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    Summary
    EudraCT Number:2017-002704-27
    Sponsor's Protocol Code Number:VBP15-004-A4
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-02-25
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2017-002704-27
    A.3Full title of the trial
    A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
    Een gerandomiseerd, dubbelblind fase IIb onderzoek met parallelgroepen, placebo- en referentiegeneesmiddelen met dubbelblinde uitbreiding ter beoordeling van de werkzaamheid en veiligheid van Vamorolone bij ambulante jongens met Duchenne spierdystrofie (DMD)

    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
    Een onderzoek om de werkzaamheid en veiligheid van Vamorolone bij jongens met Duchenne spierdystrofie (DMD) te beoordelen.
    A.3.2Name or abbreviated title of the trial where available
    VISION DMD
    A.4.1Sponsor's protocol code numberVBP15-004-A4
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03439670
    A.5.4Other Identifiers
    Name:FDA IND NumberNumber:118942
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorReveraGen BioPharma, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEuropean Commission (H2020 Grant)
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportReveraGen BioPharma, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPSR Group B.V.
    B.5.2Functional name of contact pointChrista van Kan
    B.5.3 Address:
    B.5.3.1Street AddressAntareslaan 41
    B.5.3.2Town/ cityHoofddorp
    B.5.3.3Post code2132 JE
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+3123 303 6900
    B.5.6E-mailchrista.vankan@psr-group.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/049/14
    D.3 Description of the IMP
    D.3.1Product nameVamorolone
    D.3.2Product code VBP15
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVAMOROLONE
    D.3.9.1CAS number 13209-41-1
    D.3.9.2Current sponsor codeVBP15
    D.3.9.4EV Substance CodeSUB188638
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.33
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/049/14
    D.3 Description of the IMP
    D.3.1Product nameVamorolone
    D.3.2Product code VBP15
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVAMOROLONE
    D.3.9.1CAS number 13209-41-1
    D.3.9.2Current sponsor codeVBP15
    D.3.9.4EV Substance CodeSUB188638
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePrednisone
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPREDNISONE
    D.3.9.1CAS number 53-03-2
    D.3.9.3Other descriptive namePREDNISONE
    D.3.9.4EV Substance CodeSUB10020MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne muscular dystrophy (DMD)
    Duchenne spierdystrofie
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy (DMD)
    Duchenne spierdystrofie
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and

    2. To evaluate the safety and tolerability of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg in ambulant boys ages 4 to <7 years with DMD.
    E.2.2Secondary objectives of the trial
    1. To compare the efficacy of vamorolone administered orally at daily doses of
    2.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD
    2. & 3. To compare the safety (secondary obj. 2) and the safety (secondary obj. 3) of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 24-week treatment period vs. daily prednisone
    0.75 mg/kg in ambulant boys ages 4 to <7 years with DMD;
    4. & 5. To compare the efficacy (secondary obj. 4.) and the safety (secondary obj. 5.) of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 48-wk treatm. period in ambulant boys ages 4 to <7 years with DMD vs. respectively untreated DMD historical controls and prednisone-treated DMD historical controls;
    6. To evaluate the population pharmacokinetics of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg in ambulant boys ages 4 to <7 years with DMD.
    E.2.3Trial contains a sub-study No
    E.3Principal 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 as defined as:
    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
    • 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
    • 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 ≥4 years and <7 years of age at time of enrollment in the study;
    4. Subject weighs >13.0 kg and ≤39.9 kg at the Screening Visit;
    5. Subject is able to walk independently without assistive devices;
    6. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10 seconds, as assessed at the Screening Visit;
    7. 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. [Note: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit];
    8. 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 at the Screening Visit;
    9. Subject is able to swallow tablets, as confirmed by successful test aswallowing of placebo tablets during the Screening Period; and
    10. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
    E.4Principal 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 had an acute illness within 4 weeks prior to the first dose of study medication;
    4. Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
    5. Subject has a history of primary hyperaldosteronism;
    6. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
    7. 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 indication other than DMD for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical glucocorticoids prescribed for an indication other than DMD are permitted if last use is at least 4 weeks prior to first dose of study medication or are administered at stable dose beginning at least 4 weeks prior to first dose of study medication, and are anticipated to be used at the stable dose regimen for the duration of the study];
    8. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
    9. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
    10. Subject has severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
    11. 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;
    12. Subject is taking (or has taken within 4 weeks prior to the first dose of study medication) herbal remedies and supplements which can impact muscle strength and function (e.g. Co-enzyme Q10, Creatine, Proglandine etc);
    13. Subject is taking (or has taken within 3 months prior to the first dose of study medication) any medication indicated for DMD, including Exondys51 and Translarna;
    14. Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study medication; or
    15. Subject has previously been enrolled in the study.
    Note: Any parameter/test may be repeated at the Investigator’s discretion during Screening to determine reproducibility. In addition, subjects may be rescreened if ineligible due to a transient condition which would prevent the subject from participating, such as an upper respiratory tract infection or injury, or if ineligible due to negative anti-varicella IgG antibody test result.

    E.5 End points
    E.5.1Primary end point(s)
    Primary Clinical Efficacy Endpoint
    1. Time to Stand Test (TTSTAND) velocity (rise/second): Comparison of vamorolone 6.0 mg/kg/day dose level group versus the placebo group in change from baseline to the Week 24 assessment.

