Summary

for males ages 11 years and up (full criteria)
at Sacramento, California and other locations
study started
estimated completion

Description

Summary

The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.

Official Title

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study

Details

The study is an open-label, single-group, multi-center extension study in patients with DMD receiving glucocorticoid steroids who participated in the SIDEROS study and who meet all the inclusion criteria and none of the exclusion criteria for this extension study.

The study consists of 4 study visits scheduled every 6 months (Visit 1/Baseline, Visit 2/Week 26, Visit 3/ Week 52 and Visit 4/ Week 78), and a follow-up visit 4 weeks after treatment discontinuation. Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.

Keywords

Duchenne Muscular Dystrophy respiratory function in DMD Muscular Dystrophies Muscular Dystrophy, Duchenne Ubiquinone Idebenone idebenone 150 mg film-coated tablets

Eligibility

You can join if…

Open to males ages 11 years and up

  1. Completion of the SIDEROS study at Visit 8/ Week 78
  2. Signed and dated Informed Consent Form for SIDEROS-E

You CAN'T join if...

  1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
  2. Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study
  3. Use of any investigational drug other than the study medication

Locations

  • UC Davis Department of Physical Medicine and Rehabilitation accepting new patients
    Sacramento California 95817 United States
  • Gillette Children's Specialty Healthcare accepting new patients
    Saint Paul Minnesota 55101 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Santhera Pharmaceuticals
Links
SIDEROS study website
Sign up for this study
ID
NCT03603288
Phase
Phase 3
Study Type
Interventional
Last Updated