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Trial record 1 of 1 for:    02814019
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A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02814019
Recruitment Status : Recruiting
First Posted : June 27, 2016
Last Update Posted : February 5, 2020
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Brief 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

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: Idebenone 150 mg film-coated tablets Drug: placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 266 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Study Start Date : September 2016
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : August 2021

Arm Intervention/treatment
Experimental: idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meals)
Drug: Idebenone 150 mg film-coated tablets
Placebo Comparator: placebo
matching placebo tablets
Drug: placebo

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF %p) at Week 78 [ Time Frame: 78 weeks ]

    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

  2. Change From Baseline in Forced Vital Capacity (FVC) at Week 78 [ Time Frame: 78 weeks ]

    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

  3. Change from Baseline in Inspiratory Flow Reserve (IFR) at Week 78 [ Time Frame: 78 weeks ]

    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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. 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.
  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 adjustments determined by weight changes are allowed).
  6. Ability to provide reliable FVC values at Screening and Baseline, and reproducible within 15% (relative change) at Baseline compared to Screening.
  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 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.

Exclusion Criteria:

  1. 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.
  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 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).
  3. 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.
  4. 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.
  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, anticholinergics, 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 (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).
  9. 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.
  10. Relevant history of or current drug or alcohol abuse, or use of any tobacco or marijuana products/smoking.
  11. Known individual hypersensitivity to idebenone or to any of the ingredients or excipients of the study medication.
  12. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02814019

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Contact: Jodi Wolff

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United States, Alabama
University of Alabama Recruiting
Birmingham, Alabama, United States, 35233
Contact: Shelley Coskery, MSN, CPNP    205-638-2007   
Principal Investigator: Bradley Troxler, MD         
United States, Arizona
Phoenix Children's Hospital Recruiting
Phoenix, Arizona, United States, 85016
Contact: Natalia Argel, RN-BC, CPN    602-933-0343   
Principal Investigator: James Woodward, MD         
Banner University of Arizona Medical Center Recruiting
Tucson, Arizona, United States, 85724
Contact: Elizabeth Ryan, BS, RRT    520-626-3125   
Principal Investigator: Cori Daines, MD         
United States, California
Childrens Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Claudia Dozal    323-361-5825   
Principal Investigator: Leigh Maria Ramos Platt         
David Geffen School of Medicine at UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Angela Ho    310-825-3264   
Principal Investigator: Perry Shieh, MD         
UC Davis Department of Physical Medicine and Rehabilitation Recruiting
Sacramento, California, United States, 95817
Contact: Alina Nicorici    916-734-0968   
Principal Investigator: Craig McDonald, MD         
Loma Linda University Healthcare Recruiting
San Bernardino, California, United States, 92354
Contact: John Quintans    909-558-5830 ext 55827   
Principal Investigator: David Michelson         
United States, Florida
Shriners Hospitals for Children-Tampa Recruiting
Tampa, Florida, United States, 33612
Contact: Margaret Gross-King    813-972-2250 ext 7538   
Principal Investigator: Marisia Couluris, MD         
United States, Georgia
Rare Disease Research Recruiting
Atlanta, Georgia, United States, 30318
Contact: Han Phan, MD    678-883-6897   
Principal Investigator: Han Phan, MD         
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Chandra Miller, BSN, MA    319-384-3618   
Principal Investigator: Kathy Mathews, MD         
United States, Kansas
University of Kansas Recruiting
Fairway, Kansas, United States, 66103
Contact: Kiley Higgs    913-945-9922   
Principal Investigator: Jeff Statland, MD         
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21287
Contact: Robin Worrell-Thorne    443-287-6294   
Principal Investigator: Thomas Crawford, MD         
United States, Massachusetts
Children's Hospital Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Jennifer Dauriac    617-355-2752   
Principal Investigator: Basil Darras, MD         
United States, Minnesota
Gillette Children's Specialty Healthcare Recruiting
Saint Paul, Minnesota, United States, 55101
Contact: Katherine Harrison    651-578-5223   
Principal Investigator: Randal Richardson, MD         
United States, New York
University of Rochester Medical Center Recruiting
Rochester, New York, United States, 14642
Contact: Patty Smith    585-275-4339   
Principal Investigator: Emma Ciafaloni, MD         
United States, North Carolina
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System Recruiting
Charlotte, North Carolina, United States, 28207
Contact: Johnny Jones, MS    704-446-1349   
Contact: Lisa Ranzinger    704-446-0803   
Principal Investigator: Benjamin Brooks, MD         
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Dana Davis, BS    513-636-5517   
Principal Investigator: Cuixia Tian, MD         
MetroHealth Medical Center Recruiting
Cleveland, Ohio, United States, 44109-1988
Contact: Alesia Cocklin, RN    216-778-2057   
Principal Investigator: Andre Prochoroff, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104-1771
Contact: Oscar Mayer, MD    215-590-3749   
Principal Investigator: Oscar Mayer, MD         
United States, Texas
Cook Children's Medical Center Recruiting
Fort Worth, Texas, United States, 76087
Contact: Heather Urbanek, RNC III    682-885-1244   
Principal Investigator: Warren Marks, MD         
Gottfried von Preyer'sches Kinderspital Recruiting
Wien, Austria, 1100
Contact: Sophia Zacher   
Principal Investigator: Bernert Guenther, MD         
University Hospital Leuven Recruiting
Leuven, Belgium, 3000
Contact: Gunnar Buyse, MD    +32 16 343845   
Contact: Gunnar Buyse    +32 16 34 84 96      
Principal Investigator: Gunnar Buyse, MD         
Centre de Référence Neuromusculaire, CHR Citadelle Recruiting
Liège, Belgium, 4000
Contact: Laurent Servais, MD    + 32 4 321 69 82   
Principal Investigator: Laurent Servais, MD         
Sofia Medical University Not yet recruiting
Sofia, Bulgaria, 1431
Contact: Ivailo Tournev         
Principal Investigator: Ivailo Tournev         
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre Recruiting
Lille, France, 59037
Contact: Jean-Marie Cuisset, MD    +33320444057/+33320445325   
Principal Investigator: Jean-Marie Cuisset, MD         
CHRU de Montpellier - Hôpital Gui de Chauliac Recruiting
Montpellier, France, 34295
Contact: Ulrike Walther-Louvier, MD    +33 (0)4 67 33 77 37   
Principal Investigator: Ulrike Walther-Louvier, MD         
Hôpital Hôtel Dieu Recruiting
Nantes, France, 44093
Contact: Yann Pereon    +33 (0)2 40 08 37 04   
Principal Investigator: Yann Pereon         
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20 Recruiting
Paris, France
Contact: Teresa Gidaro    +33 (0)142165885   
Principal Investigator: Teresa Gidaro, MD         
Hôpital des enfants Recruiting
Toulouse, France, 31059
Contact: Claude Cancès, MD    +33 534558728   
Principal Investigator: Claude Cancès, MD         
Universitätsmedizin Berlin Campus Virchow-Klinikum Active, not recruiting
Berlin, Germany, 13353
Universitätsklinikum Essen Recruiting
Essen, Germany, 45147
Contact: Ulrike Schara    +49 201 723 4080   
Principal Investigator: Ulrike Schara         
Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin Active, not recruiting
Freiburg, Germany
Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin Recruiting
Hamburg, Germany, 20246
Contact: Jessika Johannsen    + 49 40 7410 20400   
