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

NCT02505087 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2020-09-29

No results posted yet for this study

Summary

The objective of this study is to determine whether the administration of N-acetylcysteine (NAC) improves oxidative stress. To determine this, the study will assess the impact of oral treatment on the balance between reduced and oxidized form of glutathione in erythrocytes of peripheral blood.

Conditions

  • Selenoprotein N-related Myopathy

Interventions

DRUG

N-Acetylcysteine followed by Placebo

Arm receiving N-Acetylcysteine (NAC) for 6 months and then placebo for 6 months. Treatment periods are separated by a 2 month washout.

DRUG

Placebo followed by N-Acetylcysteine

Arm receiving placebo for 6 months then N-acetylcysteine (NAC) for 6 months. Treatment periods are separated by a 2 month washout.

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Brigitte ESTOURNET, MD, PhD · Neuro-Respiratory Rehabilitation Service, Raymond Poincaré Hospital, 92380 Garches, France

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2015-09-30
Primary Completion
2018-04-24
Completion
2020-08-31

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02505087 on ClinicalTrials.gov