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    Summary
    EudraCT Number:2021-000251-39
    Sponsor's Protocol Code Number:TAK-994-1504
    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:2021-08-17
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-000251-39
    A.3Full title of the trial
    A Dose-Blind Extension Study With Double-blind, Placebo-Controlled, Randomized Withdrawal Period to Evaluate the Safety and Explore the Pharmacokinetics and Pharmacodynamics of TAK-994 in Adults With Narcolepsy With Cataplexy (Narcolepsy Type 1)
    Uno studio di estensione in cieco della dose, con periodo di sospensione randomizzato in doppio cieco, controllato con placebo, per valutare la sicurezza ed esplorare la farmacocinetica e la farmacodinamica di TAK-994 negli adulti con narcolessia con cataplessia (narcolessia di tipo 1).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of TAK-994 in Participants With Narcolepsy Type 1
    Uno studio di TAK-994 in pazienti con narcolessia di Tipo 1
    A.3.2Name or abbreviated title of the trial where available
    TAK-994 Extension and Randomized Withdrawal Study in Adult Subjects With Narcolepsy Type 1
    Studio di estensione con periodo di sospensione randomizzato di TAK-994 in soggetti adulti con narco
    A.4.1Sponsor's protocol code numberTAK-994-1504
    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 SponsorTAKEDA DEVELOPMENT CENTER AMERICAS 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 supportTakeda Development Center Americas, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Study Registration
    B.5.2Functional name of contact pointStudy Registration Call Center
    B.5.3 Address:
    B.5.3.1Street Address95 Hayden Avenue
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018778253327
    B.5.6E-mailmedinfoUS@takeda.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTAK-994 90 mg
    D.3.2Product code [TAK-994]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNTAK-994
    D.3.9.1CAS number 2274802-95-6
    D.3.9.2Current sponsor codeTAK-994
    D.3.9.4EV Substance CodeSUB216101
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number90
    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.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTAK-994 30 mg
    D.3.2Product code [TAK-994]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNTAK-994
    D.3.9.1CAS number 2274802-95-6
    D.3.9.2Current sponsor codeTAK-994
    D.3.9.4EV Substance CodeSUB216101
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Narcolepsy With Cataplexy
    Narcolessia con cataplessia
    E.1.1.1Medical condition in easily understood language
    Extreme sleepiness with episodes of sudden muscle weakness
    Sonnolenza estrema con episodi di debolezza muscolare improvvisa
    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 PT
    E.1.2Classification code 10028713
    E.1.2Term Narcolepsy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the safety and tolerability of TAK-994 in the active drug extension period of the study over a period of up to 8 weeks.
    L'obiettivo principale è valutare la sicurezza e la tollerabilità di TAK-994 nel periodo di estensione del farmaco attivo dello studio per un periodo fino a 8 settimane.
    E.2.2Secondary objectives of the trial
    The secondary objective is to evaluate the safety and tolerability of TAK-994 versus placebo in the randomized withdrawal period of the study over a period of up to 4 weeks.
    L'obiettivo secondario è valutare la sicurezza e la tollerabilità di TAK-994 rispetto al placebo nel periodo di sospensione randomizzato dello studio per un periodo fino a 4 settimane.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject eligibility is determined according to the following criteria before entry into the study:
    1. Subject with a diagnosis of NT1 who has completed TAK-994-1501 Part B before enrollment (which will occur immediately following the final TAK-994-1501 assessments), and for whom the investigator has no clinical objection they be enrolled.
    2. Subject is capable of understanding and complying with protocol requirements.
    3. Male subject who is not sterilized and sexually active with a female partner of childbearing potential, must use barrier contraception from signing of informed consent until 5 half-lives of TAK-994 plus 90 days after the last dose of study drug. In addition, they must be advised not to donate sperm during this period.
