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    Summary
    EudraCT Number:2018-004383-65
    Sponsor's Protocol Code Number:SRK-015-002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-25
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-004383-65
    A.3Full title of the trial
    Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy
    Studio di fase 2 con trattamento attivo per valutare l’efficacia e la sicurezza di SRK-015 in pazienti con Atrofia Muscolare Spinale a Insorgenza Tardiva (TOPAZ)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy
    Studio clinico per un nuovo possibile trattamento in pazienti con Atrofia Muscolare Spinale tipo 2 o 3
    A.3.2Name or abbreviated title of the trial where available
    The TOPAZ study
    Studio TOPAZ
    A.4.1Sponsor's protocol code numberSRK-015-002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03921528
    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 SponsorScholar Rock, 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 supportScholar Rock, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace UK
    B.5.2Functional name of contact pointBente Kristiansen
    B.5.3 Address:
    B.5.3.1Street AddressWallace House, 17-21 Maxwell Place
    B.5.3.2Town/ cityStirling
    B.5.3.3Post codeFK81JU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number004540465215
    B.5.6E-mailRegSubmissions@Medpace.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/3/18/2115
    D.3 Description of the IMP
    D.3.1Product nameSRK-015
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSRK-015
    D.3.9.2Current sponsor codeSRK-015
    D.3.9.4EV Substance CodeSUB193232
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    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 numberEU/3/18/2115
    D.3 Description of the IMP
    D.3.1Product nameSRK-015
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSRK-015
    D.3.9.2Current sponsor codeSRK-015
    D.3.9.4EV Substance CodeSUB193232
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    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
    Later Onset Spinal Muscular Atrophy (SMA)
    atrofia muscolare spinale a insorgenza tardiva (SMA)
    E.1.1.1Medical condition in easily understood language
    Later Onset Spinal Muscular Atrophy (SMA) is an inherited disease of the
    motor nerves (the nerves that send signals from your brain to the
    muscles) causing muscle weakness.
    atrofia muscolare spinale a insorgenza tardiva (SMA) è
    è una malattia ereditaria dei
    nervi motori (i nervi che inviano segnali dal cervello ai
    muscoli) che causa debolezza muscolare.
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079415
    E.1.2Term Spinal muscular atrophy type III
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079415
    E.1.2Term Spinal muscular atrophy type III
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079415
    E.1.2Term Spinal muscular atrophy type III
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079416
    E.1.2Term Spinal muscular atrophy type II
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079416
    E.1.2Term Spinal muscular atrophy type II
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10079416
    E.1.2Term Spinal muscular atrophy type II
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    For both the Treatment Period and Extension Period:
    To assess safety and tolerability of SRK-015 in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)
    To assess the efficacy of SRK-015 by assessing changes in motor function outcome measures
    Sia per il periodo di trattamento che per il periodo di estensione:
    Valutare la sicurezza e la tollerabilità di SRK-015 in pazienti con atrofia muscolare spinale (SMA) a insorgenza tardiva (ad es. tipo 2 e tipo 3)
    Valutare l’efficacia di SRK-015 valutando le variazioni nelle misure di esito della funzionalità motoria
    E.2.2Secondary objectives of the trial
    For both the Treatment Period and Extension Period:
    To characterize the pharmacokinetics (PK) of SRK-015
    To evaluate the pharmacodynamic (PD) effects of SRK-015
    To evaluate time to therapeutic effect between low and high dose SRK015 in a predefined cohort (Cohort 3)
    To evaluate the immunogenicity of SRK-015
    To evaluate the effect of SRK-015 on quality of life assessments
    Sia per il periodo di trattamento che per il periodo di estensione:
    Caratterizzare la farmacocinetica (PK) di SRK-015
    Valutare gli effetti di farmacodinamica (PD) di SRK-015
    Valutare il tempo all’effetto terapeutico tra la dose bassa e alta di SRK-015 in una coorte predefinita (Coorte 3)
    Valutare l’immunogenicità di SRK-015
    Valutare l’effetto di SRK-015 sulla qualità della vita
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Age5through21 years old at the time of screening for Cohorts1 and
    2;Age=2 years old at the time of screening for Cohort3
    2.Estimated life expectancy>2years from screening
    3.ICF signed by the patient if the patient is
    legally an adult. If the patient is legally a minor, informed consent
    document signed by the patient's parent or legal guardian and patient's
    oral or written assent obtained, if applicable and in accordance with the
    regulatory and legal requirements of the participating location
    4.Documented diagnosis of 5q SMA
    5.Diagnosed as later onset (e.g.Type2or Type 3)SMA prior to
    receiving any treatment with therapy approved for SMA
    6.Non ambulatory patients must be able to sit independently (sits up
    straight with head erect for at least 10sec; does not use arms or
    hands to balance body or support position) per World Health
    Organization(WHO)motor milestones definition at screening.Patients
    who never had the ability to walk independently will be classified as
    Type2.Patients who previously had the ability to walk unaided will be
    classified as Type3.