Principal Investigator: Jessika Johannsen         
Universitätsklinikum Heidelberg Zentrum für Kinder- und Jugendmedizin Not yet recruiting
Heidelberg, Germany
Contact: Thomas Opladen, MD         
Principal Investigator: Thomas Opladen, MD         
Uniklinik Köln Recruiting
Köln, Germany, 50937
Principal Investigator: Sebahattin Cirak, MD         
Zentrum für neuromuskuläre Erkrankungen Recruiting
München, Germany
Contact: Wolfgang Müller-Felber, MD    0049(0)89/4400-55112   
Contact: Astrid Blaschek   
Principal Investigator: Wolfgang Müller-Felber, MD         
Semmelweis University 2nd Department of Paediatrics Not yet recruiting
Budapest, Hungary
Contact: Agnes Herczegfalvi, MD         
Principal Investigator: Agnes Herczegfalvi         
Children's University Hospital Recruiting
Dublin, Ireland, 1
Contact: Declan O'Rourke    +353 (1) 8784722   
Principal Investigator: Declan O'Rourke         
Institute of Neurology at Schneider Children's Medical Center of Israel Recruiting
Petah Tiqva, Israel, 4920235
Contact: Yoram Nevo, MD    972-3-9253613   
Principal Investigator: Yoram Nevo, MD         
Fondazione IRCCS Eugenio Medea Recruiting
Bosisio Parini, Italy, 23842
Contact: Sandra Gandossini    39 03 1877870    sandra.gandossini@BP.LNF.IT   
Principal Investigator: Annamaria Russo, MD         
Istituto Giannina Gaslini Recruiting
Genova, Italy, 16147
Contact: Claudio Bruno, MD    010-5636.2756   
Contact: Marina Pedemonte    010-5636.2765   
Principal Investigator: Claudio Bruno, MD         
Scientific Coordinator Nemo Sud Clinical Center Recruiting
Messina, Italy, 98125
Contact: Giuseppe Vita    +39 0902212789   
Principal Investigator: Giuseppe Vita, MD         
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital Recruiting
Milano, Italy
Contact: Valeria Sansone    +39 02914337   
Contact: Valeria Sansone    +39 02914331      
Principal Investigator: Valeria Sansone, MD         
Servizio di Cardiomiologia e Genetica Medica, AOU Università degli Studi della Campania "Luigi Vanvitelli" Recruiting
Napoli, Italy, 80131
Contact: Luisa Politano, MD    0039 0815665300   
Principal Investigator: Luisa Politano, MD         
Reparto Di Neurologia dell'Osperdale Di Padova Recruiting
Padova, Italy, 35122
Contact: Elena Pegoraro    0039 3479031890   
Contact: Luca Bello    0039 3396059583   
Principal Investigator: Elena Pegoraro         
Dipartimento di Clinica Neurologica e Psichiatrica dell'Età Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia Recruiting
Pavia, Italy, 27100
Contact: Angela Berardinelli    0039 0382380288   
Principal Investigator: Angela Berardinelli         
U.O.C. Neuropsichiatria Infantile Recruiting
Roma, Italy, 00168
Contact: Eugenio Mercuri, MD    0039 0671584657   
Principal Investigator: Eugenio Mercuri, MD         
LUMC Recruiting
Leiden, Netherlands
Contact: Marjolein van Heur    +31 71-5261089   
Principal Investigator: Chiara Straathof, MD         
Radboud university medical centre Recruiting
Nijmegen, Netherlands, 6500
Contact: Imelda J M De Groot, MD    +31-24-3614892   
Principal Investigator: Imelda J M De Groot, MD         
Hospital Sant Joan de Déu Neuropediatra Recruiting
Barcelona, Spain, 08950
Contact: Andres Nascimento    +34 93 2804000   
Principal Investigator: Andres Nascimento, MD         
Hospital Universitari Vall D' Hebron Recruiting
Barcelona, Spain, 8950
Contact: Penelope Romero   
Principal Investigator: Francina Munell, MD         
Hospital Universitario La Paz Recruiting
Madrid, Spain, 28046
Contact: Gloria Lopez Sobrino    +34 91 7277388   
Principal Investigator: Samuel Pascual         
Hospital Universitario Virgen del Rocio Recruiting
Sevilla, Spain, 41013
Contact: Marcos Madruga    +34955012431 / 639310203   
Principal Investigator: Marcos Madruga         
Hospital La Fe de Valencia Recruiting
Valencia, Spain, 106 46026
Contact: Pilar Marti   
Principal Investigator: Juan Jesus Vilchez, MD         
Sahlgrenska University Hospital Recruiting
Gothenburg, Sweden
Contact: Emma Back   
Principal Investigator: Mar Tulinius, MD         
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB) Recruiting
Basel, Switzerland, 4301
Contact: Anrea Klein    +41 61 704 29 38   
Principal Investigator: Andrea Klein, MD         
United Kingdom
Leeds Teaching Hospital NHS Trust Recruiting
Leeds, United Kingdom, LS1 3EX
Contact: Anne-Marie Childs    +44 113 3923113   
Principal Investigator: Anne-Marie Childs         
UCL, National Hospital for Neurology and Neurosurgery Recruiting
London, United Kingdom, WC1 3BG
Contact: Rosaline Quinlivan    +44203 448 8132   
Principal Investigator: Rosaline Quinlivan         
Great Ormond Street Hospital for Children Recruiting
London, United Kingdom, WC1N 3JH
Contact: Pinki Munot    +44 20 7405 9200   
Principal Investigator: Pinki Munot         
John Walton Muscular Dystrophy Research Centre Recruiting
Newcastle, United Kingdom
Contact: Michela Guglieri    +441912418757/+441912418649   
Principal Investigator: Michela Guglieri, MD         
Robert Jones and Agnes Hunt Orthopaedic Hospital Recruiting
Oswestry, United Kingdom, SY10 7AG
Contact: Tracey Willis    +44 1691 404047   
Principal Investigator: Tracey Willis         
Oxford University hospitals NHS Foundation Trust Recruiting
Oxford, United Kingdom, OX39DU
Contact: Sithara Ramdas   
Principal Investigator: Sithara Ramdas         
Royal Hallamshire Hospital Recruiting
Sheffield, United Kingdom, S10 2JF
Contact: Channa Hewamadduma   
Principal Investigator: Channa Hewamadduma         
Sponsors and Collaborators
Santhera Pharmaceuticals
Additional Information:
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Responsible Party: Santhera Pharmaceuticals Identifier: NCT02814019    
Other Study ID Numbers: SNT-III-012
First Posted: June 27, 2016    Key Record Dates
Last Update Posted: February 5, 2020
Last Verified: February 2020
Keywords provided by Santhera Pharmaceuticals:
respiratory function in DMD
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs