A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

Phase 1

• Conditions: Duchenne Muscular Dystrophy (DMD); MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2017-004279-30-BE
• Lead Sponsor: Santhera Pharmaceuticals (Switzerland) Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-05-04
• Last Update Posted: 19 October 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 266

Inclusion Criteria

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

Inclusion criteria for the optional continued treatment with idebenone beyond Visit 4 of SIDEROS-E study:
1. Completion of Visit 4/ Week 78 of SIDEROS-E study
2. Signed and dated Informed Consent Form for continued treatment
Are the trial subjects under 18? yes
Number of subjects for this age range: 266
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Enrolment in SIDEROS-E of siblings of randomized SIDEROS patients is allowed if they meet all the inclusion and none of the exclusion criteria above.

Exclusion criteria for the optional continued treatment with idebenone beyond Visit 4/ Week 78 of SIDEROS-E study:

1. Premature withdrawal from SIDEROS-E study before Visit 4/ Week 78
2. Any conditions, which, in the opinion of the Investigator might trigger a negative risk-benefit assessment for the patient
3. Use of any Investigational drug other than the study medication

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified