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    Summary
    EudraCT Number:2015-003195-68
    Sponsor's Protocol Code Number:2015-003195-68
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-10-26
    Trial results View 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 ConcernedUK - MHRA
    A.2EudraCT number2015-003195-68
    A.3Full title of the trial
    Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Testosterone therapy in DMD
    A.3.2Name or abbreviated title of the trial where available
    Testosterone therapy in Duchenne Muscular Dystrophy
    A.4.1Sponsor's protocol code number2015-003195-68
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorThe Newcastle upon Tyne NHS Hospitals Foundation Trust
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDuchenne Now
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNewcastle University
    B.5.2Functional name of contact pointProf Volker Straub
    B.5.3 Address:
    B.5.3.1Street AddressInternational Centre for Life
    B.5.3.2Town/ cityNewcastle
    B.5.3.3Post codeNE1 3BZ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01912418663
    B.5.5Fax number01912418770
    B.5.6E-mailvolker.straub@ncl.ac.uk
    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 Yes
    D.2.1.1.1Trade name Sustanon 250
    D.2.1.1.2Name of the Marketing Authorisation holderAspen Pharma Trading Limited
    D.2.1.2Country which granted the Marketing AuthorisationIreland
    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 nameSustanon 250
    D.3.2Product code PRD2175434
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNtestosterone propionate
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNtestosterone phenylpropionate
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNtestosterone isocaproate
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNtestosterone decanoate
    D.3.9.4EV Substance CodeAS4
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Muscle wasting disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    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
    Evaluate patient satisfaction in response to testosterone replacement therapy in DMD patients with pubertal delay.
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of a standard incremental regimen of intramuscular testosterone therapy on pubertal staging, testicular volume and sex hormone levels
    • Evaluate the effect of testosterone replacement therapy on growth
    • Evaluate the effect of testosterone replacement therapy on muscle strength, mass and function in adolescents with DMD
    • Evaluate the effect of testosterone replacement therapy on muscle pathology as assessed by muscle MRI in DMD patients
    • Evaluate the effect of testosterone replacement therapy on bone mineral density and body composition in adolescents with DMD
    • Characterise the side-effect profile when using a standard incremental regimen of intramuscular testosterone therapy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • A molecular diagnosis of Duchenne Muscular Dystrophy.
    • Pre-pubertal males aged between 12 and 17 years of age at time of first dosing (Tanner stage 1, testicular volume <4 mls, initial testosterone level of <2.0 nmol/l)
    • Patients should receive the standard of care for DMD as recommended by the NorthStar UK and TREAT-NMD guidelines
    • Patients should be capable of sitting upright in a wheelchair for at least an hour
    • Patients should be stable from a respiratory point of view. Artificial ventilation with either Bipap or tracheostomy is not a contraindication to the study.
    • Informed consent signed by the patient (or parent/guardian if under 16 years of age and assent)
    E.4Principal exclusion criteria
    • Participants with severe learning difficulties that would preclude them from cooperating with examination.
    • Anticipated surgery during the study period.
    • Symptomatic cardiac failure.
    • Participants/families who may have emotional or psychological problems if recruited to a study
    • Hypersensitivity to the active substance or to any of the excipients, including arachis oil or derivatives. Sustanon is therefore contraindicated in patients allergic to peanuts or soya.
    • Any contra-indication to receiving an intramuscular injection
    • Any additional chronic disease that affects androgen production
    • Anti-coagulant therapy
    • None of the current treatments for DMD are exclusion criteria
    • If participation in the study is not recommended in the opinion of the investigators
    E.5 End points
    E.5.1Primary end point(s)
    The quantitative primary outcome measure will be a patient reported questionnaire validated to assess subject/parent global satisfaction with treatment, known as the Treatment Satisfaction Questionnaire for Medication (TSQM), version 1.4 The TSQM is a self-administered questionnaire that will be used at baseline, after 1 year of follow-up and at the end of the study. It is a 14-item instrument, yielding four subscale scores: global satisfaction, effectiveness, adverse events and convenience and has been validated for adults with chronic disease. The total score from this will be the primary outcome measure to assess tolerability of testosterone given by intramuscular injection for the induction of pubertal delay. Self-report questionnaires are vital as there is a documented poor correlation between the perceived health related quality of life of adolescents with DMD and the views of their parents. Data has shown that parents grossly underestimate perception of son’s happiness and of perceived support from social networks. The participants will complete questionnaires, with help from their parents if required.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The TSQM will be carried out very 6 months.
    E.5.2Secondary end point(s)
    1. Subject’s reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment
    2. Motor function and muscle strength as measured by standardised physiotherapy assessments (Northstar Ambulatory Assessment, Performance of the Upper Limb, timed tests, knee flexion and extension, elbow flexion and extension, shoulder abduction, grip strength and assessment of workable reach space using Kinect)
    3. Bone mineral adjusted density of the lumbar spine and total body (minus head) using DXA
    4. Assessment of lean versus total body mass assessed by DXA
    5. Bone turnover markers (bone-specific alkaline phosphatase, beta crosslaps, osteocalcin, P1NP, RANKL, OPG, sclerostin)
    6. Muscle volume and fat fraction as assessed by muscle MRI of upper and lower limbs 7. Pubertal staging assessed using Tanner staging and testicular volume
    8. Bone age as assessed by wrist and hand X-Ray
    9. Hormonal assessment of pubertal staging (testosterone, LH, FSH and Inhibin B)
    10. Forced vital capacity, forced expiratory volume and peak cough flow, measured by spirometry
    11.Cardiac function, assessed by ECG and echo
    E.5.2.1Timepoint(s) of evaluation of this end point
    Bone age and interviews will be carried out at beginning and end of study.
    Functional tests, hormonal assessment, bone turnover markers, respiratory function and pubertal staging will be performed every 6 months.
    DXA, cardiac function and MRI will be carried out annually
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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 years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days2
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days2
    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: 20
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    F.4.2.2In the whole clinical trial 20
    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)
    The administration of testosterone therapy is standard care, once the research has finished they will continue to receive this as per standard care.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-03-04
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