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    Summary
    EudraCT Number:2017-002704-27
    Sponsor's Protocol Code Number:VBP15-004-A2
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-06-10
    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 ConcernedGreece - EOF
    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)
    Μία Τυχαιοποιημένη, Διπλά-Τυφλή, ελεγχόμενη με Εικονικό και Δραστικό Φάρμακο Μελέτη Παράλληλων Ομάδων Φάσης IIb με Διπλά-Τυφλή Eπέκταση προκειμένου να Αξιολογηθεί η Αποτελεσματικότητα και η Ασφάλεια της Χορήγησης Valmorone σε Περιπατητικά Αγόρια με Μυϊκή Δυστροφία Duchenne (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)
    Μια μελέτη για την αξιολόγηση της αποτελεσματικότητας και της ασφάλειας του vamorolone σε αγόρια με μυϊκή δυστροφία Duchenne (DMD)
    A.3.2Name or abbreviated title of the trial where available
    VISION DMD
    Μελέτη VISION DMD
    A.4.1Sponsor's protocol code numberVBP15-004-A2
    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 PlanP/084/2018
    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 organisationReveraGen BioPharma, Inc.
    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)
    Μυϊκή δυστροφία Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy (DMD)
    Μυϊκή δυστροφία Duchenne (DMD)
    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 2.0 mg/kg and 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. & 2. To compare the safety (secondary obj. 1.) and efficacy
    (secondary obj. 2.) of vamorolone administered orally at daily doses of
    2.0 mg/kg and 6.0 mg/kg over a 24-wk treatment period vs. daily
    prednisone 0.75 mg/kg in ambulant boys ages 4 to <7 years with DMD;
    3. To compare the efficacy of vamorolone administered orally at daily
    doses of 2.0 mg/kg vs. vamorolone administered orally at daily doses of
    6.0 mg/kg over a 24-wk treatm. period 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-offrame',
    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 1 month cumulative, with last use at least 3 months prior to first
    dose of study medication, will be considered for eligibility on a case-bycase
    basis, unless discontinued for intolerence. 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 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 has been administered a live attenuated vaccine within 14
    days prior to the first dose of study medication;
    15. 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;
    16.Subject has a sibling who is currently enrolled in any vamorolone
    study or Expanded Access Program, or who intends to enroll in any
    vamorolone study or Expanded Access Program during the subject's
    participation in the VBP15-004 study; or
    17. 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 antivaricella
    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
    each vamorolone 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 each of the scheduled study assessment time points for each treatment group up to Week 48, with comparison of each
    vamorolone dose level group versus the placebo group at each of the
    scheduled study assessment time points up to and including Week 24
    for:
    • Time to Stand Test (TTSTAND) velocity (rise/second) (other than
    Week 24);
    • 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).
    2. Change from baseline with comparison of each vamorolone dose level
    group versus the prednisone group at each of the scheduled study
    assessment time points up to and including week 24 for:
    - total distance traveled, in meters, in completing the Six-minute Walk
    test (6MWT).

    Safety Endpoints
    1. BMI z-score: Comparison of each vamorolone dose level group with
    the prednisone group in 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;
    3. Vital signs (sitting blood pressure, heart rate, respiratory rate, and
    body temperature): Change from baseline to each of the scheduled ontreatment
    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, spine BMD Z-score, 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 insufficiency at Week 24
    and Week 48. Adrenal suppression is likely if cortisol levels <18 μg/dL
    (or 500 nM) 30 or 60 minutes after stimulation with Cosyntropin.

    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
    insufficiency.
    b. Bone turnover. Measures of serum osteocalcin are reflective of bone
    formation, and measures of serum CTX1 are reflective of bone
    reabsorption. Ratios 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.
    d. Immune suppression. Glucocorticoids can cause immunosuppression.
    Measure of differential lymphocyte percentage can be a biomarker for
    immune suppression.
    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 concerned1
    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
    Czech Republic
    Germany
    Greece
    Israel
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    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 months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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.
    Υπάρχει ενημερωτικό φυλλάδιο και συγκατάθεση για γονείς. Τα παιδία έχουν ενημερωτικό φυλλάδιο με εικόνες και φόρμα συναίνεσης (Ηλικ: 4 μόνο εικόνες, Ηλικ: 5-6: φυλλάδιο ενημέρωσης με 4 εικ). Η φόρμα συναίνεσης για τις ηλικίες αυτές είναι προαιρετική
    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.
    Στους ασθενείς που θα ολοκληρώσουν τη μελέτη VBP15-004 θα τους δοθεί η επιλογή να συνεχίσουν τη θεραπεία με το vamorolone σε μία μακροχρόνια μελέτη επέκτασης υπό ξεχωριστό πρωτόκολλο. Οι ασθενείς που δεν θα συνεχίσουν τη θεραπεία με το vamorolone στη μακροχρόνια μελέτη επέκτασης, συμπεριλαμβανομένων εκείνων που θα μεταβούν σε θεραπεία πρότυπης φροντίδας της DMD, θα έχουν σταδιακή μείωση της δόσης του vamorolone σε μία περιόδο 4-εβδομάδων σταδιακής μείωσης δόσης πριν την έξοδό τους από τη μελέτη
    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 Decision2019-08-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-26
    P. End of Trial
    P.End of Trial StatusOngoing
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