# Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

> **NCT04088734** · PHASE1,PHASE2 · TERMINATED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 5 (actual)

## Conditions studied

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

## Interventions

- **DRUG:** ABO-102

## Key facts

- **NCT ID:** NCT04088734
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2019-09-18
- **Primary completion:** 2022-03-10
- **Final completion:** 2022-03-10
- **Target enrollment:** 5 (ACTUAL)
- **Why stopped:** Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
- **Last updated:** 2023-07-25

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04088734

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04088734, "Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT04088734. Licensed CC0.

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