Trial · NCT02716246
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT02716246, Phase 2, Phase 3, Recruiting, Ultragenyx Pharmaceutical Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH. Trials, record NCT02716246. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02716246
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
- NCT number
- NCT02716246
- Status
- Recruiting
- Phases
- Phase 2, Phase 3
- Enrollment
- 36 participants, estimated
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Interventions
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
- First posted
- 2016-03-23
- Last updated
- 2026-09-22
- Other registries' IDs
- eudract:2015-003904-21
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cerebrospinal Fluid (CSF) Heparan Sulfate (HS) (Disaccharide) Exposure | Up to Month 24 Visit | Exposure is defined as the time-normalized area under the curve (AUC) of the percentage reduction from baseline. |
| Secondary | Bayley Scales of Infant and Toddler Development-Third Edition (BSITD-III) Cognitive Raw Score Over Time | Up to Month 24 Visit | |
| Secondary | CSF Ganglioside Type 2 (GM2) Exposure | Up to Month 24 Visit | Exposure is defined as the time-normalized area under the curve (AUC) of the percentage change from baseline. |
| Secondary | CSF Ganglioside Type 3 (GM3) Exposure | Up to Month 24 Visit | Exposure is defined as the time-normalized area under the curve (AUC) of the percentage change from baseline. |
| Secondary | Percent Change from Baseline in CSF HS | Baseline, Up to Month 24 Visit | |
| Secondary | BSITD-III Receptive Communication Raw Score Over Time | Up to Month 24 Visit | |
| Secondary | BSITD-III Expressive Communication Raw Score Over Time | Up to Month 24 Visit | |
| Secondary | Percent Change From Baseline in Total Cortical Volume | Baseline, Up to Month 24 Visit | |
| Other | Number of Participants with Treatment-Emergent Adverse Events, and Treatment-Emergent Serious Adverse Events | Up to Month 24 Visit |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02716246
GET https://conductscience.org/api/trials/v1/NCT02716246/registered