Duchenne Muscular Dystrophy (DMD)Phase 3SIDEROSNCT02814019
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
A Phase 3 study testing Idebenone 150 mg film-coated tablets in people with Duchenne Muscular Dystrophy (DMD), run by Santhera Pharmaceuticals at 63 sites in 15 countries.
Trial summary
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids Read more about this study
- Who is it for?
- Ages 10 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Idebenone 150 mg film-coated tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 255 participants
- Where
- 63 sitesin 15 countries
- Study dates
- Sep 2016to 1 Dec 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male patients with a 35% ≤ FVC ≤ 80% of predicted value at Screening and at Baseline and who, in the opinion of the investigator are in the respiratory function decline phase.
- Minimum 10 years old at Screening.
- Signed and dated Informed Consent Form.
- 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.
- 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 adjustments determined by weight changes are allowed).
- Ability to provide reliable FVC values at Screening and Baseline, and reproducible within 15% (relative change) at Baseline compared to 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.
- Patients who prior to Screening 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.
Who cannot join
- Symptomatic heart failure (defined as patients with structural heart disease, dyspnea, fatigue and impaired tolerance to exercise; Stage C by the ACCF/AHA guideline or NYHA Classes III-IV) and/or symptomatic ventricular arrhythmias.
- 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 the US as part of the Expanded Access Program, or any approved corticosteroid product in trial for regimen optimization, for which the patient met the inclusion criterion 5).
- Ongoing exon-skipping therapy or read-through gene therapy for DMD; previous exon-skipping or read-through gene therapy is allowed if the stop date was more than 6 months prior to Screening.
- Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapidly progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 months prior to Screening.
- Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or presence of any other non-DMD respiratory illness that affects respiratory function.
- Chronic use of beta2-agonists or any use of other bronchodilating/bronchoconstricting medication (inhaled steroids, sympathomimetics, anticholinergics, antihistamines); chronic use is defined as a daily intake for more than 14 days.
Show all 13Show fewer
- Any bronchopulmonary illness that required treatment with antibiotics within 3 months prior to Screening.
- Moderate or severe hepatic impairment (use as guidance Child-Pugh class B [7 to 9 points] or Child-Pugh class C [10 to 15 points] - see Appendix B) or severe renal impairment (eGFR <30 mL/min/1.73 m2).
- Prior or ongoing medical condition or laboratory abnormality that 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 study.
- Relevant history of or current drug or alcohol abuse, or use of any tobacco or marijuana products/smoking.
- Known individual hypersensitivity to idebenone or to any of the ingredients or excipients of the study medication.
- Daytime ventilator assistance (defined as use of any assisted ventilation while awake).
- Note: Patients who suffer from a severe, unstable condition including (but not limited to) cancer, auto-immune diseases, hematological 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 Senior Clinical Research Physician.
Trial locations and contacts
63 sites in 15 countries.
63 sites in 15 countries
United States20 sites
Rare Disease Research
Atlanta, Georgia, 30318
Johns Hopkins University
Baltimore, Maryland, 21287
University of Alabama
Birmingham, Alabama, 35233
Children's Hospital Boston
Boston, Massachusetts, 02115
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte, North Carolina, 28207
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229-3039
MetroHealth Medical Center
Cleveland, Ohio, 44109-1988
University of Kansas
Fairway, Kansas, 66103
Cook Children's Medical Center
Fort Worth, Texas, 76087
University of Iowa
Iowa City, Iowa, 52242
Childrens Hospital of Los Angeles
Los Angeles, California, 90027
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-1771
Phoenix Children's Hospital
Phoenix, Arizona, 85016
University of Rochester Medical Center
Rochester, New York, 14642
UC Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Loma Linda University Healthcare
San Bernardino, California, 92354
Shriners Hospitals for Children-Tampa
Tampa, Florida, 33612
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Italy8 sites
Fondazione IRCCS Eugenio Medea
Bosisio Parini, 23842
Istituto Giannina Gaslini
Genova, 16147
Scientific Coordinator Nemo Sud Clinical Center
Messina, 98125
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milan
Servizio di Cardiomiologia e Genetica Medica, AOU Università degli Studi della Campania "Luigi Vanvitelli"
Naples, 80131
Reparto Di Neurologia dell'Osperdale Di Padova
Padova, 35122
Dipartimento di Clinica Neurologica e Psichiatrica dell'Età Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
Pavia, 27100
U.O.C. Neuropsichiatria Infantile
Roma, 00168
Germany7 sites
Universitätsmedizin Berlin Campus Virchow-Klinikum
Berlin, 13353
Uniklinik Köln
Cologne, 50937
Universitätsklinikum Essen
Essen, 45147
Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin
Freiburg im Breisgau
Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin
Hamburg, 20246
Universitätsklinikum Heidelberg Zentrum für Kinder- und Jugendmedizin
Heidelberg
Zentrum für neuromuskuläre Erkrankungen
München
United Kingdom7 sites
Leeds Teaching Hospital NHS Trust
Leeds, LS1 3EX
UCL, National Hospital for Neurology and Neurosurgery
London, WC1 3BG
Great Ormond Street Hospital for Children
London, WC1N 3JH
John Walton Muscular Dystrophy Research Centre
Newcastle
Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, SY10 7AG
Oxford University hospitals NHS Foundation Trust
Oxford, OX39DU
Royal Hallamshire Hospital
Sheffield, S10 2JF
France5 sites
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
Lille, 59037
CHRU de Montpellier - Hôpital Gui de Chauliac
Montpellier, 34295
Hôpital Hôtel Dieu
Nantes, 44093
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20
Paris
Hôpital des enfants
Toulouse, 31059
Spain5 sites
Hospital Sant Joan de Déu Neuropediatra
Barcelona, 08950
Hospital Universitari Vall D' Hebron
Barcelona, 8950
Hospital Universitario La Paz
Madrid, 28046
Hospital Universitario Virgen del Rocio
Seville, 41013
Hospital La Fe de Valencia
Valencia, 106 46026
Belgium2 sites
University Hospital Leuven
Leuven, 3000
Centre de Référence Neuromusculaire, CHR Citadelle
Liège, 4000
Netherlands2 sites
LUMC
Leiden
Radboud university medical centre
Nijmegen, 6500
Israel1 site
Institute of Neurology at Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
Switzerland1 site
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
Basel, 4301
About this study
Official 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
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
Read the detailed description
The SIDEROS trial is a randomized, placebo controlled, parallel group study of the efficacy of idebenone in delaying the loss of respiratory function, whilst also monitoring safety and tolerability of idebenone in at least 266 DMD patients taking stable dose of concomitant glucocorticoid steroids.
The study treatment period will be 18 months/ 78 weeks and the idebenone dose will be 900 mg/day. Participants can use deflazacort or prednisolone and be on any dose regimen.
Since glucocorticoid steroids are widely used in ambulant boys from an early age until late into teenage and even adult years, this study will not take age and ambulatory status into account and will only exclude patients that need daytime ventilator assistance.
The schedule of assessments will include a Screening Visit and up to 9 protocol visits, including a Follow-up Visit.
A Screening Visit will take place a maximum of 4 weeks prior to the Baseline Visit (Visit 1, study day -1). Beginning at Baseline, the patient will receive study medication to be taken at home, and will undergo regular assessments in the clinic throughout the study period until Visit 8 at Week 78 at which time the study will be completed and medication discontinued.
All patients completing Visit 8/Week 78, and considered eligible by the Investigator will be able to participate in an open-label extension study (SIDEROS-E) and will continue to receive idebenone until idebenone is commercially available for patients included in the study or SIDEROS-E is terminated by the Sponsor, whichever occurs first. The duration of the SIDEROS-E study will be defined in a separate protocol.
For all patients not participating in the extension study (SIDEROS-E), a Follow-up Visit (Visit 9/Follow-up Visit) will take place 4 weeks after end of Treatment at Visit 8/Week 78 or after premature discontinuation of study medication.
Each hospital visit will include efficacy assessments (respiratory function assessed by hospital-based spirometry, oxygen saturation, end-tidal CO2) and safety assessments (adverse events, concomitant medication, physical examination, vital signs, safety laboratory evaluations). In addition, respiratory function will be assessed weekly at home with a hand-held device in order to closely monitor respiratory function between hospital visits.
