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    Summary
    EudraCT Number:2017-000397-10
    Sponsor's Protocol Code Number:DSC/14/2357/51
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-03-15
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-000397-10
    A.3Full title of the trial
    Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study in which all the patients take the same investigational drug with the
    aim of evaluate the long-term safety, tolerability, and efficacy of
    GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy
    who have been already treated in one of the GIVINOSTAT studies in the
    past
    A.3.2Name or abbreviated title of the trial where available
    Givinostat DMD long term study
    A.4.1Sponsor's protocol code numberDSC/14/2357/51
    A.5.4Other Identifiers
    Name:INDNumber:126598
    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 SponsorITALFARMACO S.p.A.
    B.1.3.4CountryItaly
    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 supportITALFARMACO S.p.A.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationITALFARMACO S.p.A.
    B.5.2Functional name of contact pointClinical Research and Development
    B.5.3 Address:
    B.5.3.1Street AddressVia dei Lavoratori, 54
    B.5.3.2Town/ cityCinisello Balsamo (MI)
    B.5.3.3Post code20092
    B.5.3.4CountryItaly
    B.5.4Telephone number+39 02 64431
    B.5.5Fax number+39 02 64433554
    B.5.6E-mailp.bettica@italfarmaco.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 numberEU/3/12/1009
    D.3 Description of the IMP
    D.3.1Product nameGIVINOSTAT (hyrochloride monohydrate)
    D.3.2Product code ITF2357
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNGivinostat (hydrochloride monohydrate)
    D.3.9.1CAS number 732302-99-7
    D.3.9.2Current sponsor code ITF2357
    D.3.9.3Other descriptive nameGIVINOSTAT
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    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)
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy (DMD) is A neuromuscular disease characterized by rapidly progressive muscle
    weakness and wasting

    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
    To assess the long term safety and tolerability of GIVINOSTAT oral suspension in patients with Duchenne Muscular Dystrophy (DMD) following core protocols program.
    E.2.2Secondary objectives of the trial
    To evaluate the effects of long-term administration of GIVINOSTAT on muscular function and strength;
    To evaluate the effects of long-term administration of GIVINOSTAT on respiratory function;
    To evaluate the impact on daily activities and quality of life following long-term administration of GIVINOSTAT.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To clarify A.15 Answer:
    Male subjects affected by Duchenne Muscular Dystrophy previously enrolled in one of the other DMD GIVINOSTAT studies (e.g DSC/14/2357/43, DSC/14/2357/48 and DSC/14/2357/50), aged ≥7 years and 6 months

    INCLUSION CRITERIA:
    1. Must have participated in one of the previous studies with
    GIVINOSTAT in DMD and have attended the End of Study Visit;
    2.Aged ≥7 years and 6 months old
    3. Are able to give informed assent and/or consent in writing signed by
    the subject and/or parent/legal guardian (according to local
    regulations);
    4. Subjects must be willing to use adequate contraception:
    Contraceptive methods must since the previous GIVINOSTAT study
    through 3 months after the last dose of study drug, and include the
    following:
    True abstinence (absence of any sexual intercourse), when in line with
    the preferred and usual lifestyle of the subject. Periodic abstinence (e.g.
    calendar, ovulation, symptothermal, postovulation methods) and
    withdrawal are not acceptable methods of contraception.
    oCondom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, transdermal,
    injectable or implanted steroid-based contraceptive, or a diaphragm or a
    barrier method of contraception in conjunction with spermicidal jelly
    such as for example cervical cap with spermicide jelly.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will NOT be eligible for enrolment into the trial:

