Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Ultragenyx Pharmaceutical Inc
Summary
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Description
Open-label, single dose, dose-escalation clinical trial of UX111 (scAAV9.U1a.hSGSH) injected intravenously through a peripheral limb vein. A limited course of prophylactic immunomodulatory (IM) therapy will be administered. At approved sites adjuvant IM 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 IM therapy. Not all participants may receive adjuvant IM therapy. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Diagnosis of MPS IIIA confirmed by the following methods: * No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and * Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene (based upon review of documented results from a qualified laboratory, and with confirmation with Medical Monitor) * Age: * For Cohort 1-3: From birth (participating sites in USA and Australia) OR 6 months (participating sites in Spain) to 2 years of age with no BSITD-III Cognitive Development Quotient (DQ) requirement, or o…
Interventions
- BiologicalUX111
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.
- DrugProphylactic Immunomodulatory (IM) Therapy
- DrugOptimized Prophylactic IM Therapy
- DrugAdjuvant IM Therapy
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy.
Locations (5)
- Nationwide Children's HospitalColumbus, Ohio
- Children's Hospital of PittsburghPittsburgh, Pennsylvania
- Women's and Children's HospitalNorth Adelaide, South Australia
- Vall d'Hebron Barcelona Hospital CampusBarcelona
- Hospital Clínico Universitario de SantiagoSantiago de Compostela