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Rimeporide in Patients With Duchenne Muscular Dystrophy

NCT02710591 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2019-07-18

Study results available
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Summary

In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime. Sodium/proton type 1 exchanger (NHE-1) inhibition is an innovative pathway that has proved to efficiently prevent the accumulation of muscle damage (inflammation and fibrosis) in animal models of muscular dystrophies and heart failure. Based on prior safety and efficacy results in animal and humans, NHE-1 inhibition with Rimeporide represents a new therapeutic approach with no restriction on age and on genetic subtypes which could be combined to other treatments that restore or augment dystrophin.This study examines the safety and tolerability and effects on the muscles of rimeporide, in patients aged 6 to 14 years with Duchenne Muscular Dystrophy (DMD).

Conditions

  • Muscular Dystrophy, Duchenne

Interventions

DRUG

Rimeporide

Cohort 1: 50 mg TID in patients with a body weight ≤ 30kg at Baseline and 75 mg TID in patients with a body weight \> 30kg at Baseline Cohort 2: 100mg TID in patients with a body weight ≤ 30kg at baseline and 150 mg TID in patients with a body weight \> 30kg at Baseline Cohort 3: 150 mg TID in patients with a body weight ≤ 30kg at baseline and 200 mg TID in patients with a body weight \> 30kg at Baseline Cohort 4: 200 mg TID in patients with a body weight ≤ 30kg at Baseline and 300 mg TID mg TID in patients with a body weight \> 30kg at Baseline

Sponsors & Collaborators

  • EspeRare Foundation

    lead OTHER

Principal Investigators

  • Florence Porte-Thomé · R&D Director EspeRare

Study Design

Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Max Age
14 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-03-31
Primary Completion
2017-12-31
Completion
2018-02-28

Countries

  • France
  • Italy
  • Spain
  • United Kingdom

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 NCT02710591 on ClinicalTrials.gov