SIDEROS Trial Recruiting

 

A phase 3 clinical trial of the investigational drug idebenone

The main purpose of the phase 3 trial is to determine if idebenone is safe and effective at delaying the loss of respiratory function in boys and men with Duchenne muscular dystrophy. This trial is sponsored by Santhera Pharmaceuticals.

The clinical trial will compare the effectiveness of idebenone versus placebo in boys and men currently on steroids (either prednisone or deflazacort). Previous clinical trials of idebenone in Duchenne studied the ability of idebenone to slow the loss of respiratory function in boys with Duchenne who were not using steroids.

In order to enroll in this phase 3 trial, participants must be:

  • Males with Duchenne
  • 10 years or older
  • On corticosteroid for at least 12 months without dose adjustment in the last 6 months prior to the start of the trial, and
  • Have a Forced Vital Capacity (FVC) between 30% - 80% of predicted value

Additional details regarding inclusion and exclusion criteria are available on ClinicalTrials.gov (NCT02814019).

Approximately 266 participants will be recruited into this study. Each participant will be randomized to receive either idebenone tablets (900 mg/day total to be taken 3 times per day with meals) OR placebo. The study will last 18 months (78 weeks) and will include a screening visit and up to 9 protocol visits, and all visits must be performed at a designated trial site. The primary outcome measure for this study is change in FVC as measured by spirometry (lung function testing) which will be performed at each visit.

This study has >20 sites in the USA. Reasonable travel and accommodations will be provided or reimbursed.

If you wish to participate or have questions, please visit ClinicalTrials.gov (NCT02814019) or SiderosDMD.com. Contact information for each trial site is provided.

Posted 11/16/2017