A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

• Register: EUCTR
• Last refreshed on: 17 July 2012
• Main ID: EUCTR2009-012037-30-FR
• Date of registration: 17/12/2009
• Prospective Registration: Yes
• Primary sponsor: Santhera Pharmaceuticals (Switzerland) Limited
• Public title: A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS
• Scientific title: A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS
• Date of first enrolment: 19/02/2010
• Target sample size: 240
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-012037-30
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: Group Sequential Design If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Austria, Belgium, France, Germany, Italy, Netherlands, Spain, Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
1.Patients 10 – 18 years of age at Baseline.
2.Signed and dated informed consent.
3.Documented diagnosis of DMD or severe dystrophinopathy and clinical features consistent of typical DMD at diagnosis (i.e. documented delayed motor skills and muscle weakness by age 5 years). DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostain.
4.Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening).
5.Patients assessed by the investigator as willing and able to comply with the requirements of the study, possess the required cognitive abilities and are able to swallow study medication.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1.Patients dependent on assisted ventilation at Screening and/or Baseline (defined as non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous invasive ventilation).
2.Patients with documented DMD-related hypoventilation for which assisted ventilation is needed according to current standard of care guidelines (e.g. FVC< 30%) or is required in the opinion of the Investigator.
3.Patients with a percent predicted PEF > 80% at Baseline.
4.Patients unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressures.
5.Symptomatic heart failure (high probability of death within one year of Baseline) and/or symptomatic ventricular arrhythmias.
6.Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or SNT-II-001-E) for idebenone.
7.Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Baseline.
8.Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines within 30 days prior to Baseline.
9.Use of coenzyme Q10 or vitamin E (if taken at a dose of 5 times above the daily physiological requirement) within 30 days prior to Baseline.
10.Any previous use of idebenone.
11.Any concomitant medication with a depressive or stimulating effect on respiration or the respiratory tract.
12.Planned or expected spinal fixation surgery during the study period (as judged by the investigator).
13.Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any other non-DMD respiratory illness that affects PEF.
14.Use of bronchodilating medication (i.e. inhaled steroids, sympatomimetics, anti-cholinergics).
15.Moderate or severe hepatic impairment or severe renal impairment.
16.Prior or ongoing medical condition or laboratory abnormality that in the Investigator’s opinion could adversely affect the safety of the subject
17.Relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products/smoking
18.Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the study medication
19. For “glucocorticoid non-users” only
a)Chronic use of systemic glucocorticoid therapy for DMD related conditions within 12 months of Baseline (the “12 month non-use period”)
b)More than 2 rounds of acute systemic glucocorticoid burst therapy (of =2 week duration) for non-DMD related conditions within the 12 month non-use period
c)Use of any round of systemic glucocorticoid burst therapy of longer than 2 weeks duration within the 12 month non-use period
d)Use of systemic glucocorticoid burst therapy less than 8 weeks prior to baseline
20. For “glucocorticoid users” only
a)Prior to Interim Analysis 1: All glucocorticoid users
b)After the Interim Analysis 1: Initiation, cessation or any relevant change (i.e. dose change of >15% above any dose adaptation for body weight increase/decrease) in systemic glucocorticoid therapy within 6 months prior to Baseline.



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

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy
MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy

Intervention(s)

Product Name: Idebenone
Product Code: SNT-MC17
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186279
Current Sponsor code: SNT-MC17
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: •To assess the efficacy of idebenone in improving or delaying the loss of respiratory function using measures other than those used for the primary endpoint
•To assess the efficacy of idebenone in improving or delaying the loss of skeletal muscle strength/motor function
•To assess the efficacy of idebenone in improving or delaying the loss in quality of life
•To assess the safety and tolerability of idebenone in patients with DMD

Main Objective: •To assess the efficacy of idebenone, compared to placebo, in improving or delaying the loss of respiratory function in patients with DMD

Primary end point(s): The change from Baseline to Week 52 in percent predicted Peak Expiratory Flow (PEF)

Secondary ID(s)

2009-012037-30-BE

SNT-III-003