# Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

> **NCT03381729** · PHASE1 · TERMINATED · sponsor: **Novartis Gene Therapies** · enrollment: 32 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **BIOLOGICAL:** Onasemnogene Abeparvovec-xioi

## Key facts

- **NCT ID:** NCT03381729
- **Lead sponsor:** Novartis Gene Therapies
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2017-12-14
- **Primary completion:** 2021-11-18
- **Final completion:** 2021-11-18
- **Target enrollment:** 32 (ACTUAL)
- **Why stopped:** Based upon overall strategic objectives within the broader intrathecal clinical development program, Novartis Gene Therapies decided to terminate the study early.
- **Last updated:** 2026-01-26


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03381729

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03381729, "Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT03381729. Licensed CC0.

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