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

Santhera

Status and phase

Terminated

Phase 3

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: idebenone 150 mg film-coated tablets

Study type

Interventional

Funder types

Industry

Identifiers

NCT03603288

SNT-III-012-E

Details and patient eligibility

About

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.

Full 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.

Enrollment

161 patients

Sex

Male

Ages

11+ years old

Volunteers

No Healthy Volunteers

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