A Study to Assess Vamorolone in Boys Ages 2 to

Related Clinical Trial
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

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Official Title

A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Brief Summary

      This Phase II study is an open-label, multiple dose study to evaluate the safety,
      tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical
      functioning vamorolone over a treatment period of 12 weeks in steroid-naïve boys ages 2 to <4
      years, and glucocorticoid-treated and currently untreated boys ages 7 to <18 years with DMD.
    

Detailed Description

      This Phase II study is an open-label, multiple dose study to evaluate the safety,
      tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical
      functioning of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over
      a treatment period of 3 months in steroid-naïve boys ages 2 to <4, and glucocorticoid-treated
      and currently untreated boys ages 7 to <18 years with DMD.

      The study is comprised of a 5-week Pretreatment Screening Period; a 1-day Pretreatment
      Baseline Period; a 3-month open-label Treatment Period (Weeks 1-12); and a 4-week open-label
      Dose-tapering Period (Weeks 13-16) for subjects who will not transition directly to further
      vamorolone or standard of care (SoC) glucocorticoid treatment at the end of the study.

      Subjects will be enrolled into the study at the Screening Visit, at the time written informed
      consent is obtained.

      Within the 2 to <4 years age group, the initial 10 eligible subjects will be assigned to the
      2.0 mg/kg/day treatment group at the Baseline Day -1 Visit. The subsequent 10 eligible
      subjects will be assigned to the 6.0 mg/kg/day treatment group at the Baseline Day -1 Visit.

      Within the 7 to <18 years age group, both corticosteroid-treated and untreated, the initial
      12 eligible subjects will be assigned to the 2.0 mg/kg/day treatment group at the Baseline
      Day -1 Visit. The subsequent 12 eligible subjects will be assigned to the 6.0 mg/kg/day
      treatment group at the Baseline Day -1 Visit.

      The first 6 subjects in each age group at 2 mg/kg will serve as the PK/safety run-in cohorts.
      PK assessments will be performed at week 2 and together with the safety assessment during the
      first 4 weeks of treatment this will be the basis to confirm whether 2 and 6 mg/kg/day will
      be used in the subsequent patients or if a dose adjustment is needed to avoid over or
      under-exposure in patients for any of the two age groups.

      Glucocorticoid-treated subjects in the 7 to <18 years age group will take their final dose of
      SoC glucocorticoid therapy for DMD on Baseline Day -1, within 24 hours prior to
      administration of the first dose of vamorolone study medication.

      All subjects in both age groups will begin their assigned vamorolone treatment on Treatment
      Period Day 1, and will continue to receive their assigned vamorolone treatment throughout the
      duration of the 3 month Treatment Period (Weeks 1-12).

      At the end of the 3-month Treatment Period (Week 12), subjects will be given the option to
      receive vamorolone in an expanded access or compassionate use program, if possible, or to
      transition to SoC treatment for DMD (may include glucocorticoids). Subjects completing
      VBP15-006 and enrolling directly into the expanded access or compassionate use program or
      transitioning directly to SoC glucocorticoid treatment will not need to taper their
      vamorolone dose prior to participation in the expanded access or compassionate use program or
      initiation of SoC glucocorticoid treatment. All subjects who will not transition directly to
      further vamorolone or SoC glucocorticoid treatment will begin a 4 week open label Dose
      tapering Period during which the dose of study medication will be progressively reduced and
      discontinued.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change in Body Mass Index (BMI) from baseline to Week 12

Secondary Outcome

 Area under the Curve infinity (AUCinf) following oral administration

Condition

Duchenne Muscular Dystrophy

Intervention

Vamorolone

Study Arms / Comparison Groups

 Treatment Group 1
Description:  Patients in Treatment Group 1 must be ages 2-<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2.

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

44

Start Date

January 2022

Completion Date

December 2022

Primary Completion Date

December 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent
             and Health Insurance Portability and Accountability Act (HIPAA) authorization, where
             applicable, prior to any study-related procedures; participants will be asked to give
             written or verbal assent according to local requirements;

          2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis
             of DMD, defined as:

               1. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
                  deficiency, and clinical picture consistent with typical DMD, OR

               2. Identifiable mutation within the DMD gene (deletion/duplication of one or more
                  exons), where reading frame can be predicted as 'out-of-frame,' and clinical
                  picture consistent with typical DMD, OR

               3. Complete dystrophin gene sequencing showing an alteration (point mutation,
                  duplication, other) that is expected to preclude production of the dystrophin
                  protein (i.e., nonsense mutation, deletion/duplication leading to a downstream
                  stop codon), with a clinical picture consistent with typical DMD;

