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    EudraCT Number:2011-001266-17
    Sponsor's Protocol Code Number:DMD114349
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2012-03-08
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2011-001266-17
    A.3Full title of the trial
    An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy
    Studio di estensione in aperto sulla sicurezza, tollerabilita' ed efficacia a lungo termine di GSK2402968 in soggetti affetti da distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy
    Studio in aperto, a lungo termine, su GSK2402968 in soggetti affetti da distrofia muscolare di Duchenne
    A.4.1Sponsor's protocol code numberDMD114349
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/540/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.3.4CountryUnited Kingdom
    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 supportGlaxoSmithKline
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlaxoSmithKline
    B.5.2Functional name of contact pointClinical Trials Helpdesk
    B.5.3 Address:
    B.5.3.1Street AddressIron Bridge Road
    B.5.3.2Town/ cityUxbridge
    B.5.3.3Post codeUB11 1BU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44 20 8990 4466
    B.5.5Fax number+44 20 8990 1234
    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/3/08/599
    D.3 Description of the IMP
    D.3.1Product nameGSK2402968
    D.3.2Product code GSK2402968
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeGSK2402968
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number6
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    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
    Duchenne Muscular Dystrophy (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 14.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    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
    To evaluate the long term safety, tolerability and efficacy of subcutaneous 6mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044
    • valutare la sicurezza, l’efficacia e la tollerabilità a lungo termine di GSK2402968, per via sottocutanea, alla dose di 6 mg/kg/settimana in soggetti affetti da DMD che hanno partecipato o allo studio DMD114117 o allo studio DMD114044.
    E.2.2Secondary objectives of the trial
    •To evaluate the long-term PK of subcutaneous 6 mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044. •To evaluate the long-term impact on health-related quality of life (HRQoL) and functional outcomes of continued treatment with GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044. •To evaluate DMD disease progression and outcomes (clinical, HRQoL and functional) in subjects who discontinue active treatment during the conduct of study (natural history component). •To evaluate the long-term safety, efficacy and PK of an intermittent dosing option in those subjects unable to tolerate GSK2402968 6mg/kg/week dosing.
    •valutare la farmacocinetica (PK) a lungo termine di GSK2402968 per via sottocutanea alla dose di 6 mg/kg/settimana in soggetti affetti da DMD che hanno precedentemente partecipato o allo studio DMD114117 o allo studio DMD114044.•valutare l’impatto a lungo termine sulla qualità della vita correlata alla salute e gli esiti funzionali del trattamento continuativo con GSK2402968 in soggetti affetti da DMD che hanno precedentemente partecipato o allo studio DMD114117 o allo studio DMD114044.•valutare la progressione di malattia della DMD e gli esiti (clinici, HRQoL e funzionali) in soggetti che interrompono il trattamento attivo durante la conduzione dello studio (componente di storia naturale). •valutare nel lungo termine la sicurezza, l’efficacia e la PK di un’opzione di dosaggio intermittente nei soggetti che non riescono a tollerare la dose di 6 mg/kg/settimana di GSK2402968.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects who have successfully completed either the DMD114117 study or DMD114044 study, OR Subjects who withdrew due to safety/tolerability issues, which have since resolved and where in the opinion of the PI along with consultation of the Medical Monitor, it is considered that the benefit of further treatment with GSK2402968 outweighs the risk to the individual subject. OR Subjects who either complete or withdraw early from studies DMD114117 or DMD114044, who do not wish to continue treatment with GSK2402968 but who are willing to participate in the natural history observation arm. 2. Continued use of glucocorticoids with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticosteroids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor. 3. Willing and able to comply with all protocol requirements and procedures, 4. Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations). 5. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
    Un soggetto verrà ritenuto eleggibile per questo studio solo se soddisfa tutti i seguenti criteri: 1. -soggetti che hanno completato con successo o lo studio DMD114117 o lo studio DMD114004, OPPURE -soggetti che hanno interrotto lo studio per problemi di sicurezza/tollerabilità, che sono stati risolti e per cui, a giudizio dello sperimentatore e in consultazione con il Medical Monitor, si ritiene che il beneficio di una prosecuzione del trattamento con GSK2402968 superi i rischi per il singolo soggetto, OPPURE -soggetti che completano o che si ritirano prematuramente dagli studi DMD114117 o DMD114044, che non desiderano continuare il trattamento con GSK2402968, ma sono disposti a partecipare al gruppo di osservazione della storia naturale della malattia 2. uso continuativo di glucocorticosteroidi con l’aspettativa ragionevole che il soggetto rimanga in trattamento con steroidi per tutta la durata dello studio. Le variazioni o l’interruzione del trattamento con glucocorticosteroidi saranno a discrezione dello Sperimentatore Principale in consultazione con il soggetto/genitori e con il Medical Monitor 3. soggetti in grado e disposti ad aderire a tutti i requisiti e alle procedure del protocollo 4. soggetti in grado di dare il proprio assenso e/o consenso informato scritto firmato dal soggetto e/o dai genitori/legale rappresentante.
    E.4Principal exclusion criteria
    1. Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing at Visit 2. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor. 2. Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for GSK2402968, within 1 month of the first administration of study medication. 3. Current or anticipated participation in any other investigational clinical studies 4. History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current or history of renal or liver disease/impairment, history of inflammatory illness.
