A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

• Register: EUCTR
• Last refreshed on: 12 February 2018
• Main ID: EUCTR2016-000602-10-IT
• Date of registration: 06/02/2018
• Prospective Registration: No
• Primary sponsor: SANTHERA PHARMACEUTICALS
• Public title: A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids
• Scientific title: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS
• Date of first enrolment: 20/10/2016
• Target sample size: 266
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000602-10
• 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: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• 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, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: Irina Coserea
• Affiliation: Santhera Pharmaceuticals (Switzerland) Limited

• Name: Irina Coserea
• Affiliation: Santhera Pharmaceuticals (Switzerland) Limited

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male patients with a 30% = FVC = 80% of predicted value at
Screening and at Baseline.
2. Minimum 10 years old at Screening
3. Signed and dated Informed Consent Form.
4. Documented diagnosis of DMD (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 immunostaining.
5. Chronic use of systemic glucocorticoid steroids for DMD
related conditions continuously for at least 12 months prior to
Baseline without any dose adjustments on a mg/kg basis in
the last 6 months (only dose adjustment determined by weight
changes are allowed).
6. Ability to provide reliable and reproducible repeat FVC within
15% of the screening assessment at Baseline.
7. 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.
8. Patients who have been immunized with 23-valent
pneumococcal polysaccharide vaccine or any other
pneumococcal polysaccharide vaccine as per national
recommendations, as well as annually immunized with
inactivated influenza vaccine.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
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) yes
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
1. Symptomatic heart failure (defined as Stage C by ACCF/AHA
guideline or NYHA III-IV) and/or symptomatic ventricular
arrhythmias.
2. Ongoing participation in any other therapeutic trial and/or
intake of any investigational drug within 90 days prior to
Baseline (only exception allowed is use of Deflazacort in US
as part of the Expanded Access Program).
3. Prior or ongoing exon-skipping or read-through gene therapy
for DMD.
4. Planned or expected spinal fixation surgery during the study
period (as judged by the Investigator, i.e. due to rapidly
progressing scoliosis), prior spinal fixation surgery is allowed if
it took place more than 6 months prior to Screening.
5. Asthma, bronchitis/COPD, bronchiectasis, emphysema,
pneumonia or presence of any other non-DMD respiratory
illness that affects respiratory function.
6. Chronic use of beta2-agonists or any use of other
bronchodilating/bronchoconstricting medication (inhaled
steroids, sympathomimetics, anti-cholinergics, antihistamines);
chronic use is defined as a daily intake for more than 14 days.
7. Any bronchopulmonary illness that required treatment with
antibiotics within 3 months prior to Screening.
8. Moderate or severe hepatic impairment (Child-Pugh class B [7
to 9 points] or Child-Pugh class C [10 to 15 points]) or severe
renal impairment (eGFR <30 mL/min/1.73 m2).
9. Prior or ongoing medical condition or laboratory abnormality
which in the Investigator’s opinion may put the patient at
significant risk, may confound the study results or may
interfere significantly with the patient’s participation in the
study1.
10. History of or current drug or alcohol abuse or use of any
tobacco/marijuana products/smoking.
11. Known individual hypersensitivity to idebenone or to any of the
ingredients/excipients of the study medication.
12. Daytime ventilator assistance (defined as use of any assisted
ventilation while awake).


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

Health Condition(s) or Problem(s) studied

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

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

Intervention(s)

Trade Name: RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186-27-9
Current Sponsor code: IDEBENONE
Other descriptive name: IDEBENONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Main Objective: To assess the efficacy of idebenone compared to placebo, in delaying
the loss of respiratory function in patients with DMD receiving
glucocorticoid steroids as measured by changes in Forced Vital Capacity
percent predicted (FVC %p) using hospital-based spirometry

Secondary Objective: ? To assess the efficacy of idebenone compared to placebo in delaying
the loss of respiratory function in patients with DMD receiving
glucocorticoid steroids as measured by:
? Changes in Peak Expiratory Flow percent predicted (PEF %p) using
hospital-based spirometry
? Time to loss of 10% of Baseline Forced Vital Capacity (FVC) using
hospital-based spirometry
? To assess the efficacy of idebenone compared to placebo in delaying
the loss of inspiratory muscle function as measured by changes in
Inspiratory Flow Reserve (IFR) using hospital-based spirometry

Timepoint(s) of evaluation of this end point: 78 weeks

Primary end point(s): • The change from Baseline to Week 78 in FVC %p assessed by
hospital-based spirometry measurements

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Secondary endpoint 1: 78 weeks
Secondary endpoint 2: 13, 26, 39, 52, 65 and 78 weeks
Secondary endpoint 3: 78 weeks

Secondary end point(s): The secondary endpoints will be evaluated in the following order in a
hierarchical manner:
• The change from Baseline to Week 78 in PEF %p assessed by
hospital-based spirometry measurements
• The time to first 10% decline in FVC (L) during the 78-week
treatment period, assessed by hospital-based spirometry
measurements
• The change from Baseline to Week 78 in IFR assessed by
hospital-based spirometry measurements

Secondary ID(s)

103801

2016-000602-10-DE

SNT-III-012

NCT02814019

Source(s) of Monetary Support

Santhera Pharmaceuticals (Switzerland) Limited