Duchenne Muscular DystrophyPhase 3SIDEROS-ENCT03603288
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
A Phase 3 study testing idebenone 150 mg film-coated tablets in people with Duchenne Muscular Dystrophy, run by Santhera Pharmaceuticals at 39 sites in 9 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 11 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- idebenone 150 mg film-coated tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 161 participants
- Where
- 39 sitesin 9 countries
- Study dates
- 4 Jul 2018to 25 Nov 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 11 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of the SIDEROS study at Visit 8/ Week 78
- Signed and dated Informed Consent Form for SIDEROS-E
Who cannot join
- Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
- Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study
- Use of any investigational drug other than the study medication
Trial locations and contacts
39 sites in 9 countries.
39 sites in 9 countries
United States13 sites
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
Atlanta, Georgia, 30318
Johns Hopkins University
Baltimore, Maryland, 21287
University of Alabama - Birmingham, Child Health Research
Birmingham, Alabama, 35233
Children's Hospital Boston, Harvard Medical School, Department of Neurology
Boston, Massachusetts, 02115
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
Charlotte, North Carolina, 28207
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
MetroHealth Medical Center
Cleveland, Ohio, 44109-1988
University of Iowa, Department of Pediatrics
Iowa City, Iowa, 52242
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Children's Hospital of Philadelphia, Division of Pulmonology
Philadelphia, Pennsylvania, 19104-1771
UC Davis Department of Physical Medicine and Rehabilitation
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Italy8 sites
Fondazione IRCCS Eugenio Medea
Bosisio Parini, 23842
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
Genova, 16147
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
Messina, 98125
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
Milan, 20162
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'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
Pavia, 27100
U.O.C. Neuropsichiatria Infantile
Roma, 00168
France5 sites
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
Lille, 59037
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
Montpellier, 34295
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
Nantes, 44093
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
Paris, 75571
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
Toulouse, 31059
United Kingdom5 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
Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, SY10 7AG
Belgium2 sites
University Hospital Leuven
Leuven, 3000
CHR Citadelle
Liège, 4000
Germany2 sites
University Medical Center Hamburg - Eppendorf, Department of Paediatrics
Hamburg, 20246
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
München, 80337
Spain2 sites
Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
Barcelona, 08950
Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
Valencia, 46026
Switzerland1 site
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
Basel, 4301
About this study
Official title: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study
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.
Read the detailed description
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- idebenone 150 mg film-coated tablets
- 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
900 mg idebenone/day
| 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 meal) Receives: Drug: idebenone 150 mg film-coated tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From baseline until visit 4 (week 78)
Number of patients with abnormal vital signs.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Measured: 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.
Measured: From baseline until visit 4 (week 78)
Secondary outcomes (3)
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.
Measured: From baseline until visit 4 (week 78)
Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Measured: 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.
Measured: From baseline until visit 4 (week 78)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2018
- Study start4 Jul 2018
- Primary completion25 Nov 2020
- Study completion25 Nov 2020
Why it stopped: Interim analysis concluded to futility of main study SIDEROS (SNT-III-012)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Model description
- Open-Label Extension Study
- Masking
- None (open label)
- Enrolment
- 161 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
- NCT03603288
- Sponsor's protocol ID
- SNT-III-012-E
- Acronym
- SIDEROS-E
- Status last verified
- Nov 2021
- First submitted
- 31 May 2018
- Last updated
- 3 Dec 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Expanded access
- Available
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 27571420Mayer 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.
PubMed 28869486
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 Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study
- ClinicalTrials.gov ID
- NCT03603288
- Acronym / sponsor ID
- SIDEROS-E / SNT-III-012-E
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- respiratory function in DMD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Interim analysis concluded to futility of main study SIDEROS (SNT-III-012)
- Enrolment
- 161 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 4 Jul 2018 (actual)
- Primary completion
- 25 Nov 2020 (actual)
- Study completion
- 25 Nov 2020 (actual)
- First posted
- 27 Jul 2018 (actual)
- 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 long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-Label Extension Study
| 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 meal) |
Who may qualify
- Ages
- 11 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completion of the SIDEROS study at Visit 8/ Week 78
- Signed and dated Informed Consent Form for SIDEROS-E
Key exclusion criteria (3 listed)
- Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
- Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study
- Use of any investigational drug other than the study medication
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 tablets900 mg idebenone/day | Drug | idebenone 150 mg film-coated tablets |
Primary outcome
Incidence and severity of adverse events, as per ICH Topic E2A
Time frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Incidence and severity of adverse events, as per ICH Topic E2A
Time frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with premature discontinuations of study treatment due to adverse events.
Time frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters.
Time frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters.
Time frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
Time frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
Time frame: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal ECG.
Time frame: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
3 secondary outcomes
Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).
Time frame: From baseline until visit 4 (week 78)
Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
Time frame: From baseline until visit 4 (week 78)
Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)
Time frame: From baseline until visit 4 (week 78)
Results and safety
The study was stopped early (reason given: Interim analysis concluded to futility of main study SIDEROS (SNT-III-012)). 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.
- Background: PubMed 28869486Mayer 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.
- SIDEROS study clinicaltrials.gov entryLink 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.
The record lists no contact. Use the official record to reach the sponsor.
39 sites in 9 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, run by Santhera Pharmaceuticals at 39 sites in 9 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 2 inclusion and 3 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 39 sites in United States, Italy, France, United Kingdom, Belgium and 4 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. 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 of main study SIDEROS (SNT-III-012)). 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?
Incidence and severity of adverse events, as per ICH Topic E2A (time frame: From baseline until visit 4 (week 78)). The study lists 8 primary outcomes in all. 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 NCT03603288, which also lists 4 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.