# Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

> **NCT05824169** · PHASE1,PHASE2 · RECRUITING · sponsor: **GeneCradle Inc** · enrollment: 18 (estimated)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **GENETIC:** GC101

## Key facts

- **NCT ID:** NCT05824169
- **Lead sponsor:** GeneCradle Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2023-02-25
- **Primary completion:** 2026-12
- **Final completion:** 2026-12
- **Target enrollment:** 18 (ESTIMATED)
- **Last updated:** 2025-07-03


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05824169

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05824169, "Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT05824169. Licensed CC0.

---

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