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Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
idebenone 150 mg film-coated tablets
Sponsored by
Santhera Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring respiratory function in DMD

Eligibility Criteria

11 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  1. Completion of the SIDEROS study at Visit 8/ Week 78
  2. Signed and dated Informed Consent Form for SIDEROS-E

Exclusion Criteria:

  1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
  2. 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
  3. Use of any investigational drug other than the study medication

Sites / Locations

  • University of Alabama - Birmingham, Child Health Research
  • Banner University of Arizona Medical Center
  • Children's Hospital of Los Angeles
  • UC Davis Department of Physical Medicine and Rehabilitation
  • Center for Integrative Rare Disease Research, Rare Disease Research, LLC
  • University of Iowa, Department of Pediatrics
  • Johns Hopkins University
  • Children's Hospital Boston, Harvard Medical School, Department of Neurology
  • Gillette Children's Specialty Healthcare
  • Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
  • Cincinnati Children's Hospital
  • MetroHealth Medical Center
  • Children's Hospital of Philadelphia, Division of Pulmonology
  • Gottfried von Preyer'sches Kinderspital
  • University Hospital Leuven
  • CHR Citadelle
  • Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
  • CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
  • Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
  • I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
  • Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
  • University Medical Center Hamburg - Eppendorf, Department of Paediatrics
  • Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
  • Fondazione IRCCS Eugenio Medea
  • U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
  • Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
  • Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
  • Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
  • Reparto Di Neurologia dell'Osperdale Di Padova
  • Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
  • U.O.C. Neuropsichiatria Infantile
  • Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
  • Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
  • Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
  • Leeds Teaching Hospital NHS Trust
  • UCL, National Hospital for Neurology and Neurosurgery
  • Great Ormond Street Hospital for Children
  • Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
  • Robert Jones and Agnes Hunt Orthopaedic Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

idebenone 150 mg film-coated tablets

Arm Description

900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)

Outcomes

Primary Outcome Measures

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.
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.
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.
Number of patients with abnormal safety laboratory parameters.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal ECG.
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
May 31, 2018
Last Updated
November 24, 2021
Sponsor
Santhera Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT03603288
Brief Title
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
Acronym
SIDEROS-E
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
Study Type
Interventional

2. Study Status

Record Verification Date
November 2021
Overall Recruitment Status
Terminated
Why Stopped
Interim analysis concluded to futility of main study SIDEROS (SNT-III-012)
Study Start Date
July 4, 2018 (Actual)
Primary Completion Date
November 25, 2020 (Actual)
Study Completion Date
November 25, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Santhera Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
respiratory function in DMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open-Label Extension Study
Masking
None (Open Label)
Allocation
N/A
Enrollment
161 (Actual)

8. Arms, Groups, and Interventions

Arm Title
idebenone 150 mg film-coated tablets
Arm Type
Experimental
Arm Description
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
Intervention Type
Drug
Intervention Name(s)
idebenone 150 mg film-coated tablets
Intervention Description
900 mg idebenone/day
Primary Outcome Measure Information:
Title
Incidence and severity of adverse events, as per ICH Topic E2A
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Incidence and severity of adverse events, as per ICH Topic E2A
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
4 weeks after discontinuation of treatment
Title
Number of patients with premature discontinuations of study treatment due to adverse events.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Number of patients with abnormal safety laboratory parameters.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Number of patients with abnormal safety laboratory parameters.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
4 weeks after discontinuation of treatment
Title
Number of patients with abnormal vital signs.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Number of patients with abnormal vital signs.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
4 weeks after discontinuation of treatment
Title
Number of patients with abnormal ECG.
Description
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Secondary Outcome Measure Information:
Title
Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).
Description
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
Description
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)
Title
Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)
Description
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study.
Time Frame
From baseline until visit 4 (week 78)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completion of the SIDEROS study at Visit 8/ Week 78 Signed and dated Informed Consent Form for SIDEROS-E Exclusion Criteria: 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
Facility Information:
Facility Name
University of Alabama - Birmingham, Child Health Research
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Children's Hospital of Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
UC Davis Department of Physical Medicine and Rehabilitation
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Center for Integrative Rare Disease Research, Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
University of Iowa, Department of Pediatrics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Children's Hospital Boston, Harvard Medical School, Department of Neurology
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Gillette Children's Specialty Healthcare
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
Facility Name
Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28207
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
MetroHealth Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44109-1988
Country
United States
Facility Name
Children's Hospital of Philadelphia, Division of Pulmonology
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104-1771
Country
United States
Facility Name
Gottfried von Preyer'sches Kinderspital
City
Vienna
ZIP/Postal Code
1100
Country
Austria
Facility Name
University Hospital Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
CHR Citadelle
City
Liège
ZIP/Postal Code
4000
Country
Belgium
Facility Name
Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
City
Montpellier
ZIP/Postal Code
34295
Country
France
Facility Name
Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
City
Paris
ZIP/Postal Code
75571
Country
France
Facility Name
Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
University Medical Center Hamburg - Eppendorf, Department of Paediatrics
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
City
München
ZIP/Postal Code
80337
Country
Germany
Facility Name
Fondazione IRCCS Eugenio Medea
City
Bosisio Parini
ZIP/Postal Code
23842
Country
Italy
Facility Name
U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
City
Genova
ZIP/Postal Code
16147
Country
Italy
Facility Name
Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Reparto Di Neurologia dell'Osperdale Di Padova
City
Padova
ZIP/Postal Code
35122
Country
Italy
Facility Name
Dipartimento di Clinica Neurologica e Psichiatrica dell'Eta Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
U.O.C. Neuropsichiatria Infantile
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
City
Basel
ZIP/Postal Code
4301
Country
Switzerland
Facility Name
Leeds Teaching Hospital NHS Trust
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Facility Name
UCL, National Hospital for Neurology and Neurosurgery
City
London
ZIP/Postal Code
WC1 3BG
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
Facility Name
Robert Jones and Agnes Hunt Orthopaedic Hospital
City
Oswestry
ZIP/Postal Code
SY10 7AG
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25907158
Citation
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.
Results Reference
background
PubMed Identifier
27238057
Citation
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.
Results Reference
background
PubMed Identifier
27571420
Citation
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.
Results Reference
background
PubMed Identifier
28869486
Citation
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.
Results Reference
background
Links:
URL
https://www.clinicaltrials.gov/ct2/show/NCT02814019?term=SIDEROS&rank=1
Description
SIDEROS study clinicaltrials.gov entry

Learn more about this trial

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

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