    Un soggetto non sarà considerato idoneo per l’inclusione nello studio se si verifica anche uno solo dei seguenti criteri: 1. soggetti che hanno avuto un evento indesiderato grave o che hanno soddisfatto i criteri di interruzione che restano non risolti dallo studio DMD114117 o DMD114044 che, a giudizio dello sperimentatore, potrebbero essere attribuibili al farmaco in studio e che sono in corso alla Visita 2. Una volta risolti, il soggetto potrà essere eleggibile a seguito di una consultazione dello Sperimentatore Principale con il Medical Monitor 2. uso di agenti anticoagulanti, antitrombotici o antipiastrinici, o precedente trattamento con farmaci sperimentali ad eccezione di GSK2402968 nel mese precedente la prima somministrazione di trattamento in studio. 3. attuale o prevista partecipazione ad altri studi clinici 4. anamnesi di disturbo medico significativo che potrebbe confondere l’interpretazione dei dati di efficacia o di sicurezza, ad esempio anamnesi o presenza di malattia/disfunzione renale o epatica, anamnesi di malattia infiammatoria.
    E.5 End points
    E.5.1Primary end point(s)
    • Muscle function using 6 minute walking distance (6MWD) test.
    variazione rispetto al basale della distanza percorsa in 6 minuti di cammino (6 Minute Walking Distance - 6MWD) alla settimana 104.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weeks 12,24,48,72 and Week 104 or Early withdrawal
    SETTIMANE 12,24,48,72 E SETTIMANA 104 OPPURE interruzione anticipata
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints: • Timed function tests (times and grading): • Rise from floor • 4 stair climb • 10m walk/run • Muscle strength (total score): knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors (as determined by handheld myometery) • North Star Ambulatory Assessment • Creatine kinase serum concentrations • Pulmonary function (FEV1, FVC, PCF, PF, sniff pressure test) • Pediatric Quality of Life Neuromuscular module (PedsQL)• Clinician Global Impression of Improvement (CGI-I) • Health Utilities Index (HUI) • Time to major disease milestones (e.g. loss of ambulation, night time ventilation) • Frequency of accidental falls (during 6MWD) • Functional Outcomes assessment Safety endpoints: • Incidence and severity of adverse events. • Vital signs • ECG parameters • Safety haematology and biochemistry parameters including nonstandard parameters such as coagulation parameters (in particular aPTT), serum cystatin C, Complement Factor C3, haptoglobin, fibrinogen, hsCRP, MCP-1 • Urinalysis (including quantitative protein, creatinine and ratio, urine cystatin, KIM-1 and α1-microglobulin) • Echocardiogram Pharmacokinetics: • AUC[0-24], AUC[0-7d], • Cmax • tmax • C24h, • C7d, DEXA Scan Lean body mass and bone density (exploratory endpoint)
    Test di funzionalità cronometrati: o alzarsi dal pavimento o salire 4 scalini o camminare/correre per 10m • forza muscolare: flessori del ginocchio, estensori del ginocchio, flessori del gomito, estensori del gomito, adduttori delle spalle e flessori delle anche (determinazione tramite dinamometro portatile) • valutazione Ambulatory North Star • concentrazioni plasmatiche della creatin-chinasi • funzionalità polmonare (FEV1, FVC, PCF, PF, sniff pressure test) • PedsQL • Clinician Global Impression of Improvement • Health Utilities Index (HUI) • Tempo al raggiungimento delle milestone della malattia • Frequenza di cadute accidentali (durante il 6MWD) • Valutazione degli esiti funzionali .Farmacocinetica . DEXA: massa magra e densità ossea (endpoint esplorativo
    E.5.2.1Timepoint(s) of evaluation of this end point
    Functional secondary endpoints and collection of questionnaires will be performed at the same intervals as the primary endpoint. Samples for urinalysis and safety labs will be collected Baseline and every two weeks thereafter till visit 104 or Early withdrawal. Predose PK samples will be collected monthly. Serial PK at either Week 12 or 48: prior to treatment and 1/2, 2, 4 hours and between 9-12 hours and 48-72 hours after treatment. Vital signs & ECG Weeks 4,8,16,24,32,40,48,60,72,84,96, and Week 104 or Early withdrawal. Echo & DEXA: Weeks 24, 48, 72, and Week 104 or Early withdrawal.
    End point funzionali: SETTIMANE 12,24,48,72 E SETTIMANA 104 OPPURE interruzione anticipata. Safety ogni 2 settimane. PK ogni mese.
    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 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. 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 Yes
    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 trial2
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    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
    Korea, Republic of
    Russian Federation
    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
    This study has a duration of a minimum of 2 years or until treatment is commercially available locally.
    Lo studio durerà minimo 2 anni o finché il prodotto sarà disponibile in commercio localmente
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months33
    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: 200
    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. of subjects for this age range: 180
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 20
    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. of subjects incapable of giving consent
    Assent and written informed consent must be obtained from each subject and/or subject's parent/legal guardian, in accordance with applicable local regulatory guidelines, prior to participation in the study.
    prima dell'inizio dello studio verrà raccolto il consenso informato per i genitori/tutore legale e l'assenso per i pazienti
    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 91
    F.4.2.2In the whole clinical trial 200
    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 investigator is responsible for ensuring that consideration has been given to the poststudy care of the subject's medical condition whether or not GSK is providing specific post study treatment.
    lo sperimentatore é responsabile di assicurare la continuità delle cure nel periodo post studio.
    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 Decision2012-03-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-03-03
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