteria
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.
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.
Protocol 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.
Trial Locations
Location
Status
Location
Minneapolis, Minnesota, United States, 55414
Status
N/A
Location
Pittsburgh, Pennsylvania, United States, 15224
Status
N/A
Location
Barcelona, Spain, 08950
Status
N/A
Location
Birmingham, United Kingdom, B4 6NH
Status
N/A