    4. Female subject of childbearing potential who is sexually active with a male partner who is not sterilized, must agree to use highly effective methods of contraception from signing of informed consent until 5 half lives of TAK-994 plus 30 days after the last dose of study drug. In addition, they must be advised not to donate ova during this period.
    5. Subject must agree to participate by providing written informed consent.
    L’idoneità del soggetto viene determinata in base ai seguenti criteri prima dell’ammissione allo studio:
    1. Soggetto con diagnosi di NT1 che ha completato TAK-994-1501 Parte B prima dell’arruolamento (che si verificherà immediatamente dopo le valutazioni finali di TAK-994-1501) e per il cui arruolamento lo sperimentatore non ha obiezioni cliniche.
    2. Il soggetto è in grado di comprendere e rispettare i requisiti del protocollo.
    3. Il soggetto di sesso maschile che non è sterilizzato ed è sessualmente attivo con una compagna fertile deve utilizzare metodi contraccettivi di barriera dalla firma del consenso informato fino a 5 emivite di TAK-994 più 90 giorni dopo l’ultima
    dose del farmaco dello studio. Inoltre, è necessario consigliare al soggetto di non donare sperma durante questo periodo.
    4. Il soggetto di sesso femminile in età fertile che è sessualmente attivo con un compagno non sottoposto a sterilizzazione, deve accettare di usare metodi contraccettivi altamente efficaci dalla firma del consenso informato fino a 5 emivite di TAK-994 più 30 giorni dopo l’ultima dose del farmaco dello studio. Inoltre, è necessario consigliare al soggetto di non donare ovuli durante questo periodo.
    5. Il soggetto deve acconsentire a partecipare fornendo il consenso informato scritto.
    E.4Principal exclusion criteria
    1. Subject has a clinically significant moderate or severe ongoing adverse event (AE) related to the study drug from the prior study.
    2. Subject has used/uses disallowed concomitant medication.
    3. Subject, in the opinion of the investigator, is unlikely to comply with the clinical study protocol or is unsuitable for any reason.
    4. The subject is an employee of the sponsor or study site or an immediate family member (eg, spouse, parent, child, sibling) of an employee of the sponsor or study site who is directly involved in the conduct of the study.
    5. The subject has a known hypersensitivity to any component of the formulation of TAK-994 or related compounds.
    6. The subject has a positive pregnancy test or is a lactating/breastfeeding woman.
    7. The subject had major surgery or donated or lost 1 unit of blood (approximately 500 mL) within 4 weeks before Baseline I (excluding samples taken as part of TAK-994-1501).
    8. The subject's renal creatinine clearance is =50 mL/min.
    9. Has liver function tests (alanine aminotransferase, aspartate aminotransferase) higher than 1.5 times the upper limit of normal at any visit in TAK-994-1501.
    10. The subject has a risk of suicide according to endorsement of Item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) at any visit during TAK-994-1501 and/or has made a suicide attempt during TAK-994-1501.
    1. Il soggetto presenta un evento avverso (EA) in corso, moderato o grave, clinicamente significativo, correlato al farmaco dello studio dello studio precedente.
    2. Il soggetto ha fatto/fa uso di un farmaco concomitante non autorizzato.
    3. A giudizio dello sperimentatore, il soggetto non è in grado di attenersi al protocollo dello studio clinico o non è idoneo per qualche motivo.
    4. Il soggetto è un dipendente dello sponsor o del centro dello studio o un familiare prossimo (ad esempio coniuge, genitore, figlio/a, fratello/sorella) di un dipendente dello sponsor o del centro dello studio che è direttamente coinvolto nella conduzione dello studio.
    5. Il soggetto presenta ipersensibilità nota a qualsiasi componente della formulazione di TAK-994 o a composti correlati.
    6. Il soggetto risulta positivo al test di gravidanza o è una donna in fase di allattamento al seno.
    7. Il soggetto è stato sottoposto a un intervento chirurgico di entità maggiore oppure ha donato o perso 1 unità di sangue (circa 500 ml) nelle 4 settimane precedenti il Basale I (esclusi i campioni prelevati come parte di TAK-994-1501).
    8. La clearance della creatinina renale del soggetto è =50 ml/min.
    9. I risultati dei test di funzionalità epatica (alanina aminotransferasi, aspartato aminotransferasi) del soggetto sono superiori a 1,5 volte il limite superiore della norma in qualsiasi visita di TAK-994-1501.
    10. Il soggetto presenta un rischio di suicidio secondo l’articolo 4 o 5 della Scala di valutazione della gravità del rischio di suicidio formulata dalla Columbia University (C-SSRS) a qualsiasi visita durante TAK-994-1501 e/o ha effettuato un tentativo di suicidio durante TAK-994-1501.
    E.5 End points
    E.5.1Primary end point(s)
    Subjects with at least 1 treatment-emergent adverse event (TEAE) during the active drug extension period of the study.
    Subjects with at least 1 markedly abnormal value (MAV) for postdose laboratory values during the active drug extension period of the study.
    Subjects with at least 1 MAV for postdose vital signs during the active drug extension period of the study.
    Subjects with at least 1 MAV for postdose ECG parameters during the active drug extension period of the study.
    Soggetti con almeno 1 evento avverso emergente dal trattamento (TEAE) durante il periodo di estensione del farmaco attivo dello studio.
    Soggetti con almeno 1 valore significativamente anomalo (MAV) nei valori di laboratorio post-dose durante il periodo di estensione del farmaco attivo dello studio. Soggetti con almeno 1 MAV nei segni vitali durante il periodo di estensione del farmaco attivo dello studio.
    Soggetti con almeno 1 MAV nei parametri ECG post-dose durante il periodo di estensione del farmaco attivo dello studio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Active drug extension period of the study
    Periodo di estensione del farmaco attivo dello studio
    E.5.2Secondary end point(s)
    Subjects with at least 1 TEAE during the randomized withdrawal period of the study.
    Subjects with at least 1 MAV for postdose laboratory values during the randomized withdrawal period of the study.
    Subjects with at least 1 MAV for postdose vital signs during the randomized withdrawal period of the study.
    Subjects with at least 1 MAV for postdose ECG parameters during the randomized withdrawal period of the study.
    Soggetti con almeno 1 TEAE durante il periodo di sospensione randomizzata dello studio.
    Soggetti con almeno 1 MAV nei valori di laboratorio post-dose durante il periodo di ritiro randomizzato dello studio.
    Soggetti con almeno 1 MAV post-dose nei segni vitali durante il periodo di sospensione randomizzata dello studio.
    Soggetti con almeno 1 MAV nei i parametri ECG post-dose durante il periodo di sospensione randomizzata dello studio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Randomized withdrawal period of the study.
    Periodo di sospensione randomizzata 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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) 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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    Canada
    Japan
    Korea, Republic of
    United States
    Finland
    France
    Hungary
    Italy
    Netherlands
    Spain
    Czechia
    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
    The study ends when the last subject completes the last planned or follow-up visit/interaction associated with a planned visit (this can be a phone contact), withdraws from the study, or is lost to follow-up (ie, the investigator is unable to contact the subject).
    Lo studio termina quando l'ultimo soggetto completa l'ultima visita / interazione pianificata o di follow-up associata a una visita pianificata (può essere un contatto telefonico), si ritira dallo studio o si perde per il follow-up (cioè, lo sperimentatore è impossibilitato a contattare il soggetto).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months10
    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 months10
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 112
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 27
    F.4.2.2In the whole clinical trial 112
    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)
    Study drug will not be available upon completion of the subject's participation in the study. The subject should be returned to the care of a physician and standard therapies as required.
    Il farmaco in studio non sarà disponibile al completamento del tema partecipazione allo studio. Il soggetto dovrebbe essere restituito alle cure di un medico e terapie standard secondo necessità.
    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 Decision2021-07-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2021-10-05
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