# Long-Term Follow-up Study for Patients From AVXS-101-CL-101

> **NCT03421977** · — · ACTIVE_NOT_RECRUITING · sponsor: **Novartis Gene Therapies** · enrollment: 13 (actual)

## Conditions studied

- Spinal Muscular Atrophy 1

## Interventions

- **BIOLOGICAL:** Onasemnogene Abeparvovec-xioi

## Key facts

- **NCT ID:** NCT03421977
- **Lead sponsor:** Novartis Gene Therapies
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2017-09-21
- **Primary completion:** 2030-12-02
- **Final completion:** 2030-12-02
- **Target enrollment:** 13 (ACTUAL)
- **Last updated:** 2025-04-25


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03421977

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03421977, "Long-Term Follow-up Study for Patients From AVXS-101-CL-101". Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/clinical/NCT03421977. Licensed CC0.

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