# An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

> **NCT02268552** · PHASE1,PHASE2 · COMPLETED · sponsor: **Novartis Pharmaceuticals** · enrollment: 40 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **DRUG:** branaplam

## Key facts

- **NCT ID:** NCT02268552
- **Lead sponsor:** Novartis Pharmaceuticals
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2015-04-02
- **Primary completion:** 2022-12-29
- **Final completion:** 2022-12-29
- **Target enrollment:** 40 (ACTUAL)
- **Last updated:** 2025-04-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02268552

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02268552, "An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT02268552. Licensed CC0.

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