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    Summary
    EudraCT Number:2016-002629-13
    Sponsor's Protocol Code Number:CY4033
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-03-10
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2016-002629-13
    A.3Full title of the trial
    A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients with Amyotrophic Lateral Sclerosis (ALS) who Completed VITALITY-ALS (CY 4031)
    Estudio de fase 3, extensión abierta de tirasemtiv en pacientes con esclerosis lateral amiotrófica (ELA) que hayan finalizado el estudio VITALITY-ALS (CY 4031)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label Extension Study for Patients Who Completed VITALITY-ALS (CY 4031)
    Estudio de extension abierta en pacientes que hayan finalizado el estudio VITALITY-ALS (CY 4031)
    A.3.2Name or abbreviated title of the trial where available
    VIGOR-ALS
    A.4.1Sponsor's protocol code numberCY4033
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02936635
    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 SponsorCytokinetics, 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 supportCytokinetics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCytokinetics, Inc.
    B.5.2Functional name of contact pointMedical Affairs
    B.5.3 Address:
    B.5.3.1Street Address280 East Grand Avenue
    B.5.3.2Town/ citySouth San Francisco, California
    B.5.3.3Post code94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34913913443
    B.5.6E-mailmedicalaffairs@cytokinetics.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/12/970
    D.3 Description of the IMP
    D.3.1Product nameTirasemtiv
    D.3.2Product code CK-2017357
    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 INNTirasemtiv
    D.3.9.1CAS number 1005491-05-3
    D.3.9.2Current sponsor codeCK-2017357
    D.3.9.3Other descriptive name6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one
    D.3.9.4EV Substance CodeSUB93455
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis (ALS)
    Esclerosis Lateral Amiotrófica (ELA)
    E.1.1.1Medical condition in easily understood language
    Amyotrophic lateral sclerosis, or ALS, is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement.
    La esclerosis lateral amiotrófica (ELA) es una enfermedad de las células nerviosas del cerebro y la médula espinal que controlan los movimientos musculares voluntarios.
    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 19.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    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 assess the long-term safety and tolerability of tirasemtiv, in patients with ALS.
    El objetivo principal es determinar la seguridad y la tolerabilidad a largo plazo del tirasemtiv en pacientes con ELA.
    E.2.2Secondary objectives of the trial
    • To compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033
    • To compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033
    • To compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033
    • Comparar la evolución clínica de los pacientes que finalizaron el tratamiento con tirasemtiv en el estudio CY 4031 con la de los pacientes que finalizaron el tratamiento con placebo en el estudio CY 4031 durante la continuación del tratamiento con tirasemtiv de ambos grupos en el estudio CY 4033
    • Comparar la evolución clínica de los pacientes que finalizaron el tratamiento con tirasemtiv en el estudio CY 4031 durante ese estudio con su evolución clínica durante la continuación del tratamiento con tirasemtiv en el estudio CY 4033
    • Comparar la evolución clínica de los pacientes que finalizaron el tratamiento con placebo en el estudio CY 4031 durante ese estudio con su evolución clínica durante el tratamiento con tirasemtiv en el estudio CY 4033
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
    2. Completed participation on study drug and the Follow-Up Visit in the CY 4031 study
    3. Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:
    a. Use a condom during sexual intercourse with female partners who are of childbearing potential (i.e., following menarche until post-menopausal if not anatomically and physiologically incapable of becoming pregnant) AND to have female partners use a highly effective means of contraception (see below):
    OR
    b. Abstain from sexual intercourse during participation in the study.
    4. Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:
    a. Not be breastfeeding
    b. Have a negative pregnancy test
    c. Have no intention to become pregnant during participation in the study,
    AND
    d. Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure, for the duration of the study such as:
    − Combined (estrogen and progestogen containing) oral, intravaginal, or transdermal hormonal contraception associated with inhibition of ovulation
    − Progestogen-only oral, injectable, or implantable hormonal contraception associated with inhibition of ovulation
    − Intrauterine device (IUD)
    − Intrauterine hormone-releasing system (IUS)
    − Bilateral tubal occlusion
    1. Ser capaz de comprender y estar dispuesto a firmar un documento de consentimiento informado (ICF, Informed Consent Form). Si el consentimiento se otorgara de forma verbal, un representante legal del paciente deberá firmar el documento de consentimiento informado
    2. Haber finalizado la participación en el estudio CY 4031 en tratamiento con el fármaco del estudio y haber realizado la visita de seguimiento de dicho studio
    3. Los pacientes varones que no hayan sido vasectomizados Y sin un número de espermatozoides igual a cero confirmado, durante su participación en el estudio deberán aceptar:
    a. Utilizar un preservativo durante sus relaciones sexuales con mujeres potencialmente fértiles (es decir, desde después de la menarquía hasta después de la menopausia si no existe una causa anatómica y fisiológica por la que no puedan quedarse embarazadas) Y que sus parejas femeninas utilicen un método anticonceptivo altamente eficaz (véase más adelante):
    O BIEN
    b. Abstenerse de mantener relaciones sexuales durante la participación en el estudio.
