Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Related Clinical Trial
Investigation of the Validity and Reliability of the Kinesthetic and Visual Imagery Questionnaire Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment of a Single Patient With CRD-TMH-001 E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home” Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy The Relationship Between Foot Posture and Performance and Ambulation in Patients With Duchenne Muscular Dystrophy Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy Once Weekly Infant Corticosteroid Trial for DMD Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy Bicycle Ergometer Training in Duchenne Muscular Dystrophy Telerehabilitation in Duchenne Muscular Dystrophy Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy A New Gait Assessment Scale for Duchenne Muscular Dystrophy: Test Development, Validity and Reliability Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy Tadalafil as Adjuvant Therapy for DMD A Study to Assess Vamorolone in Boys Ages 2 to A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy Extension Study of NS-089/NCNP-02 in DMD A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3) A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD) The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy CureDuchenne Link™: A Resource for Research Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) The Effect of Telerehabilitation of Patients With Duchenne Muscular Dystrophy Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) The Effects of Trunk Exercises on Upper Extremity and Respiratory Functions in DMD A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy(REACH-DMD) Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy Study of DS-5141b in Participants With Duchenne Muscular Dystrophy Open-label Extension of the HOPE-2 Trial User-centred Assistive System for Arm Functions in Neuromuscular Subjects Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders Pediatric Radio Frequency Coils Generic Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS) Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. Sodium Nitrate to Improve Blood Flow Weekend Steroids and Exercise as Therapy for DMD The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy Safety Study of BLS-M22 in Healthy Volunteers Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker. Spironolactone Versus Prednisolone in DMD Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families Treatment Effect of Tamoxifen on Patients With DMD HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20 A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Gentamicin Treatment of Muscular Dystrophy Stacking Exercises Aid the Decline in FVC and Sick Time An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort CoQ10 and Prednisone in Non-Ambulatory DMD Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy Extension Study of Drisapersen in DMD Subjects A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) PDE Inhibitors in DMD Study (Acute Dosing Study) Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping Cardiomyopathy in DMD: Lisinopril vs. Losartan Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients Ramipril Versus Carvedilol in Duchenne and Becker Patients Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy Safety and Efficacy of P-188 NF in DMD Patients Microdystrophin Gene Transfer Study in Adolescents and Children With DMD Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) Bisoprolol in DMD Early Cardiomyopathy AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD Relationship Between PFTs and Pdi in DMD Phase I/II Study of SRP-4053 in DMD Patients Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study Open Label Extension Study of HT-100 in Patients With DMD Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) Natural History of Duchenne Muscular Dystrophy A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Study of Eteplirsen in DMD Patients Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD) Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon Skipping A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON) A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy A Device for Rapid, Painless, Bedside Muscle Evaluation of Children Pentoxifylline in Duchenne Muscular Dystrophy Exploratory Study of NS-065/NCNP-01 in DMD Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD) The Burden of Access in Duchenne Muscular Dystrophy in the US A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to Aerobic Exercise in Duchenne Muscular Dystrophy Study of SRP-4045 and SRP-4053 in DMD Patients Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients The Duchenne Registry A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (-)- Epicatechin Becker Muscular Dystrophy Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study) Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography Tadalafil in Becker Muscular Dystrophy Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation Exploratory Study of NS-089/NCNP-02 in DMD A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy Safety Study of Flavocoxid in Duchenne Muscular Dystrophy Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy Rimeporide in Patients With Duchenne Muscular Dystrophy Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life. Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy Virtual Reality in Individuals With Duchenne Muscular Dystrophy Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy Upper Limb Assessment in Duchenne Muscular Dystrophy A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) Physical Activity Level of Norwegian Boys With Duchenne Muscular Dystrophy A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children Duchenne Muscular Dystrophy Heart Study Finding the Optimum Regimen for Duchenne Muscular Dystrophy Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons Duchenne Muscular Dystrophy Clinical Trial Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) A Study of Tadalafil for Duchenne Muscular Dystrophy Tamoxifen in Duchenne Muscular Dystrophy Tadalafil and Sildenafil for Duchenne Muscular Dystrophy Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy A Study of TAS-205 for Duchenne Muscular Dystrophy Research of Biomarkers in Duchenne Muscular Dystrophy Patients Digestive Events in Duchenne Muscular Dystrophy Patients An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy Strength Training in Duchenne Muscular Dystrophy Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD) Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy Prognostic Factors Affecting Duchenne Muscular Dystrophy Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance Study of DS-5141b in Patients With Duchenne Muscular Dystrophy Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) Stem Cell Therapy in Duchenne Muscular Dystrophy Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy L-citrulline and Metformin in Duchenne’s Muscular Dystrophy Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD) Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne’s Muscular Dystrophy High-dose Prednisone in Duchenne Muscular Dystrophy Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM. A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy – Phase 1-2

Brief Title

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Official Title

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy

Brief Summary

      The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary
      function, motor function, muscle strength and quality of life in patients with DMD.
      Furthermore, the safety and tolerability of idebenone was assessed.
    

