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Trial record 1 of 6 for:    idebenone | Duchenne Muscular Dystrophy
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Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03603288
Recruitment Status : Recruiting
First Posted : July 27, 2018
Last Update Posted : October 14, 2019
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE May 31, 2018
First Posted Date  ICMJE July 27, 2018
Last Update Posted Date October 14, 2019
Actual Study Start Date  ICMJE July 4, 2018
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 26, 2018)
  • 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03603288 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 26, 2018)
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
Official Title  ICMJE 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
Brief 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.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
Open-Label Extension Study
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: idebenone 150 mg film-coated tablets
900 mg idebenone/day
Study Arms  ICMJE Experimental: idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
Intervention: Drug: idebenone 150 mg film-coated tablets
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 26, 2018)
266
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2024
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

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

Exclusion Criteria:

  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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 11 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jodi Wolff +1 (520) 373-0069 sideros@santhera.com
Listed Location Countries  ICMJE Austria,   Belgium,   France,   Germany,   Italy,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03603288
Other Study ID Numbers  ICMJE SNT-III-012-E
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Santhera Pharmaceuticals
Study Sponsor  ICMJE Santhera Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Santhera Pharmaceuticals
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP