# Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

> **NCT01474343** · PHASE1,PHASE2 · COMPLETED · sponsor: **LYSOGENE** · enrollment: 4 (actual)

## Conditions studied

- Mucopolysaccharidosis Type III A
- Sanfilippo Disease Type A

## Interventions

- **GENETIC:** SAF-301

## Key facts

- **NCT ID:** NCT01474343
- **Lead sponsor:** LYSOGENE
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2011-08
- **Primary completion:** 2013-05
- **Final completion:** 2013-05
- **Target enrollment:** 4 (ACTUAL)
- **Last updated:** 2014-05-06


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01474343

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01474343, "Intracerebral Gene Therapy for Sanfilippo Type A Syndrome". Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/clinical/NCT01474343. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*