# Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

> **NCT01009294** · PHASE2 · TERMINATED · sponsor: **PTC Therapeutics** · enrollment: 6 (actual)

## Conditions studied

- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy

## Interventions

- **DRUG:** Ataluren
- **DRUG:** Chronic Corticosteroid Therapy

## Key facts

- **NCT ID:** NCT01009294
- **Lead sponsor:** PTC Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2010-01-13
- **Primary completion:** 2010-03-23
- **Final completion:** 2010-03-23
- **Target enrollment:** 6 (ACTUAL)
- **Why stopped:** Terminated early because a similar study with Ataluren exhibited lack of efficacy at the high dose (not due to safety concerns).
- **Last updated:** 2020-07-29

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01009294

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01009294, "Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)". Retrieved via AI Analytics 2026-05-30 from https://api.ai-analytics.org/clinical/NCT01009294. Licensed CC0.

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