          3. Subject is male, 2 to <4 years or 7 to <18 years of age at time of enrollment in the
             study;

          4. If 7 to <18 years of age and currently taking standard of care glucocorticoids for
             treatment of DMD, subject has been taking standard of care glucocorticoids at stable
             dose for at least 3 months prior to enrollment in the study, and will continue the
             same stable dose regimen through the date of the Baseline Day -1 Visit. [Note: Inhaled
             and/or topical glucocorticoids are permitted if last use is at least 4 weeks prior to
             enrollment or if administered at stable dose beginning at least 4 weeks prior to
             enrollment and anticipated to be used at the stable dose regimen for the duration of
             the study];

          5. If 7 to <18 years of age, and not currently glucocorticoid-treated, subject has not
             received oral glucocorticoids or other oral immunosuppressive agents for at least 3
             months prior to enrollment. [Note: Inhaled and/or topical glucocorticoids are
             permitted if last use is at least 4 weeks prior to enrollment or if administered at
             stable dose beginning at least 4 weeks prior to enrollment and anticipated to be used
             at the stable dose regimen for the duration of the study];

          6. Clinical laboratory test results are within the normal range at the Screening Visit,
             or if abnormal, are not clinically significant, in the opinion of the Investigator.
             [Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all
             must be ≤ upper limit of the normal range at the Screening Visit. An abnormal vitamin
             D level that is considered clinically significant will not exclude a subject from
             participating];

          7. Subject has evidence of chicken pox immunity as determined by:

               -  Presence of IgG antibodies to varicella, as documented by a positive test result
                  from the local laboratory from blood collected during the Screening Period; OR

               -  Documentation, provided at the Screening Visit, that the subject has had 2 doses
                  of varicella vaccine, with or without serologic evidence of immunity; the second
                  of the 2 immunizations must have been given at least 14 days prior to assignment
                  to a dose group;

          8. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled
             visits, study drug administration plan, and study procedures.

        Exclusion Criteria:

          1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus
             or immunosuppression;

          2. Subject has current or history of chronic systemic fungal or viral infections;

          3. Subject has used mineralocorticoid receptor agents, such as spironolactone,
             eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), or
             mexrenone (mexrenoate potassium) within 4 weeks prior to enrollment;

          4. Subject has a history of primary hyperaldosteronism;

          5. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac
             abnormality on investigation would not be exclusionary];

          6. If 2 to <4 years of age, subject is currently being treated or has received previous
             treatment with oral glucocorticoids or other immunosuppressive agents [Notes: Past
             transient use of oral glucocorticoids or other oral immunosuppressive agents for no
             longer than 1 month cumulative, with last use at least 3 months prior to enrollment,
             will be considered for eligibility on a case-by-case basis, unless discontinued for
             intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at
             least 4 weeks prior to enrollment or if administered at stable dose beginning at least
             4 weeks prior to enrollment and anticipated to be used at the stable dose regimen for
             the duration of the study];

          7. Subject has an allergy or hypersensitivity to the study medication or to any of its
             constituents;

          8. Subject has used idebenone within 4 weeks prior to enrollment;

          9. Subject has severe behavioral or cognitive problems that preclude participation in the
             study, in the opinion of the Investigator;

         10. Subject has previous or ongoing medical condition, medical history, physical findings
             or laboratory abnormalities that could affect safety, make it unlikely that treatment
             and follow-up will be correctly completed or impair the assessment of study results,
             in the opinion of the Investigator;

         11. Subject is taking (or has taken within 4 weeks prior to enrollment) herbal remedies
             and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10,
             creatine, etc);

         12. Subject is taking (or has taken within 3 months prior to enrollment) any medication
             indicated for DMD, including Exondys51, Exondys53, Exondys45, Viltepso and Translarna;

         13. Subject has been administered a live attenuated vaccine within 14 days prior to the
             first dose of study medication;

         14. Subject is currently taking any other investigational drug or has taken any other
             investigational drug within 3 months prior to enrollment;

         15. Subject has previously been enrolled in the VBP15-006 study or any other vamorolone
             study.
      

Gender

Male

Ages

2 Years - 17 Years

Accepts Healthy Volunteers

No

Contacts

Jean K Mah, M.D., 301-762-7980, [email protected]



Administrative Informations


NCT ID

NCT05185622

Organization ID

VBP15-006


Responsible Party

Sponsor

Study Sponsor

ReveraGen BioPharma, Inc.

Collaborators

 Santhera Pharmaceuticals

Study Sponsor

Jean K Mah, M.D., Study Chair, Alberta Children's Hospital Research Institute, University of Calgary


Verification Date

December 2021