    4. Las pacientes que no sean posmenopáusicas (≥ 1 año) o no estén esterilizadas, deberán:
    a. No amamantar
    b. Tener una prueba de embarazo negative
    c. No tener intención de quedarse embarazadas durante la participación en el estudio,
    Y
    d. Practicar la abstinencia sexual, que se define como la abstención de relaciones sexuales durante todo el estudio O, si el compañero sexual no está vasectomizado con un número de espermatozoides de cero confirmado, será necesario utilizar un preservativo Y emplear un método anticonceptivo altamente eficaz durante todo el estudio, como, por ejemplo:
    - Anticonceptivo hormonal combinado (con estrógenos y gestágenos) oral, intravaginal o transdérmico resultante en inhibición de la ovulación
    - Anticonceptivo hormonal con gestágenos sólo, por vía oral, inyectable o implantable resultante en inhibición de la ovulación
    - Dispositivo intrauterino (IUD, intrauterine device)
    - Sistema intrauterino (IUS, intrauterine system) de liberación hormonal
    - Oclusión tubárica bilateral
    E.4Principal exclusion criteria
    1. Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study
    2. Has taken any investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
    3. Use of tizanidine and theophylline-containing medications during study participation
    4. Participation or planning to participate in another clinical trial involving stem cell therapy for the treatment of ALS or another investigational drug
    1. Ser portador de un marcapasos diafragmático (DPS, diaphragm pacing system) al entrar en el estudio o estar prevista su colocación en el transcurso del studio
    2. Haber recibido un fármaco en investigación (distinto del tirasemtiv) antes de la administración prevista del tirasemtiv, en los 30 días anteriores a este o cinco semividas del fármaco previo, eligiéndose el mayor de estos valores
    3. Empleo de medicamentos con tizanidina y teofilina durante la participación en el studio
    4. Estar participando o tener pensado participar en cualquier forma de terapia con células madre para el tratamiento de la esclerosis lateral amiotrófica o con otro fármaco en investigación
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the incidence of adverse events (AEs) in the patient population.
    El criterio principal de valoración es la incidencia de acontecimientos adversos (AE, adverse events) en la población de pacientes.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Entire duration of the study.
    Duración completa del estudio.
    E.5.2Secondary end point(s)
    • Time to first use of assisted ventilation or death
    • Time to the first occurrence of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for >/=22 hours per day for >/=10 consecutive days) or death
    • Time to death
    • Decline in percent predicted SVC from baseline
    • Decline in ALSFRS-R score from baseline
    • Slope of the change from baseline in percent predicted SVC
    • Slope of the change from baseline in ALSFRS-R
    • Tiempo transcurrido hasta el primer uso de ventilación asistida o hasta la muerte
    • Tiempo hasta la primera aparición de insuficiencia respiratoria (definida como traqueotomía o empleo de ventilación no invasiva [NIV, non-invasive ventilation] durante >/=22 horas al día y >/=10 días consecutivos) o hasta la muerte
    • Tiempo transcurrido hasta la muerte
    • Descenso porcentual de la capacidad vital lenta (SVC, slow vital capacity) predicha respecto a su valor basal
    • Descenso de la puntuación de la ALS Functional Rating Scale –Revised (ALSFRS-R) respecto a la puntuación basal
    • Pendiente de la variación porcentual de la SVC predicha respecto al valor basal
    • Pendiente de la variación de la ALSFRS-R respecto al momento basal
    E.5.2.1Timepoint(s) of evaluation of this end point
    The time to event endpoints will be assessed from the start of CY 4031 and from the start of CY 4033 to the end of CY 4033. The change from baseline endpoints will be assessed from the start of CY 4031 and from the start of CY 4033 to Week 24 and Week 48 of CY 4033. The slope of change endpoints will be assessed during the first 24 weeks and first 48 weeks of either CY 4031 or CY 4033.
    Los criterios de valoración del tiempo hasta el acontecimiento se evaluarán desde el comienzo del CY 4031 y desde el comienzo del CY 4033 hasta el final del CY 4033. La variación respecto a los criterios de valoración basales se evaluará desde el comienzo del CY 4031 y desde el comienzo del CY 4033 hasta la semana 24 y la semana 48 del CY 4033. Los criterios de valoración de la pendiente de variación se evaluarán durante las primeras 24 semanas y las primeras 48 semanas ya sea del CY 4031 o del CY 4033.
    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 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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Canada
    France
    Germany
    Ireland
    Italy
    Netherlands
    Portugal
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    última visita del último paciente
    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 months0
    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 months0
    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: 250
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 114
    F.4.2.2In the whole clinical trial 350
    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)
    None
    Ninguno
    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 Decision2017-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-03-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-10-26
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