    Subjects presenting with any of the following criteria will not be
    included in the study:
    1. Use of any pharmacologic treatment, other than corticosteroids, that
    might have had an effect on muscle strength or function within 3 months
    prior to be enrolled in this study (e.g., growth hormone); Vitamin D,
    calcium, and any other supplements will be allowed;
    2. Use of any current investigational drug other than Givinostat;
    3. Have presence of other clinically significant disease, which, in the
    Investigator's opinion, could adversely affect the safety of the subject,
    making it unlikely that the course of treatment or follow-up would be
    completed, or could impair the assessment of study results;
    4. Have a diagnosis of other uncontrolled neurological diseases or
    presence of relevant uncontrolled somatic disorders that are not related
    to DMD;
    5. Have platelets count, White Blood Cell and Hemoglobin at screening <
    Lower Limit of Normal (LLN) (for abnormal screening laboratory test
    results (<LLN), the platelets count, White Blood Cell and Hemoglobin will
    be repeated once; if the repeat test result is still <LLN, then
    exclusionary);
    6.Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition at
    screening visit* (for abnormal screening laboratory test results (>ULN),
    the triglycerides will be repeated once; if the repeat test result is still
    >ULN, then exclusionary);
    7.Have inadequate renal function, as defined by serum Cystatin C >2 x
    the upper limit of normal (ULN) at screening visit*. If the value is >2 x
    ULN, the serum Cystatin C will be repeated once; if the repeated test
    result is still >2 x ULN, the subject should be excluded);
    8. Have heart failure (New York Heart Association Class III or IV)
    9. Have a current liver disease or impairment, including but not limited
    to an elevated total bilirubin(i.e. > 1.5 x ULN), unless secondary to
    Gilbert disease or pattern consistent with Gilbert's;
    10. Have a baseline QTcF >450 msec, (as the mean of 3 consecutive
    readings 5 minutes apart) or history of additional risk factors for
    torsades de pointes (e.g., heart failure, hypokalemia, or family history of
    long QT syndrome);
    11. Have a psychiatric illness/social situations rendering the potential
    subject unable to understand and comply with the muscle function tests
    and/or with the study protocol procedures.
    12. Have any hypersensitivity to the components of study medication;
    13. Have a sorbitol intolerance or sorbitol malabsorption, or have the
    hereditary form of fructose intolerance.
    E.5 End points
    E.5.1Primary end point(s)
    Type, incidence, and severity of treatment related/not related AEs and
    SAEs

    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline through week 48 and then yearly till the end of the study
    E.5.2Secondary end point(s)
    Secondary endpoints

    For ambulant patients:
    • Change from baseline in physical function as measured by 6MWT,
    NSAA, Time function tests (e.g. time to rise from floor, time to climb 4-
    stairs, time to 10m walk)(week 48 and then yearly till the end of the
    study)
    • Change from baseline in muscle strength (e.g. knee extension and
    elbow flexion) as measured by HHM (week 48 and then yearly till the
    end of the study)
    • Time to major disease milestones (e.g. time to loss of ambulation).
    For non-ambulant patients:
    • Change in physical function from baseline in the Egen Klassifikation
    (EK) score (week 48 and then yearly till the end of the study)
    • Change in patient and/or parent/caregiver reports of activities of daily
    living as measured by Barthel Index (week 48 and then yearly till the
    end of the study
    • Change in upper limbs muscle strength (week 48 and then yearly till
    the end of the study) evaluated by handheld myometry (HHM)
    For all patients:
    • Change from baseline in physical function as measured by the
    Performance of Upper Limb (PUL) and MFM (week 48 and then yearly till
    the end of the study)
    • Change from baseline in respiratory function (week 48 and then yearly
    till the end of the study) (e.g. FVC, FEV1, PEF)
    • Change in patient and/or parent/caregiver reports of quality of life as
    measured by PedsQL (week 48 and then yearly till the end of the study),
    • Time to major disease milestones (e.g. time to respiratory support
    needed during the day, time to scoliosis surgery, time to death).
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 48 and then yearly till the end of the study, summary description can be found in study synopsis of protocol

    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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.8.1.7.1Other trial design description
    This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Belgium
    Canada
    France
    Germany
    Italy
    Netherlands
    Spain
    United Kingdom
    United States
    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. The end of the trial is defined as the date of the last visit of the last subject undergoing the trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years6
    E.8.9.2In all countries concerned by the trial months5
    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: 175
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 138
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 37
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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
    those aged ≥7 years and 6 months - age range indicates the inclusion of those too young to give written informed consent- parental permission
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state48
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 153
    F.4.2.2In the whole clinical trial 185
    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 treatment may continue up to the Marketing Authorization of
    GIVINOSTAT or different decision taken by the Sponsor and /or the
    Competent Authorities, based on safety and/or efficacy reasons, for all
    the DMD population
    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 Decision2019-08-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-20
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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