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NCT02716246 · ClinicalTrials.gov registry record · Phase 2
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
A Phase 2 study of Sanfilippo Syndrome and MPS Iiia, sponsored by Ultragenyx Pharmaceutical.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
- 5
- Study locations
NCT02716246 is a Phase 2 study of Sanfilippo Syndrome and MPS Iiia that is actively recruiting participants, run by Ultragenyx Pharmaceutical. The registered enrollment target is 36 participants. The trial reports 5 study locations across 5 states.
The verdict
NCT02716246, a Phase 2 study of Sanfilippo Syndrome and MPS Iiia, is actively recruiting participants, sponsored by Ultragenyx Pharmaceutical.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
- 5
- Study locations
Study Summary
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Conditions Studied
Interventions
- BIOLOGICAL UX111
- DRUG Prophylactic Immunomodulatory (IM) Therapy
- DRUG Optimized Prophylactic IM Therapy
- DRUG Adjuvant IM Therapy
Study Locations (5)
Ohio
- Nationwide Children's Hospital - Columbus
Pennsylvania
- Children's Hospital of Pittsburgh - Pittsburgh
South Australia
- Women's and Children's Hospital - North Adelaide
Barcelona
- Vall d'Hebron Barcelona Hospital Campus - Barcelona
Other
- Hospital Clínico Universitario de Santiago - Santiago de Compostela
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2016-04-25 |
| Est. Completion | 2027-07 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02716246
The ClinicalTrials.gov registry entry for NCT02716246 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 4 conditions, with Sanfilippo Syndrome appearing as the primary indexed condition, and to 4 interventions - of which UX111 is the first listed.
NCT02716246 reports 5 study locations spanning 5 distinct geographic areas - top geographies include Ohio, Pennsylvania, South Australia.
Frequently Asked Questions
What is clinical trial NCT02716246 about?
NCT02716246 is a clinical study titled "Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH". The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
What is the current status of trial NCT02716246?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2016-04-25. Estimated completion is 2027-07.
What conditions does trial NCT02716246 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome, MPS Iiia, Sanfilippo A, Mucopolysaccharidosis III.
What interventions are being tested in trial NCT02716246?
The interventions under investigation include: UX111 (BIOLOGICAL), Prophylactic Immunomodulatory (IM) Therapy (DRUG), Optimized Prophylactic IM Therapy (DRUG), Adjuvant IM Therapy (DRUG).
Who is sponsoring clinical trial NCT02716246?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02716246 being conducted?
This trial has 5 study locations across Ohio, Pennsylvania, South Australia, Barcelona. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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