Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
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ClinicalTrials.gov Identifier: NCT02716246 |
Recruitment Status :
Active, not recruiting
First Posted : March 23, 2016
Last Update Posted : November 10, 2022
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Sponsor:
Ultragenyx Pharmaceutical Inc
Collaborator:
Abeona Therapeutics, Inc
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc
Tracking Information | |||||
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First Submitted Date ICMJE | March 17, 2016 | ||||
First Posted Date ICMJE | March 23, 2016 | ||||
Last Update Posted Date | November 10, 2022 | ||||
Actual Study Start Date ICMJE | March 2016 | ||||
Estimated Primary Completion Date | December 2024 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
Development of unacceptable toxicity: [ Time Frame: 24 months ] Determination of safety based on the development of unacceptable toxicity: defined as the occurrence of two or more unanticipated Grade III or higher treatment-related toxicity.
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | ||||
Official Title ICMJE | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA | ||||
Brief Summary | Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein | ||||
Detailed Description | Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE | Biological: ABO-102
Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein.
Other Names:
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
28 | ||||
Original Estimated Enrollment ICMJE |
9 | ||||
Estimated Study Completion Date ICMJE | December 2024 | ||||
Estimated Primary Completion Date | December 2024 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Australia, Spain, United States | ||||
Removed Location Countries | France, Germany | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT02716246 | ||||
Other Study ID Numbers ICMJE | ABT001 UX111-CL301 ( Other Identifier: Ultragenyx Pharmaceutical Inc ) 2015-003904-21 ( EudraCT Number ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Ultragenyx Pharmaceutical Inc | ||||
Original Responsible Party | Kevin Flanigan, Nationwide Children's Hospital, Professor of Pediatrics | ||||
Current Study Sponsor ICMJE | Ultragenyx Pharmaceutical Inc | ||||
Original Study Sponsor ICMJE | Kevin Flanigan | ||||
Collaborators ICMJE | Abeona Therapeutics, Inc | ||||
Investigators ICMJE |
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PRS Account | Ultragenyx Pharmaceutical Inc | ||||
Verification Date | November 2022 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |