# Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

> **NCT03505099** · PHASE3 · COMPLETED · sponsor: **Novartis Gene Therapies** · enrollment: 30 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **BIOLOGICAL:** onasemnogene abeparvovec-xioi

## Key facts

- **NCT ID:** NCT03505099
- **Lead sponsor:** Novartis Gene Therapies
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2018-04-02
- **Primary completion:** 2021-06-15
- **Final completion:** 2021-06-15
- **Target enrollment:** 30 (ACTUAL)
- **Last updated:** 2026-01-26

## Collaborators

- PRA Health Sciences

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03505099

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03505099, "Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2". Retrieved via AI Analytics 2026-05-18 from https://api.ai-analytics.org/clinical/NCT03505099. Licensed CC0.

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