The study medication, all medical procedures and laboratory testing, and the visits to the study centre are free of charge. In addition the patients will receive a travel allowance to cover reasonable expenses to and from the study centre. Participants will not otherwise be compensated for this study.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Idebenone 150 mg film-coated tablets, placebo
- Keywords
- respiratory function in DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Idebenone 150 mg film-coated tabletsDrug
placeboDrug
| Study group | Role | What they receive |
|---|---|---|
| idebenone 150 mg film-coated tablets | Experimental | 900 mg idebenone/day (2 tablets to be taken 3 times a day with meals) Receives: Drug: Idebenone 150 mg film-coated tablets |
| placebo | Placebo comparator | matching placebo tablets Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Forced Vital Capacity percent predicted (FVC %p) at Week 78
Delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in FVC %p from Baseline to Week 78 using hospital based spirometry.
Measured: 78 weeks
Secondary outcomes (3)
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF %p) at Week 78
Delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by:
•The change from Baseline to Week 78 in PEF %p assessed by hospital-based spirometry measurements
Measured: 78 weeks
Change From Baseline in Forced Vital Capacity (FVC) at Week 78
Delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by:
•The time to first 10% decline in FVC (L) during the 78-week treatment period, assessed by hospital-based spirometry measurements
Measured: 78 weeks
Change from Baseline in Inspiratory Flow Reserve (IFR) at Week 78
Delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by:
•The change from Baseline to Week 78 in IFR assessed by hospital-based spirometry measurements
Measured: 78 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2016 (estimated)
- Study startSep 2016
- Primary completion1 Dec 2020
- Study completion1 Dec 2020
Why it stopped: Interim analysis concluded to futility
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- 255 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02814019
- Sponsor's protocol ID
- SNT-III-012
- Acronym
- SIDEROS
- Status last verified
- Nov 2021
- First submitted
- 17 Jun 2016
- Last updated
- 3 Dec 2021
- Results posted
- No
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 25907158McDonald CM, Meier T, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM; DELOS Study Group. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2016 Aug;26(8):473-80. doi: 10.1016/j.nmd.2016.05.008. Epub 2016 May 12.
PubMed 27238057Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM; DELOS Study Group. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatr Pulmonol. 2017 Apr;52(4):508-515. doi: 10.1002/ppul.23547. Epub 2016 Aug 29.
PubMed 27571420
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02814019
- Acronym / sponsor ID
- SIDEROS / SNT-III-012
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- respiratory function in DMD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Interim analysis concluded to futility
- Enrolment
- 255 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2016
- Primary completion
- 1 Dec 2020 (actual)
- Study completion
- 1 Dec 2020 (actual)
- First posted
- 27 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
Detailed description
The SIDEROS trial is a randomized, placebo controlled, parallel group study of the efficacy of idebenone in delaying the loss of respiratory function, whilst also monitoring safety and tolerability of idebenone in at least 266 DMD patients taking stable dose of concomitant glucocorticoid steroids.
The study treatment period will be 18 months/ 78 weeks and the idebenone dose will be 900 mg/day. Participants can use deflazacort or prednisolone and be on any dose regimen.
Since glucocorticoid steroids are widely used in ambulant boys from an early age until late into teenage and even adult years, this study will not take age and ambulatory status into account and will only exclude patients that need daytime ventilator assistance.
The schedule of assessments will include a Screening Visit and up to 9 protocol visits, including a Follow-up Visit.
A Screening Visit will take place a maximum of 4 weeks prior to the Baseline Visit (Visit 1, study day -1). Beginning at Baseline, the patient will receive study medication to be taken at home, and will undergo regular assessments in the clinic throughout the study period until Visit 8 at Week 78 at which time the study will be completed and medication discontinued.
All patients completing Visit 8/Week 78, and considered eligible by the Investigator will be able to participate in an open-label extension study (SIDEROS-E) and will continue to receive idebenone until idebenone is commercially available for patients included in the study or SIDEROS-E is terminated by the Sponsor, whichever occurs first. The duration of the SIDEROS-E study will be defined in a separate protocol.
