Trial · NCT04360265
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
NCT04360265, Phase 3, Enrolling by invitation, Ultragenyx Pharmaceutical Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA. Trials, record NCT04360265. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04360265
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
- NCT number
- NCT04360265
- Status
- Enrolling by invitation
- Phases
- Phase 3
- Enrollment
- 41 participants, estimated
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
- Interventions
- No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
- First posted
- 2020-04-24
- Last updated
- 2026-07-17
- Other registries' IDs
- eudract:2019-002979-34
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) | Up to Year 5 | |
| Primary | Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time | Up to Year 5 | |
| Secondary | Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure | Baseline, Up to Month 36 | |
| Secondary | Percent Change From Baseline in Prior Trial in CSF HS | Baseline, Up to Month 36 | |
| Secondary | BSITD-III Receptive Communication Raw Score Over Time | Up to Year 5 | |
| Secondary | BSITD-III Expressive Communication Raw Score Over Time | Up to Year 5 | |
| Secondary | Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume | Baseline, Up to Month 36 | |
| Secondary | Survival | Up to Year 5 |
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.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT04360265
GET https://conductscience.org/api/trials/v1/NCT04360265/registered