# Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

> **NCT02122952** · PHASE1 · COMPLETED · sponsor: **Novartis Gene Therapies** · enrollment: 15 (actual)

## Conditions studied

- Spinal Muscular Atrophy 1

## Interventions

- **BIOLOGICAL:** AVXS-101

## Key facts

- **NCT ID:** NCT02122952
- **Lead sponsor:** Novartis Gene Therapies
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2014-05-05
- **Primary completion:** 2017-12-15
- **Final completion:** 2017-12-15
- **Target enrollment:** 15 (ACTUAL)
- **Last updated:** 2022-09-15


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02122952

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02122952, "Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT02122952. Licensed CC0.

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