# Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

> **NCT04360265** · PHASE3 · ENROLLING_BY_INVITATION · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 41 (estimated)

## Conditions studied

- Mucopolysaccharidosis IIIA
- MPS IIIA
- Sanfilippo Syndrome
- Sanfilippo A

## Interventions

- **OTHER:** No Investigational Product
- **DRUG:** Adjuvant Immunomodulatory (IM) Therapy

## Key facts

- **NCT ID:** NCT04360265
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2020-09-28
- **Primary completion:** 2027-08
- **Final completion:** 2027-08
- **Target enrollment:** 41 (ESTIMATED)
- **Last updated:** 2026-04-24


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04360265

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04360265, "Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT04360265. Licensed CC0.

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