Muscular Dystrophy, DuchennePhase 3DELOSNCT01027884
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
A Phase 3 study testing Idebenone in people with Muscular Dystrophy, Duchenne and Ambulatory Care, run by Santhera Pharmaceuticals at 23 sites in 10 countries.
Trial summary
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed. Read more about this study
- Who is it for?
- Ages 10 to 18 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne, Ambulatory Care
- What is being tested?
- Idebenone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 65 participants
- Where
- 23 sitesin 10 countries
- Study dates
- Jul 2009to Apr 2014
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients 10 - 18 years of age at Baseline.
- Signed and dated informed consent.
- 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.
- Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening).
- 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.
Who cannot join
- Patients dependent on assisted ventilation at Screening and/or Baseline (defined as non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous invasive ventilation).
- 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.
- Patients with a percent predicted PEF > 80% at Baseline.
- Patients unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressures.
- Symptomatic heart failure (high probability of death within one year of Baseline) and/or symptomatic ventricular arrhythmias.
- Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or SNT-II-001-E) for idebenone.
Show all 25Show fewer
- Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Baseline.
- Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines within 30 days prior to Baseline.
- 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.
- Any previous use of idebenone.
- Any concomitant medication with a depressive or stimulating effect on respiration or the respiratory tract.
- Planned or expected spinal fixation surgery during the study period (as judged by the investigator).
- Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any other non-DMD respiratory illness that affects PEF.
- Chronic use of beta-2 agonists or any use of other bronchodilating medication (e.g. inhaled steroids, sympathomimetics, anticholinergics).
- Please note: Chronic use if defined as a daily intake for more than 14 days.
- Moderate or severe hepatic impairment or severe renal impairment.
- Prior or ongoing medical condition or laboratory abnormality that in the Investigator's opinion could adversely affect the safety of the subject.
- Please note: Patients who suffer from a severe, unstable condition including (but not limited to) cancer, auto-immune diseases, haematological diseases, metabolic disorders or immunodeficiencies, and who are at risk of an aggravation unrelated to the study condition, can only be included in the study if accepted in writing by the Sponsor's Medical Monitor.
- Relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products/smoking
- Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the study medication
- Systemic glucocorticoid therapy
- Chronic use of systemic glucocorticoid therapy for DMD related conditions within 12 months of Baseline (the "12 month non-use period")
- 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
- Use of any round of systemic glucocorticoid burst therapy of longer than 2 weeks duration within the 12 month non-use period
- Use of systemic glucocorticoid burst therapy less than 8 weeks prior to baseline
Trial locations and contacts
23 sites in 10 countries.
23 sites in 10 countries
United States9 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Carolinas Medical Center, Neurosciences and Spine Institute
Charlotte, North Carolina, 28207
University of Texas Southwestern Medical Center
Dallas, Texas, 75390-9105
University of Florida
Gainesville, Florida, 32610
Methodist Neurological Institute
Houston, Texas, 77030
Monroe Carell, Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-1771
University of California Davis Medical Center
Sacramento, California, 95817
Seattle Children's Hospital
Seattle, Washington, 98105
Italy3 sites
Fondazione IRCCS "Eugenio Medea"
Bosisio Parini, Lecco, 23842
Azienda Ospedaliera Niguarda Ca' Granda Centro Clinico Nemo
Milan, 20162,
Azienda Ospedaliera Universitaria della Seconda Università degli Studi di Napoli
Naples, 80138
France2 sites
Hôpital Roger Salengro, CHRU Lille
Lille, 59037
Prof. Thomas Voit , MD, PhD
Paris, 75651
Germany2 sites
Universitätsklinikum Essen, Zentrum für Kinderheikunde
Essen, D-45122
Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin
Freiburg im Breisgau, 79106
Spain2 sites
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario y Politécnico La Fe
Valencia, 46009
Netherlands1 site
Ass. Prof. Jan Verschuuren , MD, PhD
Leiden, P.O. Box 9600, 2300 RC
About this study
Official 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
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed.
