Sanfilippo Syndrome Clinical Trial
Official title:
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
The main objective of this study is to evaluate the efficacy and safety of ABO-102 for the treatment of MPS IIIA.
Open-label, single dose, dose-escalation clinical trial of ABO-102 (scAAV9.U1a.hSGSH) injected intravenously through a peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least three months. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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HSCT for High Risk Inherited Inborn Errors
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Phase 2 | |
| Terminated |
NCT04088734 -
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
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Phase 1/Phase 2 | |
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Study of Cannabidiol in Sanfilippo Syndrome
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Phase 2/Phase 3 | |
| Recruiting |
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The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
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||
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Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
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Phase 1/Phase 2 | |
| Completed |
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Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
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Phase 2 | |
| Terminated |
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An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
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Phase 2 |