    7.Ambulatory patients must have the ability to independently ambulate
    without aids or orthotics over 10meters in 30sec or less at
    screening
    8.For Cohort1, Revised Hammersmith Scale(RHS)score no greater
    than 63 at screening
    9.For Cohorts2and3, Hammersmith Functional Motor Scale Expanded
    (HFMSE) score no less than10 at screening
    10.Receiving the same background SMA therapy (e.g.on an approved
    survival motor neuron(SMN)upregulator therapy such as nusinersen, or
    not on any SMA therapy)for at least 6months prior to screening and
    anticipated to remain on that therapy throughout the duration of the
    study
    10a.If receiving the SMN upregulator therapy nusinersen, must have
    completed the loading regimen and initiated maintenance dosing(i.e.
    completed at least one maintenance dose)with at least 4weeks after
    the first maintenance dose having elapsed prior to screening
    11.Nutritional status stable over the past 6months and anticipated to
    be stable throughout the duration of the study
    12Have no physical limitations that would prevent the patient from
    undergoing motor function outcome measures throughout the duration
    of the study
    13.Able to receive study drug infusions and provide blood samples
    through the use of a peripheral intravenous(IV)or a long-term IV
    access device that the patient has placed for reasons independent from
    the study(i.e.for background medical care and not for the purpose of
    receiving SRK-015 in the study),throughout the duration of the study
    14.Able to adhere to the requirements of the protocol, including travel
    to the study center and completing all study procedures and study visits
    15.For patients who are expected to have reached reproductive maturity
    by the end of the study, adhere to study specific contraception
    requirements
    15a.Females of childbearing potential must have a negative pregnancy
    test at screening and agree to employ highly effective contraceptive
    measures(failure rate of 1% or less per year when used consistently
    and correctly)for the duration of the study and for 18 weeks following
    the last dose of study drug. Effective contraception methods are
    restricted t to combined (estrogen and progestogen containing) hormonal
    contraception associated with inhibition of ovulation (oral,intravaginal,
    transdermal),progestogen-only hormonal contraception associated with
    inhibition of ovulation(oral,injectable,implantable),intrauterine device
    (IUD),intrauterine hormone-releasing system(IUS),bilateral tubal
    occlusion,vasectomized partner,or sexual abstinence.In the context of
    this guidance, sexual abstinence is considered a highly effective method
    only if defined as refraining from heterosexual intercourse during the
    entire period of risk associated with the study treatments.The reliability
    of sexual abstinence needs to be evaluated in relation to to the duration of the clinical trial and the preferred and usual lifestyle of the patient.
