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.

• Register: EUCTR
• Last refreshed on: 23 November 2020
• Main ID: EUCTR2017-004279-30-SE
• Date of registration: 15/05/2018
• Prospective Registration: Yes
• Primary sponsor: Santhera Pharmaceuticals (Switzerland) Limited
• Public title: 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.
• Scientific title: 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 - SIDEROS-E
• Target sample size: 266
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004279-30
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: Quentin Desvigne
• Affiliation: Santhera Pharmaceuticals (Switzerland) Limited

• Name: Quentin Desvigne
• Affiliation: Santhera Pharmaceuticals (Switzerland) Limited

Key inclusion & exclusion criteria

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:
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.

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


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Raxone
Product Name: Idebenone
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186-27-9
Other descriptive name: IDEBENONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Main Objective: To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
(up to Visit 4/Week 78)

Timepoint(s) of evaluation of this end point: Electrocardiogram: baseline
Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, post-completion follow-up visit

Primary end point(s): Standard safety assessments, including number of premature discontinuations of study treatment due to adverse events, incidence and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG).

Secondary Objective: To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study, classified by background factors including, but not limited to age, DMD history (e.g. time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study.
(up to Visit 4/Week 78)

Secondary Outcome(s)

Secondary end point(s): Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p).

Feasibility of continued idebenone treatment, measured with number of discontinuations of study treatment due to adverse events, will be the only applicable endpoint for the optional continued treatment period (beyond Visit 4).

Timepoint(s) of evaluation of this end point: Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF)Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks.FVC and PEF in optional continued period : 130 weeks, 182
weeks.

Adverse events for the optional continued treatment period : 104 weeks, 130 weeks, 156 weeks, 182 weeks

Secondary ID(s)

2017-004279-30-BE

SNT-III-012-E

103801

Source(s) of Monetary Support

Santhera Pharmaceuticals (Switzerland) Limited