Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   43207   clinical trials with a EudraCT protocol, of which   7151   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2012-004987-23
    Sponsor's Protocol Code Number:CY4026
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-01-17
    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 ConcernedGermany - BfArM
    A.2EudraCT number2012-004987-23
    A.3Full title of the trial
    A Phase IIb, multi-national, double-blind, randomised, placebo-controlled study to evaluate the safety, tolerability and efficacy of CK-2017357 in patients with amyotrophic lateral sclerosis (ALS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in which patients suffering from amyotrophic lateral sclerosis (ALS) may be assigned randomly to receive either the treatment or placebo to look at how safe the product is, whether it has an effect and is easy to take.
    A.4.1Sponsor's protocol code numberCY4026
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01709149
    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,
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016506242929
    B.5.5Fax number0016506243010
    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 CK2017357
    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 name 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one
    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.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 Yes
    D.2.1.1.1Trade name Rilutek
    D.2.1.1.2Name of the Marketing Authorisation holderAventis Pharma S.A
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameRiluzole
    D.3.4Pharmaceutical form Capsule
    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 INNRILUZOLE
    D.3.9.1CAS number 1744-22-5
    D.3.9.4EV Substance CodeSUB10319MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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
    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
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    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
    Amyotrophic lateral sclerosis
    E.1.1.1Medical condition in easily understood language
    Amyotropic lateral sclerosis is a form of motor neurone disease where attacks of the nerve cells responsible for sending instructions to the muscles lead to weakness, muscle waste and paralysis.
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    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
    To assess the effect of CK-2017357 versus placebo on the ALSFRS-R total score when administered twice daily at each patients's maximum tolerated dose, up to a maximum of 500 mg daily.
    E.2.2Secondary objectives of the trial
    Exploration of alternative methods to assess the effect of CK-2017357 versus placebo on the change from baseline in the ALSFRS-R score and to assess the effect of CK-2017357 versus placebo on certain measures of respitratory and skeletal muscle function.
    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)
    2. Male or female 18 years or older
    3. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology EI escorial criteria)
    4. Upright Slow Vital Capacity (SVC) > 50% of predicted for age, height and sex
    5. At least 4 of the 12 ALSFRS-R questions must be scored 2 or 3
    6. Diminished but measurable maximum voluntary grip strength in at least one hand; i.e. between 10 and 50 pounds (females) and 10 and 70 pounds (males)
    7. Able to swallow tablets without crushing, and in the opinion of the Investigator, is expected to do so for the duration of the study
    8. A caregive (if one is needed) who can and will observe and report the patient's status
    9. Pre-study clinical laboratory findings within normal range, or if outside, of the normal range, deemed not clinically significant by the Investigator
    10. Male patients must agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reporduction potential and to have female partners use an additional effective means of contraception (e.g. diaphragm plus spermicide, or oral contraceptives) or the male patients must agree to abstain from sexual intercourse during and for 10 weeks after the end of the study
    11. Female patients must be post-menopausal (≥ 1 year) or sterilized, or, if of childbearing potential, not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and use contraceptive drugs or devices as detailed in item 10 for the duration of the study and for 10 weeks after the end of the study.
    12. Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 dats prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.
    E.4Principal exclusion criteria
    1. Any use of non-invasive positive pressure ventilation (NIPPV, e.g. CPAP or BiPAP) for any protion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation.
    2. Patients with a diaphragm pacing system (DPS) at study entry or who anticipates DPS placement during the course of the study.
    3. Body Mass Index (BMI) of 19.0 kg/m² or lower
    4. Unwilling to discontinue tizanidine and theophylline-containing medications during the study participation
    5. Serum chloride < 100 mmol/L
    6. Neurological impairment due to a condition other than ALS, including a history of TIA within the past year
    7. Presence at screening of any medically significant cardiac, pulmonary, GI, musculoskeletal or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data, including, but not limited to:
    a. Poorly controlled hypertension
    b. NYHA Class II or greater congestive heart failure
    c. Clinically significant ECG abnormalities
    d. Chronic obstructive pulmonary disease or asthma requiring frequent use of bronchodilator medications
    e. History of sleep apnea requiring CPAP or oxygen supplementation
    f. GI disorder that might impair absorption of study drug from the gastorintestinal tract
    g. History of significant liver disease defined by bilirubin > 2 times the upper limit of normal (ULN) or alanine transferase (ALT) or aspartate aminotransferase (AST) > 3 times the ULN on repeat testing
    h. Poorly controlled diabetes mellitus
    i. History of vertigo within 3 months of study entry
    j. History of syncope without an explainable or treated cause
    k. History of untreated intracranial aneurysm or poorly controlled seizure disorder
    l. Amputation of a limb
    m. Cognitive impairment, related to ALS or otherwise, sufficient to impair the patient's ability to understand and/or comply with study procedures and giving informed consent
    n. Cancer with metatstic potential (other than basal cell carcinoma, carcinoma in situ of cervix, or squamous cell carcinoma of the skin excised with clean margins) daignosed and treated within the last 2 years
    o. Any other condition, impairment or social circumstance that, in the opinion of the Investigator, would render the patient not suitable to participate in the study.
    p. Patient judged to be actively suicidal and a suicide risk by the Investigator.
    8. Has taken any investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing.
    9. Previously received CK-2017357 in any previous trial
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the change from baseline to the average of the ALSFRS-R total score obtained at Visits 6 and 7 (i.e. after approximately 8 and 12 weeks of double-blind treatment)
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 8-12 weeks of double-blind treatment
    E.5.2Secondary end point(s)
    1. ALSFRS-R enpoints for secondary analyses
    2. Respiratory function
    - Maximum Voluntary Ventilation (MVV)
    - Sniff Nasal Inspiratory Pressure (SNIP)
    - Slow Vital Capacity (SVC)
    3. Skeletal muscle function
    - Handgrip strength and fatigability
    -Maximum handgrip strenth
    -Sub-maximum handgrip fatigue
    -Muscle strength by hand-held dynamometry (HDD) as determined by the megascore of:
    -Elbow flexion (bilateral)
    - Wrist extension (bilateral)
    - Knee extension (bilateral)
    -Ankle dorsiflexion (bilateral)

    E.5.2.1Timepoint(s) of evaluation of this end point
    At visit 5, 6, 7, 8 and follow-up
    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 No
    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) 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA19
    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
    Canada
    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
    Last visit last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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: 700
    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 state65
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 280
    F.4.2.2In the whole clinical trial 700
    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 but patients that were currently taking Riluzole can resume their prescribed dose of riluzole (50mg twice daily) starting the last da after their last dose of study drug.
    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 Decision2013-03-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-03-21
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2023 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA