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    Summary
    EudraCT Number:2020-003136-25
    Sponsor's Protocol Code Number:CTD-TCNPC-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-08-23
    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 number2020-003136-25
    A.3Full title of the trial
    A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl ß cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1
    Studio di fase III, in doppio cieco, randomizzato, controllato con placebo, a gruppi paralleli, multicentrico per valutare la sicurezza, tollerabilità ed efficacia di 2000 mg/kg di Trappsol® Cyclo™ (idrossipropil-ß-ciclodestrina) e terapia standard rispetto a placebo e terapia standard in pazienti affetti da malattia di Niemann-Pick di tipo C1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1
    Studio per valutare la sicurezza, tollerabilità ed efficacia di 2000 mg/kg di Trappsol® Cyclo™ (idrossipropil-ß-ciclodestrina) e terapia standard rispetto a placebo e terapia standard in pazienti affetti da malattia di Niemann-Pick di tipo C1
    A.3.2Name or abbreviated title of the trial where available
    Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Stand
    Studio per valutare la sicurezza, tollerabilità ed efficacia di 2000 mg/kg di Trappsol® Cyclo™ (idro
    A.4.1Sponsor's protocol code numberCTD-TCNPC-301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/162/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCyclo Therapeutics, 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 supportCyclo Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCyclo Therapeutics, Inc.
    B.5.2Functional name of contact pointChief Scientific Officer
    B.5.3 Address:
    B.5.3.1Street Address16th Street, Suite B
    B.5.3.2Town/ cityGainesville
    B.5.3.3Post code6714 NW
    B.5.3.4CountryUnited States
    B.5.4Telephone number3864188060
    B.5.6E-mailsharon.hrynkow@cyclodex.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/3/11/895
    D.3 Description of the IMP
    D.3.1Product nameTrappsol® Cyclo™
    D.3.2Product code [N/A]
    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 INNN/A
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive nameHYDROXYPROPYLBETADEX
    D.3.9.4EV Substance CodeSUB32181
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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 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 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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Niemann Pick Disease Type C1
    Niemann Pick Disease Type C1
    E.1.1.1Medical condition in easily understood language
    Rare inherited lysosomal lipidosis resulting in a progressive and fatal neurological deterioration
    Rara lipidosi lisosomiale ereditaria con conseguente deterioramento neurologico progressivo e fatale
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10029403
    E.1.2Term Niemann-Pick disease
    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
    This study has dual primary objectives, such that for the FDA and countries following the FDA guidance, the primary objective is to evaluate the effectiveness of Trappsol Cyclo and standard of care (SOC) compared to placebo and SOC as measured by an improvement in the 4 domain Niemann-Pick disease Type C-Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech, and Swallow]) composite score at either Week 48 or Week 96 and for the EU and countries following EMA guidance, the primary objective is to evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC as measured by an improvement in the 5 domain Niemann Pick disease Type C Severity Scale (5D NPC SS) (Ambulation, Fine Motor, Speech, Swallow, and Cognition) composite score at either Week 48 or Week 96. The objective that is not primary for a specific country will be an exploratory objective.
    Questo studio ha due obiettivi primari: per la FDA e i paesi che seguono le linee guida dell’FDA, l'obiettivo primario è quello di valutare l'efficacia di Trappsol Cyclo e terapia standard (SOC) rispetto a placebo e SOC misurato da un miglioramento del punteggio composito nella scala di gravità a 4 domini per la malattia di Niemann-Pick di tipo C (4D-NPC-SS [deambulazione, abilità fino motoria, parlato e deglutizione]) alla Settimana 48 o alla Settimana 96 e per l'UE e i paesi che seguono le linee guida dell’EMA, l'obiettivo primario è quello di valutare l'efficacia di Trappsol Cyclo e SOC rispetto a placebo e SOC misurato da un miglioramento del punteggio composito nella scala di gravità a 5 domini per la malattia di Niemann-Pick di tipo C (5D-NPC-SS) (deambulazione, abilità fino motoria, parlato, deglutizione e cognizione) alla Settimana 48 o alla Settimana 96.
    L'obiettivo che non è primario per un paese specifico sarà un obiettivo esplorativo.
    E.2.2Secondary objectives of the trial
    •To determine the ability of Trappsol Cyclo and SOC compared to placebo and SOC to improve ataxia as assessed by the Spinocerebellar Ataxia Functional Index (SCAFI)
    •To evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC as assessed by the Vineland Adaptive Behavior Scale 2nd edition (Vineland 2) composite raw score, including the optional Motor Skills domain
    •To evaluate the effectiveness of Trappsol Cyclo and SOC compared to placebo and SOC with regards to the patient's ability to swallow as assessed by the Penetration Aspiration Scale (PAS)
    • Determinare la capacità di Trappsol Cyclo e SOC rispetto a placebo e SOC di migliorare l'atassia come valutato dall'Indice Funzionale dell'Atassia Spinocerebellare (SCAFI)
    • Valutare l'efficacia di Trappsol Cyclo e SOC rispetto a placebo e SOC come valutato dal punteggio grezzo composito della 2° edizione della Scala del comportamento adattivo di Vineland (Vineland-2), incluso il dominio opzionale delle Abilità motorie
    • Valutare l'efficacia di Trappsol Cyclo e SOC rispetto a placebo e SOC per quanto riguarda la capacità di deglutizione del paziente come valutata dalla scala penetrazione- aspirazione (PAS)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: A substudy in patients newborn to <3 years of age will be conducted the European Union (EU) and countries following the European Medicines Agency (EMA) guidelines and will not be conducted in the United States (US) or countries following Food and Drug Administration (FDA) guidelines; this substudy is presented in Section 14.4 of the full protocol.
    This substudy will enroll patients newborn to <3 years of age in an exploratory module to evaluate the safety of Trappsol Cyclo in these patients, and to obtain descriptive data regarding global severity and improvement in response to Trappsol Cyclo from both Investigator and patient perspective (CGI-S, CGI-C, CaGI-S, CaGI-C, and CaGI C24). These patients will not be randomized, rather, these patients will all receive open-label Trappsol Cyclo and SOC. These patients will not contribute to the efficacy or safety data sets addressing the principal hypotheses of the study. These patients are eligible for 192 weeks of treatment with Trappsol Cyclo.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Un sottostudio in pazienti da 0 a <3 anni di età sarà condotto nell'Unione Europea (UE) e nei paesi che seguono le linee guida dell'Agenzia europea per i medicinali (EMA) e non sarà condotto negli Stati Uniti (USA) o nei paesi che seguono le linee guida della Food and Drug Administration (FDA); questo sottostudio è presentato nella sezione 14.4 del protocollo completo.
    Questo sottostudio arruolerà pazienti di età inferiore a 3 anni in un modulo esplorativo per valutare la sicurezza di Trappsol Cyclo in questi pazienti e per ottenere dati descrittivi sulla gravità globale e sul miglioramento della risposta a Trappsol Cyclo sia dal punto di vista dello sperimentatore che dal punto di vista del paziente (CGI-S, CGI-C, CaGI-S, CaGI-C; CaGI C24).
    Questi pazienti non saranno randomizzati, ma riceveranno tutti Trappsol Cyclo e SOC in aperto.
    Questi pazienti non contribuiranno ai set di dati di efficacia o sicurezza che affrontano le principali ipotesi dello studio.
    Questi pazienti possono beneficiare di 192 settimane di trattamento con Trappsol Cyclo.
    E.3Principal inclusion criteria
    1.Diagnosis of NPC1 confirmed by:
    a.Genetically confirmed (deoxyribonucleic acid sequence analysis) by mutations in both alleles of NPC1, OR
    b.Mutation in only 1 allele of NPC1 and either positive filipin staining in skin or vertical supranuclear gaze palsy (VSGP)
    2.Patients with an ASIS score between 0.5 to 2.0 at Screening using the 17-Domain Niemann-Pick Type C Severity Scale (17D NPC SS) composite score
    3.Treated or not treated with miglustat
    a.If a patient is receiving treatment with miglustat, the dose must have
    been stable for at least 3 continuous months prior to the first Screening
    Visit
    b.If a patient has been discontinued from prescribed treatment with miglustat, she/he must have been discontinued for at least 3 continuous months prior to the first Screening Visit
    4.Body weight >4.5 to =125 kg
    5.Presenting at least 1 neurological symptom of the disease (including, but not limited to, hearing loss, VSGP, ataxia, dementia, dystonia, history of seizures, dysarthria, or dysphagia)
    6.Willing and capable to participate in all aspects of trial design, including blood sampling (efficacy, PK, blood biomarkers, and safety laboratory tests). Adequate compliance with the assessments to obtain complete data can become a discussion between the Investigator and the medical monitor prior to randomization at the Baseline Visit
    7.Ability to travel to the corresponding clinical trial site at the scheduled visit times for evaluation and follow-up
    8.Contraception requirements:
    a.All sexually active WOCBP (post menarche) must use highly effective contraception during the study and until 3 months after the last dose of study treatment
    b.Highly effective birth control methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; or vasectomized partner menarche) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of study treatment
    d.Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during
    the study and for 3 months after the last dose of study treatment for sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient
    9.The patient or legally authorized representative has read and signed the informed consent (or assent, as applicable) form prior to any study related procedures
    10.The legally authorized representative (if applicable) agrees for the patient to participate in all aspects of the trial
    11.The patient's caregiver (as applicable) agrees to participate in all of the protocol-specified assessment scales, questionnaires, and interviews for the duration of the trial

    Criteria to Switch to Open label Trappsol Cyclo Treatment Prior to Week 96
    1.Received double-blind treatment for =36 weeks, AND
    2.CGI-S Overall Disease deterioration of =2 levels from Baseline at 2 consecutive assessment visits 12 weeks apart per SoA
    Note that based on these criteria the earliest timepoint that a patient may be switched to open-label Trappsol Cyclo due to deterioration is following completion of Week 48 assessments. Trappsol Cyclo will be administered at subsequent visits.

    Criteria to Participate in the Open-label Extension
    1.Completed double-blind treatment through Week 92 and completed Week 96 assessments, OR
    2.Discontinued early from double-blind treatment for reasons other than safety but completed all assessments through Week 96
    1. Diagnosi di NPC1 confermata da:
    a. Confermata geneticamente (analisi della sequenza di acido desossiribonucleico) da mutazioni in entrambi gli alleli della NPC1, OPPURE
    b. Mutazione in 1 solo allele della NPC1 e colorazione cutanea con filipin positiva o paralisi sopranucleare dello sguardo verticale (VSGP)
    2. Pazienti con un punteggio ASIS compreso tra 0,5 e 2,0 allo Screening utilizzando il punteggio composito della scala di gravità a 17 domini per malattia di Niemann-Pick di tipo C (17D-NPC-SS)
    3. Trattato o non trattato con miglustat
    a. Se un paziente è in trattamento con miglustat, la dose deve essere stabile per almeno 3 mesi continuativi prima della prima visita di Screening
    b. Se un paziente è stato sospeso dal trattamento prescritto con miglustat, la sospensione deve essere avvenuta per almeno 3 mesi continuativi prima della prima visita di Screening
    4. Peso corporeo da >4,5 a =125 kg
    5. Presentare almeno 1 sintomo neurologico della malattia (tra cui, in via esemplificativa ma non esclusiva, perdita dell'udito, VSGP, atassia, demenza, distonia, anamnesi di crisi epilettiche, disartria o disfagia)
    6. Volontà e capacità di partecipare a tutti gli aspetti del disegno della sperimentazione, compreso il prelevamento di campioni di sangue (efficacia, PK, biomarcatori del sangue e test di laboratorio per la sicurezza). L'adeguata conformità alle valutazioni per ottenere dati completi può essere oggetto di una discussione tra lo Sperimentatore e il medical monitor prima della randomizzazione alla visita Baseline.
    7. Possibilità di recarsi al centro della sperimentazione clinica negli orari di visita previsti per la valutazione e il follow-up
    8. Requisiti di contraccezione:
    a. Tutte le WOCBP (post -menarca) sessualmente attive devono utilizzare contraccettivi altamente efficaci durante lo studio e fino a 3 mesi dopo l'ultima dose di trattamento in studio
    b. I metodi anticoncezionali altamente efficaci includono la contraccezione ormonale combinata (contenente estrogeni e progestinici) associata all'inibizione dell'ovulazione (orale, intravaginale o transdermica); contraccezione ormonale solo progestinica associata all'inibizione dell'ovulazione (orale, iniettabile o impiantabile); dispositivo intrauterino; sistema di rilascio ormonale intrauterino; occlusione bilaterale delle tube; o partner vasectomizzato
    c. Tutti i pazienti maschi sessualmente attivi con partner WOCBP (post-menarca) devono utilizzare un preservativo con o senza spermicida oltre al metodo contraccettivo utilizzato dalla partner durante lo studio e fino a 3 mesi dopo l'ultima dose di trattamento in studio
    d. L'astinenza sessuale è considerata un metodo anticoncezionale altamente efficace solo se viene definita come astinenza da rapporti eterosessuali durante lo studio e per 3 mesi dopo l'ultima dose di trattamento in studio per le WOCBP e per i pazienti di sesso maschile con partner WOCBP. L'affidabilità dell'astinenza sessuale deve essere valutata dallo Sperimentatore in relazione alla durata della sperimentazione clinica e allo stile di vita preferito e abituale del paziente.
    9. Il paziente o il rappresentante legalmente autorizzato ha letto e firmato il modulo di consenso informato (o di assenso, come applicabile) prima di qualsiasi procedura correlata allo studio
    10. Il rappresentante legalmente autorizzato (se applicabile) accetta che il paziente partecipi a tutti gli aspetti della sperimentazione
    11. Il caregiver del paziente (come applicabile) si impegna a partecipare a tutte le scale di valutazione, i questionari e i colloqui previsti dal protocollo per tutta la durata della sperimentazione
    E.4Principal exclusion criteria
    1.Recipient of a liver transplant or planned liver transplantation
    2.Patients with active liver disease from any cause other than NPC1 or prolonged icterus or malformation of organs other than NPC1
    3.Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalized ratio >1.8
    4.Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2. In patients aged =18 years, eGFR is calculated according to the Schwartz equation (Schwartz and Work, 2009), and in patients aged >18 years eGFR is calculated using the Modification of Diet in Renal Disease equation
    5.Use of curcumin or fish oil within 12 weeks prior to enrollment
    6.Known or suspected allergy or intolerance to the study treatment
    7.In the opinion of the Investigator, the patient's clinical condition does not allow for the blood collection required as per the protocol specified procedures
    8.Treatment with any investigational drug during the 3 months prior to entering the study, including leucine in a clinical trial; however, leucine as a nutraceutical is allowed
    9.Treatment with HPßCD prior to entering the study
    10.Treatment with any other investigational drug during the study
    11.Pregnancy or breastfeeding
    12.Current participation in another trial is not permitted unless it is a noninterventional study and the sole purpose of the trial is for long term follow up describing clinical features or survival data (registry)
    13.Patients with uncontrolled, severe epileptic seizure periods (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to completion of informed consent (or assent, as applicable). This includes patients with ongoing seizures that are not stable in frequency or type or duration over a 2-month period prior to enrollment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2-month period prior to enrollment, or requiring 3 or more antiepileptic medications to control seizures over a 2-month period prior to enrollment
    14.Neurologically asymptomatic patients
    15.Inability to participate in the primary study assessment (4D-NPC-SS or 5D NPC SS, depending on jurisdiction) as determined by the Investigator
    1. Destinatario di un trapianto di fegato o trapianto di fegato pianificato
    2. Pazienti con malattia epatica attiva avente causa diversa dalla NPC1 o ittero prolungato o malformazione di organi diversi dalla NPC1
    3. Evidenza clinica di malattia epatica acuta, compresi sintomi di ittero o dolore al quadrante superiore destro o rapporto internazionale normalizzato >1,8
    4. Malattia renale cronica di stadio 3 o peggiore, come indicato dalla velocità di filtrazione glomerulare stimata (eGFR) <60 mL/min/1,73 m2. Nei pazienti di età =18 anni, l'eGFR viene calcolata secondo l'equazione di Schwartz (Schwartz eWork, 2009), e nei pazienti di età >18 anni l'eGFR viene calcolata utilizzando l’equazione della Modifica della dieta nella malattia renale
    5. Uso di curcumina o olio di pesce entro 12 settimane prima dell'arruolamento
    6. Allergia o intolleranza nota o sospetta al trattamento in studio
    7. Secondo l'opinione dello Sperimentatore, le condizioni cliniche del paziente non consentono il prelievo di sangue richiesto secondo le procedure specificate dal protocollo
    8. Trattamento con qualsiasi farmaco sperimentale durante i 3 mesi precedenti l'ingresso nello studio, inclusa leucina nell’ambito di una sperimentazione clinica; tuttavia, la leucina come nutraceutico è consentita
    9. Trattamento con HPßCD prima di entrare nello studio
    10. Trattamento con qualsiasi altro farmaco sperimentale durante lo studio
    11. Gravidanza o allattamento al seno
    12. L'attuale partecipazione ad un altro studio non è consentita, a meno che non si tratti di uno studio non interventistico e l'unico scopo dello studio sia un follow-up a lungo termine che descriva le caratteristiche cliniche o i dati di sopravvivenza (registro)
    13. Pazienti con periodi di crisi epilettiche gravi e incontrollate (almeno 3 crisi epilettiche gravi consecutive che hanno richiesto farmaci) entro 2 mesi prima del completamento del consenso informato (o dell’assenso, come applicabile). Ciò include i pazienti con crisi epilettiche in corso che non sono stabili in termini di frequenza, tipo o durata per un periodo di 2 mesi prima dell'arruolamento, che richiedono una modifica alla dose di farmaci antiepilettici (che non sia l'aggiustamento per il peso) per un periodo di 2 mesi prima dell'arruolamento, o che richiedono 3 o più farmaci antiepilettici per controllare le crisi per un periodo di 2 mesi prima dell'arruolamento
    14. Pazienti neurologicamente asintomatici
    15. Incapacità di partecipare alla valutazione primaria dello studio (4D-NPC-SS o 5D-NPC-SS a seconda della giurisdizione) come determinato dallo Sperimentatore
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoints of this study for the EMA and countries following the EMA guidelines are:
    •Mean change in the 5D NPC SS composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition) from Baseline (Week 0) to 48 weeks
    •Mean change in the 5D NPC SS composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition) from Baseline (Week 0) to 96 weeks
    • Variazione media del punteggio composito 5D-NPC-SS (deambulazione, abilità fino-motorie, parlato, deglutizione e cognizione) dalla Baseline (Settimana 0) a 48 settimane
    • Variazione media del punteggio composito 5D-NPC- SS (deambulazione, abilità fino-motorie, parlato, deglutizione e cognizione) dalla Baseline (Settimana 0) a 96 settimane
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 and 96 weeks
    48 e 96 settimane
    E.5.2Secondary end point(s)
    •Mean change from Baseline in the total SCAFI composite score at 48 and 96 weeks
    •Mean change from Baseline in the Vineland 2 composite raw score, including the optional Motor Skills domain, at 48 and 96 weeks
    •Mean change from Baseline in the patient's ability to swallow as assessed by the PAS, at 48 and 96 weeks (at select sites)
    • Variazione media del punteggio composito 4D-NPC-SS (deambulazione, abilità fino-motorie, parlato e deglutizione) dalla Baseline (Settimana 0) a 48 settimane
    • Variazione media del punteggio composito 4D-NPC- SS (deambulazione, abilità fino-motorie, parlato e deglutizione) dalla Baseline (Settimana 0) a 96 settimane
    • Variazione media dalla Baseline nel punteggio totale SCAFI a 48 e 96 settimane
    • Variazione media rispetto alla Baseline nel punteggio grezzo composito Vineland-2, compreso il dominio opzionale delle Abilità motorie, a 48 e 96 settimane
    • Variazione media rispetto alla Baseline nella capacità di deglutizione del paziente valutata dalla PAS, a 48 e 96 settimane (presso centri selezionati)
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 and 96 weeks
    48 e 96 settimane
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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.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 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
    Eligible patients may enter an Open-label extension upon completion of blinded treatment period
    Eligible patients may enter an Open-label extension upon completion of blinded treatment period
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 EEA12
    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
    Australia
    Israel
    United States
    France
    Germany
    Italy
    Poland
    Spain
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 19
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 17
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 63
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    children and newborn: 0-10 years old
    bambini e neonati: 0-10 anni
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 105
    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)
    not indicated
    non indicato
    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 Decision2021-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-01
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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