Read the detailed description
This study was a Phase III, multicenter, randomized, double-blind, placebo-controlled efficacy and safety study. DMD patients (ambulatory and non-ambulatory) at age 10-18 years were enrolled at sites in Europe and North America. Study subjects were randomized in a 1:1 ratio to receive either idebenone (900 mg/day) or placebo 3 times a day with meals for 52 weeks. The primary endpoint was the difference between Catena®/Raxone® and placebo in the change from Baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p, a measure of respiratory muscle strength) as measured by hospital-based spirometry. PEF was also measured by the patient at home using the hand-held ASMA-1 device (secondary endpoint). Other respiratory endpoints included Forced Expiratory Volume in 1 second (as percent predicted, FEV1%p, an additional measure of respiratory muscle strength) and Forced Vital Capacity (as percent predicted, FVC%p, a measure of restrictive lung disease predictive of morbidity and mortality in DMD).
- Conditions
- Muscular Dystrophy, Duchenne, Ambulatory Care
- Being tested
- Placebo, Idebenone
- Keywords
- Idebenone, Duchenne Muscular Dystrophy (DMD), Respiratory function, Ambulatory and non-ambulatory patients, Subjects not using glucocorticoids
- Medical subject headings
- Muscular Dystrophy, Duchenne, Respiratory Aspiration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Placebo (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals
IdebenoneDrug
Also called CATENA®, RAXONE®
Idebenone (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo 900 mg/day Receives: Drug: Placebo |
| Idebenone | Experimental | Idebenone 900 mg/day Receives: Drug: Idebenone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Measured: Baseline and Week 52
Secondary outcomes (4)
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Measured: Baseline and Week 52
Change From Baseline to Week 52 in Muscle Strength
The change from Baseline to Week 52 in muscle strength as measured by Hand-Held Myometry (HHM) was performed following standardized procedures. As almost all patients were non-ambulatory, only analyses of upper limb muscle strength were performed. Results for elbow flexors and for elbow extensors are reported below.The highest value of 3 consecutive measurements with an interval of at least 10 seconds were recorded.
The HHM was measured using MicroFET2, a digital hand held muscle tester. The selected unit of measure was Newtons (N).
Measured: Baseline and Week 52
Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory
PedsQL Quality of Life Inventory contains paediatric HRQOL measurements: Physical, Emotional,Social and School Functioning.
Item Scaling:
5-point Likert scale from 0 (Never) to 4 (Almost always). 3-point scale: 0 (Not at all), 2 (Sometimes) and 4 (A lot) for the Young Child (ages 5-7).
Scores are transformed on a scale from 0 to 100 ( 0=100, 1=75, 2=50, 3=25, 4=0) Total Score: Sum of all the items over the number of items answered on all the Scales.
The values reported below are overall scores on Paediatric Quality of Life Inventory in Child/Teen Report. These scores were obtained by averaging scores for all the described subscales. The overall scores range between 0-100 with 0 = worst outcome and 100= best outcome
Measured: Baseline and Week 52
Percentage of Patients Reporting Adverse Events
Measured: 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2009 (estimated)
- Study startJul 2009
- Primary completionJan 2014
- Study completionApr 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Prof. Gunnar Buyse, MD, PhD., Principal investigator, University Hospitals Leuven, B-3000, BelgiumDr. Ulrike Schara, MD, PhD, Principal investigator, Universitätsklinikum Essen, D-45122 Essen, GermanyAss. Prof. Jan Verschuuren, MD, PhD, Principal investigator, Leiden University Medical Center (LUMC), 2300 RC Leiden, the NetherlandsDr. Pierre-Yves Jeannet, Médecin Associé, MER, Principal investigator, Unité de Neuropédiatrie, CHUV - BH11, 1011 Lausanne-CH, SwitzerlandProf. Thomas Voit, MD, PhD, Principal investigator, Université Pierre et Marie curie VI - Institut de Myologie - groupe hospitalier Pitié Salpétrière - 47/83 boulevard de l'hôpital, 75651 Paris Cedex 13, FranceProf. Thomas Sejersen, MD, PhD, Principal investigator, Astrid Lindgrens Barnsjukhus- Karolinska Universitetssjukhuset, SE-17176 Stockholm, SwedenDr. Günther Bernert, Prim. Univ. Doz., Principal investigator, Vorstand der Abteilung für Kinder- und Jugendheilkunde, Gottfried v. Preyer'sches Kinderspital, 1100 Wien, AustriaGihan Tennekoon, MD, Principal investigator, Division of Neurology - The Children's Hospital of Philadelphia - 34th Street and Civic Center Blvd, Philadelphia, PA 19104-1771, USA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01027884
- Sponsor's protocol ID
- SNT-III-003
- Acronym
- DELOS
- Status last verified
- Sep 2015
- First submitted
- 8 Dec 2009
- Last updated
- 19 Oct 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T; DELOS Study Group. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet. 2015 May 2;385(9979):1748-1757. doi: 10.1016/S0140-6736(15)60025-3. Epub 2015 Apr 20.
PubMed 25907158Meier T, Rummey C, Leinonen M, Spagnolo P, Mayer OH, Buyse GM; DELOS Study Group. Characterization of pulmonary function in 10-18 year old patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2017 Apr;27(4):307-314. doi: 10.1016/j.nmd.2016.12.014. Epub 2017 Jan 6.
PubMed 28189481
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT01027884
- Acronym / sponsor ID
- DELOS / SNT-III-003
- Conditions
- Muscular Dystrophy, Duchenne, Ambulatory Care
- Keywords
- Idebenone, Duchenne Muscular Dystrophy (DMD), Respiratory function, Ambulatory and non-ambulatory patients, Subjects not using glucocorticoids
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jul 2009
- Primary completion
- Jan 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 9 Dec 2009 (estimated)
- Results
- Posted 19 Oct 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed.
Detailed description
This study was a Phase III, multicenter, randomized, double-blind, placebo-controlled efficacy and safety study. DMD patients (ambulatory and non-ambulatory) at age 10-18 years were enrolled at sites in Europe and North America. Study subjects were randomized in a 1:1 ratio to receive either idebenone (900 mg/day) or placebo 3 times a day with meals for 52 weeks. The primary endpoint was the difference between Catena®/Raxone® and placebo in the change from Baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p, a measure of respiratory muscle strength) as measured by hospital-based spirometry. PEF was also measured by the patient at home using the hand-held ASMA-1 device (secondary endpoint). Other respiratory endpoints included Forced Expiratory Volume in 1 second (as percent predicted, FEV1%p, an additional measure of respiratory muscle strength) and Forced Vital Capacity (as percent predicted, FVC%p, a measure of restrictive lung disease predictive of morbidity and mortality in DMD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo 900 mg/day |
| Idebenone | Experimental | Drug: IdebenoneIdebenone 900 mg/day |
Who may qualify
- Ages
- 10 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients 10 - 18 years of age at Baseline.
- Signed and dated informed consent.
- 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.
- Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening).
- 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.
Key exclusion criteria (25 listed)
- Patients dependent on assisted ventilation at Screening and/or Baseline (defined as non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous invasive ventilation).
- 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.
- Patients with a percent predicted PEF > 80% at Baseline.
- Patients unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressures.
- Symptomatic heart failure (high probability of death within one year of Baseline) and/or symptomatic ventricular arrhythmias.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboPlacebo (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals | Drug | Placebo |
| IdebenoneAlso: CATENA®, RAXONE®Idebenone (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals | Drug | Idebenone |
Primary outcome
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Time frame: Baseline and Week 52
Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
4 secondary outcomes
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Time frame: Baseline and Week 52
Change From Baseline to Week 52 in Muscle Strength
Time frame: Baseline and Week 52
Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory
Time frame: Baseline and Week 52
Percentage of Patients Reporting Adverse Events
Time frame: 52 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 25907158Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T; DELOS Study Group. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet. 2015 May 2;385(9979):1748-1757. doi: 10.1016/S0140-6736(15)60025-3. Epub 2015 Apr 20.
- Derived from this study: PubMed 28189481Meier T, Rummey C, Leinonen M, Spagnolo P, Mayer OH, Buyse GM; DELOS Study Group. Characterization of pulmonary function in 10-18 year old patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2017 Apr;27(4):307-314. doi: 10.1016/j.nmd.2016.12.014. Epub 2017 Jan 6.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Prof. Gunnar Buyse, MD, PhD. principal investigator
University Hospitals Leuven, B-3000, Belgium
Dr. Ulrike Schara, MD, PhD principal investigator
Universitätsklinikum Essen, D-45122 Essen, Germany
Ass. Prof. Jan Verschuuren, MD, PhD principal investigator
Leiden University Medical Center (LUMC), 2300 RC Leiden, the Netherlands
23 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Idebenone in people with Muscular Dystrophy, Duchenne and Ambulatory Care, run by Santhera Pharmaceuticals at 23 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Italy, France, Germany, Spain and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Oct 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52 (time frame: Baseline and Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01027884, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2015.