    Please refer to protocol
    1.Età compresa tra 5 e 21 anni al momento dello screening per le coorti 1e2; età =2 anni al momento dello screening per la coorte3
    2.Aspettativa di vita stimata>2anni dallo screening
    3.Documento di consenso informato firmato dal paziente, se legalmente adulto.Se il paziente è legalmente minorenne, acquisire il documento di consenso informato firmato dal genitore o dal tutore legale del paziente, e l’assenso orale o scritto del paziente, se applicabile, in conformità con i requisiti normativi e legali della sede partecipante
    4.Diagnosi documentata di SMA 5q
    5.Diagnosi di SMA a insorgenza tardiva (ad es. tipo 2 o tipo 3) prima della somministrazione di qualsiasi terapia per SMA approvata
    6.I pazienti non deambulanti devono essere in grado di sedersi in modo indipendente(seduti dritti con la testa eretta per almeno 10 secondi, senza l’uso di braccia o mani per bilanciare il corpo o la posizione di supporto)secondo la definizione delle tappe motorie dell’Organizzazione Mondiale della Sanità (OMS) allo screening. I pazienti che non hanno mai avuto la capacità di camminare autonomamente rientreranno nel tipo 2.I pazienti che in precedenza avevano la capacità di camminare autonomamente rientreranno nel tipo3
    7.I pazienti deambulanti devono avere la capacità di deambulare in maniera indipendente, senza aiuti o plantari,percorrendo 10metri in 30secondi o meno allo screening
    8.Per la coorte1, punteggio sulla scala Hammersmith revisionata(RHS) non superiore a 63 allo screening
    9.Per le coorti 2 e 3, punteggio sulla scala motoria funzionale Hammersmith estesa (HFMSE) non inferiore a 10 allo screening
    10.Assunzione della stessa terapia di base per SMA(es. terapia di up-regolazione per sopravvivenza del motoneurone[SMN] approvata, come nusinersen, o nessuna terapia per SMA)per almeno 6 mesi prima dello screening e previsione di assunzione di tale terapia per tutta la durata dello studio
    a.Se sta ricevendo la terapia di up-regolazione SMN a base di nusinersen, deve aver completato il regime di carico e avviato il dosaggio di mantenimento (cioè, almeno una dose di mantenimento completata) almeno 4 settimane dopo la prima dose di mantenimento precedente lo screening
    11.Stato nutrizionale stabile negli ultimi 6mesi, con previsione di stabilità per tutta la durata dello studio
    12.Nessuna limitazione fisica che potrebbe impedire al paziente di sottoporsi alle misure di esito della funzionalità motoria durante tutta la durata dello studio
    13.In grado di ricevere infusioni di farmaco in studio e fornire campioni di sangue attraverso l’uso di un dispositivo endovenoso(EV)periferico o un dispositivo di accesso EV a lungo termine applicato al paziente per motivi indipendenti dallo studio(es. per le cure mediche di base e non per ricevere SRK-015 nello studio)per tutta la durata dello studio
    14.In grado di rispettare i requisiti del protocollo, compresi i viaggi al centro dello studio e il completamento di tutte le procedure e le visite dello studio
    15.Per i pazienti che si prevede abbiano raggiunto la maturità riproduttiva entro la fine dello studio, è richiesto il rispetto dei requisiti specifici di contraccezione dello studio
    a.Le donne potenzialmente fertili devono presentare un test di gravidanza negativo allo screening e accettare di utilizzare misure contraccettive altamente efficaci(tasso annuo di fallimento pari o inferiore all’1% se usate in modo coerente e corretto)per tutta la durata dello studio e per 18settimane dopo l’ultima dose del farmaco in studio.
    (si prega di fare riferimento alla sinossi del protocollo)
    E.4Principal exclusion criteria
    1. Use of tracheostomy with positive pressure
    2. Use of chronic daytime non invasive ventilatory support for >16 hours
    daily in the 2 weeks prior to dosing, or anticipated to regularly receive
    such daytime ventilator support chronically over the duration of the
    study
    3. Any acute or comorbid condition interfering with the well being of the patient within 14 days of screening, including active systemic infection,
    the need for acute treatment or inpatient observation due to any reason
    4. Severe scoliosis and/or contractures at screening. Based on clinical
    judgement, any scoliosis or contractures present must be stable over the
    past 6 months, anticipated to be stable for the duration of the study and
    not prevent the patient from being evaluated on any functional outcome
    measures throughout the duration of the study.
