Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Public ClinicalTrials.gov record NCT02716246. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Study identification
- NCT ID
- NCT02716246
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Enrollment
- 36 participants
Conditions and interventions
Interventions
- Adjuvant IM Therapy Drug
- Optimized Prophylactic IM Therapy Drug
- Prophylactic Immunomodulatory (IM) Therapy Drug
- UX111 Biological
Drug · Biological
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 24, 2016
- Primary completion
- Jun 30, 2027
- Completion
- Jun 30, 2027
- Last update posted
- Apr 23, 2026
2016 – 2027
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Completed |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Completed |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02716246, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 23, 2026 · Synced May 18, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02716246 live on ClinicalTrials.gov.