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

November 24, 2021 updated by: Santhera Pharmaceuticals

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

161

Phase

  • Phase 3

Expanded Access

Temporarily not available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Vienna, Austria, 1100
        • Gottfried von Preyer'sches Kinderspital
  • Belgium
      • Leuven, Belgium, 3000
        • University Hospital Leuven
      • Liège, Belgium, 4000
        • CHR Citadelle
  • France
      • Lille, France, 59037
        • Service de neuropédiatrie Pôle Pédiatrie CHRU de Lille - Hôpital Jeanne de Flandre
      • Montpellier, France, 34295
        • CHRU de Montpellier - Hôpital Gui de Chauliac, Département de pédiatrie - neuropédiatrie
      • Nantes, France, 44093
        • Hôpital Hôtel Dieu, Service Explorations Fonctionnelles - Centre de Référence de Maladies Neuromusculaires rares
      • Paris, France, 75571
        • I-Motion - Plateforme d'essais cliniques pédiatriques Hôpital Armand Trousseau bâtiment Lemariey porte 20, 2ème étage
      • Toulouse, France, 31059
        • Hôpital des enfants, Pédiatrie Neurologie et infectiologie Pôle enfants
  • Germany
      • Hamburg, Germany, 20246
        • University Medical Center Hamburg - Eppendorf, Department of Paediatrics
      • München, Germany, 80337
        • Center for neuromuscular disorders, Dr. v. Haunersche Kinderklinik, Universität München
  • Italy
      • Bosisio Parini, Italy, 23842
        • Fondazione IRCCS Eugenio Medea
      • Genova, Italy, 16147
        • U.O. Malattie Neuromuscolari, Istituto Giannina Gaslini
      • Messina, Italy, 98125
        • Scientific Coordinator Nemo Sud Clinical CenterAOU Policlinico "G. Martino"
      • Milano, Italy, 20162
        • Centro Clinico NEMO (NEuroMuscular Omnicentre), Niguarda Hospital
      • Napoli, Italy, 80131
        • Servizio di Cardiomiologia e Genetica Medica AOU Università degli Studi della Campania Luigi Vanvitelli
      • Padova, Italy, 35122
        • Reparto Di Neurologia dell'Osperdale Di Padova
      • Pavia, Italy, 27100
        • Dipartimento di Clinica Neurologica e Psichiatrica dell'Età Evolutiva della Fondazione IRCCS "C. Mondino" di Pavia
      • Roma, Italy, 00168
        • U.O.C. Neuropsichiatria Infantile
  • Spain
      • Barcelona, Spain, 08950
        • Hospital Sant Joan de Deu Neuropediatra, Unidad de patologia nueromuscular, Servicio de Neurologia
      • Valencia, Spain, 46026
        • Hospital La Fe de Valencia Avinguda de Fernando Abril Martorell Servicio de Neurologia Torre D
  • Switzerland
      • Basel, Switzerland, 4301
        • Center for neuromuscular disorders, Universitäts-Kinderspital beider Basel (UKBB)
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds Teaching Hospital NHS Trust
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • London, United Kingdom, WC1 3BG
        • UCL, National Hospital for Neurology and Neurosurgery
      • Newcastle Upon Tyne, United Kingdom, NE1 4LP
        • Clinical Research Facility Level 6 Leazes Wing Royal Victoria Infirmary
      • Oswestry, United Kingdom, SY10 7AG
        • Robert Jones and Agnes Hunt Orthopaedic hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama - Birmingham, Child Health Research
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Banner University of Arizona Medical Center
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital of Los Angeles
      • Sacramento, California, United States, 95817
        • UC Davis Department of Physical Medicine and Rehabilitation
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Center for Integrative Rare Disease Research, Rare Disease Research, LLC
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa, Department of Pediatrics
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children's Hospital Boston, Harvard Medical School, Department of Neurology
    • Minnesota
      • Saint Paul, Minnesota, United States, 55101
        • Gillette Children's Specialty Healthcare
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Neurosciences Institute, Neurology - Charlotte Carolinas Healthcare System
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
      • Cleveland, Ohio, United States, 44109-1988
        • MetroHealth Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-1771
        • Children's Hospital of Philadelphia, Division of Pulmonology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

11 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
Drug: idebenone 150 mg film-coated tablets
900 mg idebenone/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From baseline until visit 4 (week 78)
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.
4 weeks after discontinuation of treatment
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.
From baseline until visit 4 (week 78)
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.
From baseline until visit 4 (week 78)
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.
4 weeks after discontinuation of treatment
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.
From baseline until visit 4 (week 78)
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.
4 weeks after discontinuation of treatment
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.
From baseline until visit 4 (week 78)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).
Time Frame: 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 Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)
Time Frame: 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)
Time Frame: 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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 4, 2018

Primary Completion (Actual)

November 25, 2020

Study Completion (Actual)

November 25, 2020

Study Registration Dates

First Submitted

May 31, 2018

First Submitted That Met QC Criteria

July 26, 2018

First Posted (Actual)

July 27, 2018

Study Record Updates

Last Update Posted (Actual)

December 3, 2021

Last Update Submitted That Met QC Criteria

November 24, 2021

Last Verified

November 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-III-012-E

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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