# Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

> **NCT04851873** · PHASE3 · COMPLETED · sponsor: **Novartis Pharmaceuticals** · enrollment: 24 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **GENETIC:** OAV101

## Key facts

- **NCT ID:** NCT04851873
- **Lead sponsor:** Novartis Pharmaceuticals
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2021-09-08
- **Primary completion:** 2023-06-13
- **Final completion:** 2023-06-13
- **Target enrollment:** 24 (ACTUAL)
- **Last updated:** 2024-10-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04851873

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04851873, "Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)". Retrieved via AI Analytics 2026-09-17 from https://api.ai-analytics.org/clinical/NCT04851873. Licensed CC0.

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