Detailed Description

      This study was a Phase III, multicenter, randomized, double-blind, placebo-controlled
      efficacy and safety study. DMD patients (ambulatory and non-ambulatory) at age 10-18 years
      were enrolled at sites in Europe and North America. Study subjects were randomized in a 1:1
      ratio to receive either idebenone (900 mg/day) or placebo 3 times a day with meals for 52
      weeks. The primary endpoint was the difference between Catena®/Raxone® and placebo in the
      change from Baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p, a
      measure of respiratory muscle strength) as measured by hospital-based spirometry. PEF was
      also measured by the patient at home using the hand-held ASMA-1 device (secondary endpoint).
      Other respiratory endpoints included Forced Expiratory Volume in 1 second (as percent
      predicted, FEV1%p, an additional measure of respiratory muscle strength) and Forced Vital
      Capacity (as percent predicted, FVC%p, a measure of restrictive lung disease predictive of
      morbidity and mortality in DMD).
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52

Secondary Outcome

 Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52

Condition

Muscular Dystrophy, Duchenne

Intervention

Placebo

Study Arms / Comparison Groups

 Placebo
Description:  Placebo 900 mg/day

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

65

Start Date

July 2009

Completion Date

April 2014

Primary Completion Date

January 2014

Eligibility Criteria

        Inclusion Criteria:

          1. Patients 10 - 18 years of age at Baseline.

          2. Signed and dated informed consent.

          3. Documented diagnosis of DMD or 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 immunostain.

          4. Ability to provide reliable and reproducible repeat PEF within 15% of the first
             assessment (i.e. Baseline vs. Screening).

          5. 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.

        Exclusion Criteria:

          1. Patients dependent on assisted ventilation at Screening and/or Baseline (defined as
             non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous
             invasive ventilation).

          2. Patients with documented DMD-related hypoventilation for which assisted ventilation is
             needed according to current standard of care guidelines (e.g. FVC< 30%) or is required
             in the opinion of the Investigator.

          3. Patients with a percent predicted PEF > 80% at Baseline.

          4. Patients unable to form a mouth seal to allow precise respiratory flow measurements
             and mouth pressures.

          5. Symptomatic heart failure (high probability of death within one year of Baseline)
             and/or symptomatic ventricular arrhythmias.

          6. Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or
             SNT-II-001-E) for idebenone.

          7. Participation in any other therapeutic trial and/or intake of any investigational drug
             within 90 days prior to Baseline.

          8. Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines within 30
             days prior to Baseline.

          9. Use of coenzyme Q10 or vitamin E (if taken at a dose of 5 times above the daily
             physiological requirement) within 30 days prior to Baseline.

         10. Any previous use of idebenone.

         11. Any concomitant medication with a depressive or stimulating effect on respiration or
             the respiratory tract.

         12. Planned or expected spinal fixation surgery during the study period (as judged by the
             investigator).

         13. Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any
             other non-DMD respiratory illness that affects PEF.

         14. Chronic use of beta-2 agonists or any use of other bronchodilating medication (e.g.
             inhaled steroids, sympathomimetics, anticholinergics).

             Please note: Chronic use if defined as a daily intake for more than 14 days.

         15. Moderate or severe hepatic impairment or severe renal impairment.

         16. Prior or ongoing medical condition or laboratory abnormality that in the
             Investigator's opinion could adversely affect the safety of the subject.

             Please note: Patients who suffer from a severe, unstable condition including (but not
             limited to) cancer, auto-immune diseases, haematological 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
             Medical Monitor.

         17. Relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana
             products/smoking

         18. Known individual hypersensitivity to idebenone or to any of the ingredients/excipients
             of the study medication

         19. Systemic glucocorticoid therapy

               1. Chronic use of systemic glucocorticoid therapy for DMD related conditions within
                  12 months of Baseline (the "12 month non-use period")

               2. More than 2 rounds of acute systemic glucocorticoid burst therapy (of ≤2 week
                  duration) for non-DMD related conditions within the 12 month non-use period

               3. Use of any round of systemic glucocorticoid burst therapy of longer than 2 weeks
                  duration within the 12 month non-use period

               4. Use of systemic glucocorticoid burst therapy less than 8 weeks prior to baseline
      

Gender

Male

Ages

10 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

Prof. Gunnar Buyse, MD, PhD., , 

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT01027884

Organization ID

SNT-III-003


Responsible Party

Sponsor

Study Sponsor

Santhera Pharmaceuticals


Study Sponsor

Prof. Gunnar Buyse, MD, PhD., Principal Investigator, University Hospitals Leuven, B-3000, Belgium


Verification Date

September 2015