# Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

> **NCT05901987** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **GeneCradle Inc** · enrollment: 33 (estimated)

## Conditions studied

- SMA II

## Interventions

- **GENETIC:** GC101

## Key facts

- **NCT ID:** NCT05901987
- **Lead sponsor:** GeneCradle Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2023-08-01
- **Primary completion:** 2025-04-22
- **Final completion:** 2028-12
- **Target enrollment:** 33 (ESTIMATED)
- **Last updated:** 2025-07-03


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05901987

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05901987, "Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT05901987. Licensed CC0.

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