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Interim analysis concluded to futility of main study SIDEROS (SNT-III-012)
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The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
The study is an open-label, single-group, multi-center extension study in patients with DMD receiving glucocorticoid steroids who participated in the SIDEROS study and who meet all the inclusion criteria and none of the exclusion criteria for this extension study.
The study consists of 4 study visits scheduled every 6 months (Visit 1/Baseline, Visit 2/Week 26, Visit 3/ Week 52 and Visit 4/ Week 78), and a follow-up visit 4 weeks after treatment discontinuation. Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| idebenone 150 mg film-coated tablets | Experimental | 900 mg idebenone/day (2 tablets to be taken 3 times a day with meal) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| idebenone 150 mg film-coated tablets | Drug | 900 mg idebenone/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of adverse events, as per ICH Topic E2A | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Incidence and severity of adverse events, as per ICH Topic E2A | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | 4 weeks after discontinuation of treatment |
| Number of patients with premature discontinuations of study treatment due to adverse events. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Number of patients with abnormal safety laboratory parameters. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Number of patients with abnormal safety laboratory parameters. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | 4 weeks after discontinuation of treatment |
| Number of patients with abnormal vital signs. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Number of patients with abnormal vital signs. |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p). | To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama - Birmingham, Child Health Research | Birmingham | Alabama | 35233 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25907158 | Background | 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. | |
| 27238057 |
| Label | URL |
|---|---|
| SIDEROS study clinicaltrials.gov entry | View source |
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Open-Label Extension Study
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To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
| 4 weeks after discontinuation of treatment |
| Number of patients with abnormal ECG. | To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. |
| From baseline until visit 4 (week 78) |
| Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p) | To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study. | From baseline until visit 4 (week 78) |
| Banner University of Arizona Medical Center |
| Tucson |
| Arizona |
| 85724 |
| United States |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States |
| UC Davis Department of Physical Medicine and Rehabilitation | Sacramento | California | 95817 | United States |
| Center for Integrative Rare Disease Research, Rare Disease Research, LLC | Atlanta | Georgia | 30318 | United States |
| University of Iowa, Department of Pediatrics | Iowa City | Iowa | 52242 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| Children's Hospital Boston, Harvard Medical School, Department of Neurology | Boston | Massachusetts | 02115 | United States |
| Gillette Children's Specialty Healthcare | Saint Paul | Minnesota | 55101 | United States |
| Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System | Charlotte | North Carolina | 28207 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| MetroHealth Medical Center | Cleveland | Ohio | 44109-1988 | United States |
| Children's Hospital of Philadelphia, Division of Pulmonology | Philadelphia | Pennsylvania | 19104-1771 | United States |
| Gottfried von Preyer'sches Kinderspital | Vienna | 1100 | Austria |
| University Hospital Leuven | Leuven | 3000 | Belgium |
| CHR Citadelle | Liège | 4000 | Belgium |
| Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre | Lille | 59037 | France |
| CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie | Montpellier | 34295 | France |
| Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares | Nantes | 44093 | France |
| I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage | Paris | 75571 | France |
| Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants | Toulouse | 31059 | France |
| University Medical Center Hamburg - Eppendorf, Department of Paediatrics | Hamburg | 20246 | Germany |
| Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München | München | 80337 | Germany |
| Fondazione IRCCS Eugenio Medea | Bosisio Parini | 23842 | Italy |
| U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini | Genova | 16147 | Italy |
| Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino" | Messina | 98125 | Italy |
| Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital | Milan | 20162 | Italy |
| Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli | Naples | 80131 | Italy |
| Reparto Di Neurologia dell'Osperdale Di Padova | Padova | 35122 | Italy |
| Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia | Pavia | 27100 | Italy |
| U.O.C. Neuropsichiatria Infantile | Roma | 00168 | Italy |
| Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia | Barcelona | 08950 | Spain |
| Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D | Valencia | 46026 | Spain |
| Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB) | Basel | 4301 | Switzerland |
| Leeds Teaching Hospital NHS Trust | Leeds | LS1 3EX | United Kingdom |
| UCL, National Hospital for Neurology and Neurosurgery | London | WC1 3BG | United Kingdom |
| Great Ormond Street Hospital for Children | London | WC1N 3JH | United Kingdom |
| Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Robert Jones and Agnes Hunt Orthopaedic Hospital | Oswestry | SY10 7AG | United Kingdom |
| Background |
| 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. |
| 27571420 | Background | 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. |
| 28869486 | Background | Mayer OH, Leinonen M, Rummey C, Meier T, Buyse GM; DELOS Study Group. Efficacy of Idebenone to Preserve Respiratory Function above Clinically Meaningful Thresholds for Forced Vital Capacity (FVC) in Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2017;4(3):189-198. doi: 10.3233/JND-170245. |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jun 30, 2022 | Jul 26, 2022 | 13 | ||
| Mar 31, 2023 | Apr 21, 2023 | 14 | ||
| Jun 15, 2023 | Jul 5, 2023 | 15 |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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