Trials / Recruiting
RecruitingNCT02716246
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
- Status
- Recruiting
- Phase
- Phase 2 / Phase 3
- Study type
- Interventional
- Enrollment
- 36 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Detailed 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 in August 2022.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | UX111 | 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. |
| DRUG | Prophylactic Immunomodulatory (IM) Therapy | |
| DRUG | Optimized Prophylactic IM Therapy | |
| DRUG | Adjuvant 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. |
Timeline
- Start date
- 2016-04-25
- Primary completion
- 2027-07-01
- Completion
- 2027-07-01
- First posted
- 2016-03-23
- Last updated
- 2026-03-25
Locations
5 sites across 3 countries: United States, Australia, Spain
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02716246. Inclusion in this directory is not an endorsement.