Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
← catalyst calendarNCT04360265 · readout ≤ 376 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
Phase 3
Status
Enrolling by invitation
Study type
Interventional
Design
Non randomized · None · Other
Enrollment
41estimated
Sites
4
Countries
Australia, Spain, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2020-09-28 | actual | When the study began enrolling |
| Primary completion | Aug 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Aug 2027 | estimated | The whole study's end, after follow-up |
| First posted | 2020-04-24 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-17 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Mucopolysaccharidosis IIIA
- MPS IIIA
- Sanfilippo Syndrome
- Sanfilippo A
Interventions
- Other: No Investigational Product
- Drug: Adjuvant Immunomodulatory (IM) Therapy
Primary outcomes
what the primary-completion date above is the date OFNumber of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
measured Up to Year 5
Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time
measured Up to Year 5
Permalink · RARE's whole pipeline · Catalyst calendar · Every registered study · What changed