# Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

> **NCT03461289** · PHASE3 · COMPLETED · sponsor: **Novartis Gene Therapies** · enrollment: 33 (actual)

## Conditions studied

- SMA

## Interventions

- **BIOLOGICAL:** Onasemnogene Abeparvovec-xioi

## Key facts

- **NCT ID:** NCT03461289
- **Lead sponsor:** Novartis Gene Therapies
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2018-08-16
- **Primary completion:** 2020-09-11
- **Final completion:** 2020-09-11
- **Target enrollment:** 33 (ACTUAL)
- **Last updated:** 2026-01-26


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03461289

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03461289, "Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1". Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/clinical/NCT03461289. Licensed CC0.

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