# Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

> **NCT03300453** · PHASE1,PHASE2 · COMPLETED · sponsor: **UniQure Biopharma B.V.** · enrollment: 4 (actual)

## Conditions studied

- Sanfilippo Syndrome B

## Interventions

- **DRUG:** rAAV2/5-hNAGLU

## Key facts

- **NCT ID:** NCT03300453
- **Lead sponsor:** UniQure Biopharma B.V.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2013-09-17
- **Primary completion:** 2019-11-27
- **Final completion:** 2019-11-27
- **Target enrollment:** 4 (ACTUAL)
- **Last updated:** 2019-12-02

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03300453

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03300453, "Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome". Retrieved via AI Analytics 2026-05-31 from https://api.ai-analytics.org/clinical/NCT03300453. Licensed CC0.

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