Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Recruiting Phase 2 study of Sanfilippo Syndrome and MPS Iiia, sponsored by Ultragenyx Pharmaceutical.
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, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Exposure is defined as the time-normalized area under the curve (AUC) of the percentage reduction from baseline.
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 |
What NCT02716246 shows while recruiting
NCT02716246 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
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 lists 5 locations in 5 states (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.
Learn More About Clinical Trials
Similar trials for Sanfilippo Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT02716246's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00929006 · 36 participants · Early Phase 1
Acute Progesterone Suppression of Wake vs. Sleep Luteinizing Hormone Pulse Frequency in Pubertal Girls With and Without Hyperandrogenism
- NCT01891318 · 36 participants · Phase 1
Neoadjuvant Radiosurgery for Resectable Brain Metastases: Phase I/II Study
- NCT02291848 · 36 participants · Phase 1
Tumor-Associated Antigen-Specific Cytotoxic T-Lymphocytes for Multiple Myeloma
- NCT02366819 · 36 participants · Phase 4
Genetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI