# Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

> **NCT06207552** · EARLY_PHASE1 · RECRUITING · sponsor: **Children's Hospital of Fudan University** · enrollment: 6 (estimated)

## Conditions studied

- Fabry Disease

## Interventions

- **GENETIC:** BBM-F101 injection

## Key facts

- **NCT ID:** NCT06207552
- **Lead sponsor:** Children's Hospital of Fudan University
- **Sponsor class:** OTHER
- **Phase:** EARLY_PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2024-02-20
- **Primary completion:** 2025-06
- **Final completion:** 2029-06
- **Target enrollment:** 6 (ESTIMATED)
- **Last updated:** 2024-07-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06207552

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06207552, "Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT06207552. Licensed CC0.

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