# Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)

> **NCT02505087** · PHASE2,PHASE3 · TERMINATED · sponsor: **Assistance Publique - Hôpitaux de Paris** · enrollment: 7 (actual)

## Conditions studied

- Selenoprotein N-related Myopathy

## Interventions

- **DRUG:** N-Acetylcysteine followed by Placebo
- **DRUG:** Placebo followed by N-Acetylcysteine

## Key facts

- **NCT ID:** NCT02505087
- **Lead sponsor:** Assistance Publique - Hôpitaux de Paris
- **Sponsor class:** OTHER
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2015-09
- **Primary completion:** 2018-04-24
- **Final completion:** 2020-08-31
- **Target enrollment:** 7 (ACTUAL)
- **Last updated:** 2020-09-29


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02505087

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02505087, "Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT02505087. Licensed CC0.

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