For all patients not participating in the extension study (SIDEROS-E), a Follow-up Visit (Visit 9/Follow-up Visit) will take place 4 weeks after end of Treatment at Visit 8/Week 78 or after premature discontinuation of study medication.
Each hospital visit will include efficacy assessments (respiratory function assessed by hospital-based spirometry, oxygen saturation, end-tidal CO2) and safety assessments (adverse events, concomitant medication, physical examination, vital signs, safety laboratory evaluations). In addition, respiratory function will be assessed weekly at home with a hand-held device in order to closely monitor respiratory function between hospital visits.
The study medication, all medical procedures and laboratory testing, and the visits to the study centre are free of charge. In addition the patients will receive a travel allowance to cover reasonable expenses to and from the study centre. Participants will not otherwise be compensated for this study.
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 |
|---|---|---|
| idebenone 150 mg film-coated tablets | Experimental | Drug: Idebenone 150 mg film-coated tablets900 mg idebenone/day (2 tablets to be taken 3 times a day with meals) |
| placebo | Placebo comparator | Drug: placebomatching placebo tablets |
Who may qualify
- Ages
- 10 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Male patients with a 35% ≤ FVC ≤ 80% of predicted value at Screening and at Baseline and who, in the opinion of the investigator are in the respiratory function decline phase.
- Minimum 10 years old at Screening.
- Signed and dated Informed Consent Form.
- 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.
- 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 adjustments determined by weight changes are allowed).
Key exclusion criteria (13 listed)
- Symptomatic heart failure (defined as patients with structural heart disease, dyspnea, fatigue and impaired tolerance to exercise; Stage C by the ACCF/AHA guideline or NYHA Classes III-IV) and/or symptomatic ventricular arrhythmias.
- 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 the US as part of the Expanded Access Program, or any approved corticosteroid product in trial for regimen optimization, for which the patient met the inclusion criterion 5).
- Ongoing exon-skipping therapy or read-through gene therapy for DMD; previous exon-skipping or read-through gene therapy is allowed if the stop date was more than 6 months prior to Screening.
- Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapidly progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 months prior to Screening.
- Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or presence of any other non-DMD respiratory illness that affects respiratory function.
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 |
|---|---|---|
| Idebenone 150 mg film-coated tablets | Drug | idebenone 150 mg film-coated tablets |
| placebo | Drug | placebo |
Primary outcome
Change From Baseline in Forced Vital Capacity percent predicted (FVC %p) at Week 78
Time frame: 78 weeks
Delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in FVC %p from Baseline to Week 78 using hospital based spirometry.
3 secondary outcomes
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF %p) at Week 78
Time frame: 78 weeks
Change From Baseline in Forced Vital Capacity (FVC) at Week 78
Time frame: 78 weeks
Change from Baseline in Inspiratory Flow Reserve (IFR) at Week 78
Time frame: 78 weeks
Results and safety
The study was stopped early (reason given: Interim analysis concluded to futility). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2021.
- Background: 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.
- Background: PubMed 27238057McDonald CM, Meier T, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM; DELOS Study Group. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. Neuromuscul Disord. 2016 Aug;26(8):473-80. doi: 10.1016/j.nmd.2016.05.008. Epub 2016 May 12.
- Background: PubMed 27571420Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM; DELOS Study Group. Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatr Pulmonol. 2017 Apr;52(4):508-515. doi: 10.1002/ppul.23547. Epub 2016 Aug 29.
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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.
The record lists no contact. Use the official record to reach the sponsor.
63 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Idebenone 150 mg film-coated tablets in people with Duchenne Muscular Dystrophy (DMD), run by Santhera Pharmaceuticals at 63 sites in 15 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 8 inclusion and 13 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: stopped early, as of the sponsor's last update on 3 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 63 sites in United States, Italy, Germany, United Kingdom, France and 10 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?
The study was stopped early (reason given: Interim analysis concluded to futility). Results are not available in the source record.
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 Forced Vital Capacity percent predicted (FVC %p) at Week 78 (time frame: 78 weeks). 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 NCT02814019, which also lists 3 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 3 Dec 2021.