    For safety endpoints and pharmacodynamic endpoints see E.5.2
    E.5.1.1Timepoint(s) of evaluation of this end point
    See E.5.1.
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints
    1. Change from baseline to Week 24 for the following comparisons:
    • TTSTAND velocity, vamorolone 2.0 mg/kg/day vs placebo
    • 6 Minute Walk Test (6MWT) meters walked, vamorolone 6.0 mg/kg/day vs. placebo
    • 6 Minute Walk Test (6MWT) meters walked, vamorolone 2.0 mg/kg/day vs. placebo
    • Time to Run/Walk (TTRW) 10 meters velocity, vamorolone 6.0 mg/kg/day vs. placebo
    • Time to Run/Walk (TTRW) 10 meters velocity, vamorolone 2.0 mg/kg/day vs. placebo
    • 6MWT meters walked, vamorolone 6.0 mg/kg/day vs. prednisone
    • 6MWT meters walked, vamorolone 2.0 mg/kg/day vs. prednisone
    2. Change from baseline to each of the scheduled study assessment time points for each treatment group up to Week 48 for:
    • Time to Stand Test (TTSTAND) velocity (rise/second);
    • Time to Climb (4 Steps) Test (TTCLIMB) velocity (tasks/second);
    • Time to Run/Walk Test (TTRW) velocity (meters/second) to complete 10 meters of a 14 meter course;
    • Total distance traveled, in meters, in completing the Six-minute Walk Test (6MWT);
    • North Star Ambulatory Assessment (NSAA);
    • Hand-held myometry (elbow flexors and knee extensors); and
    • Range of motion in the ankles (ROM)

    Safety Endpoints
    1. BMI z-score: Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points;
    2. Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) by system organ class (SOC): Overall by treatment, by treatment and relationship, and by treatment and intensity (see Section 7.5);
    3. Vital signs (sitting blood pressure, heart rate, respiratory rate, and body temperature): Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points;
    4. Body weight and height: Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points;
    5. Cushingoid features: Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points (changes from baseline will be recorded as AEs);
    6. Clinical laboratory values: Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points in:
    • Hematology and clinical chemistry
    • Lipid profile (triglycerides, total cholesterol, low density lipoprotein [LDL],
    high density lipoprotein [HDL])
    • Vitamin D level
    • Urinalysis;
    7. 12-lead electrocardiogram (ECG): Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points;
    8. 2D-echocardiogram: Change from baseline to Week 24 and Week 48;
    9. Dual-energy x-ray absorptiometry (DXA) scan: Change from baseline to Week 24 and Week 48 in spine BMD, total body BMD, spine and total body bone mass, and total body composition (lean mass, fat mass, fat-free mass, Lean Mass Index, and Fat Mass Index);
    10. Spine x-rays: Change from baseline to Week 24 assessment;
    11. Eye examination for detection of clinically significant abnormalities (cataracts and/or glaucoma) at Week 24 and Week 48 assessments compared to baseline;
    12. ACTH Stimulation Test: measure of adrenal suppression at Week 24 and Week 48. Percentage of subjects in each treatment group with cortisol levels <18 µg/dL (or 500 nM) 30 or 60 minutes after stimulation with Cosyntropin;
    13. Linear growth velocity: Change from baseline to each of the scheduled on-treatment and post-treatment assessment time points in height percentile for age.

    Pharmacodynamic Endpoints
    1. The following pharmacodynamic biomarkers are considered secondary outcome measures focusing on safety outcomes. In each case, the biomarkers studied reflect safety concerns of glucocorticoids:
    a. Adrenal suppression. First-in-morning serum cortisol levels will be measured. Cortisol measures falling below 3.6 µg/dL (or 100 nM) will be considered to be indicative of the development of adrenal suppression.
    ACTH Stimulation Test will be performed at the Screening Visit and at the Week 24 Follow-up Visit (48 ± 3 hours after the final dose of Treatment Period #1 study medication) and at the Week 48 Follow-up Visit (48 ± 3 hours after the final dose of Treatment Period #2 study medication): cortisol levels <18 µg/dL (or 500 nM) 30 or 60 minutes after stimulation with Cosyntropin (250 µg) will be considered to be indicative of adrenal suppression.
    b. Bone turnover. Measures of serum osteocalcin are reflective of bone formation, and measures of serum CTX1 are reflective of bone reabsorption. Levels of osteocalcin and CTX1 predict later clinical safety concerns of osteopenia and bone fragility.
    c. Insulin resistance. Glucocorticoids cause both acute and chronic insulin resistance, with serum elevations of both insulin and glucose. Measures of hyperinsulinemia and hyperglycemia are accepted measures of insulin resistance.
    2. Exploratory biomarkers for aspects of safety and efficacy
    E.5.2.1Timepoint(s) of evaluation of this end point
    See E.5.2.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    At week 28, subjects in prednisone or placebo arm switch to VBP15 (2 or 6 mg/kg) daily for 20 weeks
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial6
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Canada
    Czechia
    Germany
    Greece
    Israel
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 120
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 120
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    There is an information leaflet & consent form for the parents. The children will have an illustrated information letter and an assent form (Age 4: pictures only, Age 5-6: 4 pg information leaflet).
    The assent form for this age range is optional.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 72
    F.4.2.2In the whole clinical trial 120
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who complete the VBP15-004 study will be given the option of continuing vamorolone treatment in a long-term extension study under separate protocol. Subjects who will not continue vamorolone treatment in a long term extension study, including those subjects who will transition to standard of care treatment for DMD, will have their vamorolone dose tapered during a 4-week dose-tapering period, prior to discharge from the study.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-10
    P. End of Trial
    P.End of Trial StatusCompleted
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