    5. Pregnant or breastfeeding
    6. Major orthopedic or other interventional procedure, including spine or
    hip surgery, considered to have the potential to substantially limit the
    ability of the patient to be evaluated on any functional outcome
    measures, within 6 months prior to screening, or anticipated for the
    duration of the study
    7. Prior history of a hypersensitivity reaction to a monoclonal antibody
    or recombinant protein bearing an Fc domain (such as a soluble
    receptor-Fc fusion protein)
    8. Use of systemic corticosteroids within 60 days prior to screening.
    Inhaled or topical steroids are allowed.
    9. Treatment with investigational drugs within 3 months prior to
    screening
    10. Use of therapies with potentially significant muscle effects (such as
    androgens, insulin like growth factor, growth hormone, systemic beta
    agonist, botulinum toxin, or muscle relaxants) or muscle-enhancing
    supplements within 60 days prior to screening
    11. Patient has any other condition, which in the opinion of the
    Investigator may compromise safety or compliance, would preclude the
    patient from successful completion of the study, or interfere with the
    interpretation of the results
    1.Uso di tracheotomia con pressione positiva.
    2.Uso di supporto ventilatorio giornaliero cronico non invasivo per >16 ore al giorno nelle 2 settimane precedenti la somministrazione o previsione di utilizzo regolare di supporto ventilatorio diurno cronico per tutta la durata dello studio.
    3.Qualsiasi condizione acuta o di comorbilità interferente con il benessere del paziente entro 14 giorni dallo screening, tra cui infezione sistemica attiva, necessità di trattamento acuto o osservazione in regime di ricovero per qualsiasi motivo.
    4.Grave scoliosi e/o contratture allo screening. Secondo il giudizio clinico, qualsiasi forma di scoliosi o contrattura presente deve essere stabile da almeno 6 mesi, con previsione di stabilità per tutta la durata dello studio, e non deve impedire al paziente di essere valutato sulla base di qualsiasi misura di esito funzionale per tutta la durata dello studio.
    5.Gravidanza o allattamento.
    6.Intervento chirurgico maggiore, ortopedico o di altro tipo, inclusa chirurgia della colonna vertebrale o dell’anca, considerato potenzialmente in grado di limitare in maniera sostanziale la valutazione del paziente sulla base di qualsiasi misura di esito funzionale nei 6 mesi precedenti lo screening o previsto durante lo svolgimento dello studio.
    7.Anamnesi precedente di reazione da ipersensibilità ad un anticorpo monoclonale (mAb) o proteina ricombinante recante un dominio Fc (come una proteina di fusione del recettore Fc solubile).
    8.Uso di corticosteroidi sistemici somministrati nei 60 giorni precedenti lo screening. È consentito l’uso di steroidi per via inalatoria o topica.
    9.Trattamento con farmaci sperimentali nei 3 mesi precedenti lo screening.
    10.Uso di terapie con effetti muscolari potenzialmente significativi (come androgeni, fattore di crescita insulino-simile, ormone somatotropo, beta-agonista sistemico, tossina botulinica o miorilassanti) o integratori per il potenziamento muscolare nei 60 giorni precedenti lo screening.
    11.Il paziente presenta qualsiasi altra condizione che, secondo il parere dello sperimentatore, potrebbe compromettere la sicurezza o la conformità, precludere al paziente il corretto completamento dello studio o interferire con l’interpretazione dei risultati.
    E.5 End points
    E.5.1Primary end point(s)
    Cohort 1; Ambulatory Type 3 patients:
    Change from Baseline in the revised Hammersmith Scale (RHS) total score at Day 364 (Visit 15)
    Administration of the RHS includes the timed rise from floor and 10
    meter walk/run tests. The RHS is designed for SMA, validated in SMA,
    and considered clinically meaningful.
    Cohort 2 and Cohort 3;Type 2 and non ambulatory Type 3 patients:
    Change from Baseline in Hammersmith Functional Motor Skills Expanded
    (HFMSE) total score at Day 364 (Visit 15). HFMSE is designed for SMA, validated in SMA, and
    considered clinically meaningful.
    Coorte 1: pazienti deambulanti di tipo 3
    Variazione dal basale nel punteggio RHS totale al Giorno 364 (Visita 15)
    L'amministrazione del RHS comprende il rialzo cronometrato dal suolo e 10 metri di cammino/corsa. RHS è creato per la SMA, convalidato nella SMA, e considerato clinicamente significativo.
    Coorte 2 e coorte 3: pazienti di tipo 2 e non deambulanti di tipo 3
    Variazione dal basale nella scala HFMSE al Giorno 364 (Visita 15). HFMSE è creato per la SMA, convalidato nella SMA, e considerato clinicamente significativo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ongoing throughout the study
    Durante il corso dello studio
    E.5.2Secondary end point(s)
    Cohort 1; Ambulatory Type 3 patients:
    Change from Baseline in the RHS total score at other prespecified timepoints
    Proportion of patients achieving various magnitudes of change in RHS
    score from Baseline
    Change from Baseline in 6 minute walk test (6MWT)
    Change from Baseline in 30 second sit to stand
    Change from Baseline in 10 meter walk/run (from the RHS)
    Change from Baseline in timed rise from floor (from the RHS)
    Cohort 2 and Cohort 3;Type 2 and non ambulatory Type 3 patients:
    Change from Baseline in the RHS total score at other prespecified timepoints
    Proportion of patients achieving various magnitudes of change in HFMSE
    score from Baseline
    Change from Baseline in Revised Upper Limb Module (RULM)
    Change from Baseline in number of WHO motor development milestones attained
    Proportion of patients achieving various magnitudes of change in RULM
    score from Baseline
    Proportion of patients who attain a new WHO motor development
    milestone relative to Baseline
    Coorte 1: pazienti deambulanti di tipo 3
    Variazione dal basale del punteggio HFMSE totale in altri punti temporali predefiniti
    Percentuale di pazienti che raggiungono diversi livelli di variazione nel punteggio RHS rispetto al basale
    Variazione dal basale nel test del cammino di 6 minuti (6MWT)
    Variazione dal basale nella capacità di passare dalla posizione seduta alla posizione in piedi per 30 secondi
    Variazione dal basale nella capacità di camminare/correre per 10 metri (da RHS)
    Variazione dal basale nel test cronometrato di sollevamento da terra (da RHS)
    Coorte 2 e coorte 3: pazienti di tipo 2 e non deambulanti di tipo 3
    Variazione dal basale del punteggio HFMSE totale in altri punti temporali predefiniti
    Percentuale di pazienti che raggiungono diversi livelli di variazione nel punteggio HFMSE rispetto al basale
    Variazione dal basale nel modulo degli arti superiori revisionato (RULM)
    Variazione dal basale nel numero di tappe di sviluppo motorio raggiunte secondo quanto previsto dall’OMS
    Percentuale di pazienti che raggiungono diversi livelli di variazione nel punteggio RULM rispetto al basale
    Percentuale di pazienti che raggiungono una nuova tappa di sviluppo motorio rispetto al basale secondo quanto previsto dall’OMS
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ongoing throughout the study
    Durante il corso dello studio
    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 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 Yes
    E.8.1.7.1Other trial design description
    Coorte 1 e 2 in aperto, coorte 3 in doppio cieco randomizzata
    Cohort 1 and 2 are open label and Cohort 3 is double blind randomized
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Per la coorte 3: due differenti dosi di farmaco in studio (2 e 20 Mg/Kg)
    For Cohort 3: two different doses (2 and 20 Mg/kg) of the study drug
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Italy
    Netherlands
    Spain
    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
    LVLP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months4
    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 months11
    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.1.1.1In 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: 35
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 16
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    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 Yes
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 55
    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)
    Patients that complete the 52-week treatment period have the option
    to enroll into an Extension Period, for an additional 52-week period.
    I pazienti che completano il periodo di trattamento di 52 settimane hanno la possibilità di essere arruolati in un periodo di estensione, per ulteriori 52 settimane
    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 Decision2019-06-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-05
    P. End of Trial
    P.End of Trial StatusOngoing
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