A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
NCT ID: NCT02814019
Last Updated: 2021-12-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE3
255 participants
INTERVENTIONAL
2016-09-30
2020-12-01
Brief Summary
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Detailed Description
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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
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meals)
Idebenone 150 mg film-coated tablets
placebo
matching placebo tablets
placebo
Interventions
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Idebenone 150 mg film-coated tablets
placebo
Eligibility Criteria
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Inclusion Criteria
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
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.
10 Years
MALE
No
Sponsors
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Santhera Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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University of Alabama
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
Banner University of Arizona Medical Center
Tucson, Arizona, United States
Childrens Hospital of Los Angeles
Los Angeles, California, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
UC Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, United States
Loma Linda University Healthcare
San Bernardino, California, United States
Shriners Hospitals for Children-Tampa
Tampa, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas
Fairway, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Children's Hospital Boston
Boston, Massachusetts, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
University of Rochester Medical Center
Rochester, New York, United States
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte, North Carolina, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
MetroHealth Medical Center
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Gottfried von Preyer'sches Kinderspital
Vienna, , Austria
University Hospital Leuven
Leuven, , Belgium
Centre de Référence Neuromusculaire, CHR Citadelle
Liège, , Belgium
Sofia Medical University
Sofia, , Bulgaria
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
Lille, , France
CHRU de Montpellier - Hôpital Gui de Chauliac
Montpellier, , France
Hôpital Hôtel Dieu
Nantes, , France
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20
Paris, , France
Hôpital des enfants
Toulouse, , France
Universitätsmedizin Berlin Campus Virchow-Klinikum
Berlin, , Germany
Uniklinik Köln
Cologne, , Germany
Universitätsklinikum Essen
Essen, , Germany
Universitätsklinik Freiburg Zentrum für Kinderheilkunde und Jugendmedizin
Freiburg im Breisgau, , Germany
Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin
Hamburg, , Germany
Universitätsklinikum Heidelberg Zentrum für Kinder- und Jugendmedizin
Heidelberg, , Germany
Zentrum für neuromuskuläre Erkrankungen
München, , Germany
Semmelweis University 2nd Department of Paediatrics
Budapest, , Hungary
Children's University Hospital
Dublin, , Ireland
Institute of Neurology at Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
Fondazione IRCCS Eugenio Medea
Bosisio Parini, , Italy
Istituto Giannina Gaslini
Genova, , Italy
Scientific Coordinator Nemo Sud Clinical Center
Messina, , Italy
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milan, , Italy
Servizio di Cardiomiologia e Genetica Medica, AOU Università degli Studi della Campania "Luigi Vanvitelli"
Napoli, , Italy
Reparto Di Neurologia dell'Osperdale Di Padova
Padua, , Italy
Dipartimento di Clinica Neurologica e Psichiatrica dell'Età Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
Pavia, , Italy
U.O.C. Neuropsichiatria Infantile
Roma, , Italy
LUMC
Leiden, , Netherlands
Radboud university medical centre
Nijmegen, , Netherlands
Hospital Sant Joan de Déu Neuropediatra
Barcelona, , Spain
Hospital Universitari Vall D' Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Hospital La Fe de Valencia
Valencia, , Spain
Sahlgrenska University Hospital
Gothenburg, , Sweden
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
Basel, , Switzerland
Leeds Teaching Hospital NHS Trust
Leeds, , United Kingdom
UCL, National Hospital for Neurology and Neurosurgery
London, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
John Walton Muscular Dystrophy Research Centre
Newcastle, , United Kingdom
Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, , United Kingdom
Oxford University hospitals NHS Foundation Trust
Oxford, , United Kingdom
Royal Hallamshire Hospital
Sheffield, , United Kingdom
Countries
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References
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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.
McDonald 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.
Buyse 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.
Other Identifiers
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SNT-III-012
Identifier Type: -
Identifier Source: org_study_id