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Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

key information

study id #: NCT03603288

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

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.

intervention: idebenone 150 mg film-coated tablets

mechanism of action: Antioxidant to support respiratory health

results: https://clinicaltrials.gov/ct2/show/results/NCT03603288

last updated: April 27, 2019

study details

start date: July 4, 2018

estimated completion: December 2023

phase of development: Phase 3

size / enrollment: 266

study description: The study will be 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). Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.

primary outcomes:

  • Incidence and severity of adverse events, as per ICH Topic E2A [ Time Frame: From baseline until visit 4 (week 78) ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Incidence and severity of adverse events, as per ICH Topic E2A [ Time Frame: 4 weeks after discontinuation of treatment ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with premature discontinuations of study treatment due to adverse events. [ Time Frame: From baseline until visit 4 (week 78) ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with abnormal safety laboratory parameters. [ Time Frame: From baseline until visit 4 (week 78) ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with abnormal safety laboratory parameters. [ Time Frame: 4 weeks after discontinuation of treatment ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with abnormal vital signs. [ Time Frame: From baseline until visit 4 (week 78) ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with abnormal vital signs. [ Time Frame: 4 weeks after discontinuation of treatment ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
  • Number of patients with abnormal ECG. [ Time Frame: From baseline until visit 4 (week 78) ]
    To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

secondary outcomes:

  • Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). [ Time Frame: From baseline until visit 4 (week 78) ]
    To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
  • Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) [ Time Frame: From baseline until visit 4 (week 78) ]
    To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
  • Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p) [ Time Frame: From baseline until visit 4 (week 78) ]
    To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.

inclusion criteria:
• Completion of the SIDEROS study at Visit 8/ Week 78
• Signed and dated Informed Consent Form for SIDEROS-E

exclusion criteria:
• Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
• 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
• Use of any investigational drug other than the study medication

study contacts

sponsor: Santhera Pharmaceuticals

contacts: Jodi Wolff, +1 (520) 373-0069, sideros@santhera.com

locations: United States, Austria, Belgium, France, Italy, Spain, Switzerland, United Kingdom