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    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   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).

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    Summary
    EudraCT Number:2020-003986-20
    Sponsor's Protocol Code Number:LUCAS
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2021-03-15
    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 ConcernedGermany - BfArM
    A.2EudraCT number2020-003986-20
    A.3Full title of the trial
    A Phase II, Open-Label, Multicenter Study of Orally Administered CA-4948 for the Treatment of Anemia in Patients with Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS)
    Eine offene Phase-II-Studie mit oral verabreichtem CA-4948 zur Behandlung von Anämie bei Patienten mit myelodysplastischen Syndromen (MDS) mit sehr niedrigem, niedrigem oder mittlerem Risiko
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of anemia with CA-4948 in very low, low, or intermediate risk MDS patients.
    Behandlung einer Anämie mit CA-4948 bei MDS Patienten mit sehr niedrigem, niedrigem oder mittlerem Risiko.
    A.3.2Name or abbreviated title of the trial where available
    LUCAS
    LUCAS
    A.4.1Sponsor's protocol code numberLUCAS
    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 SponsorLeipzig University
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCuris, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationZentrum für Klinische Studien Leipzig
    B.5.2Functional name of contact pointSusanne Melzer
    B.5.3 Address:
    B.5.3.1Street AddressHaertelstr. 16-18
    B.5.3.2Town/ cityLeipzig
    B.5.3.3Post code04107
    B.5.3.4CountryGermany
    B.5.4Telephone number+ 49 34197 16 317
    B.5.5Fax number+49 34197 16 189
    B.5.6E-mailsusanne.melzer@zks.uni-leipzig.de
    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 nameEmavusertib
    D.3.2Product code CA-4948
    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.3.9.1CAS number 1801344-14-8
    D.3.9.2Current sponsor codeCA-4948
    D.3.9.3Other descriptive name(R)-N-(5-(3-hydroxypyrrolidin-1-yl)-2-morpholinooxazolo[4,5-b]pyridin-6-yl)-2-(2-methylpyridin-4-yl)oxazole-4-carboxamide
    D.3.9.4EV Substance CodeSUB208117
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Anemia in patients with very low, low or intermediate risk myelodysplastic syndromes (MDS)
    Anämie bei Patienten mit myelodysplastischen Syndromen (MDS) mit sehr niedrigem, niedrigem oder mittlerem Risiko
    E.1.1.1Medical condition in easily understood language
    Anemia
    Anämie
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10028533
    E.1.2Term Myelodysplastic syndrome
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the proportion of patients who have an erythroid response (HI-E) according to IWG 2018 criteria separately for both independent substudies.
    Erhebung des Anteils der Patienten, die eine erythroide Reaktion (HI-E) haben, gemäss den Kriterien der IWG 2018, getrennt für beide unabhängigen Kohorten.
    E.2.2Secondary objectives of the trial
    - HI-E response duration
    - Time to HI-E
    - Hemoglobin changes from baseline
    - Red blood cell transfusion (RBC) independence ≥ 8 and ≥ 12 weeks
    - Frequency of RBC transfusions in transfusion dependent patients
    - Neutrophil and platelet (HI-N and HI-P) responses
    - Disease progression according to IWG 2018 criteria
    - Safety and toxcicity profile of CA-4948
    - Quality of life (QoL)
    - Mutational pattern and burden of selected genes and their influence on response
    - Dauer des HI-E-Ansprechens
    - Zeit bis HI-E
    - Unabhängigkeit der Erythrozytentransfusion (RBC) ≥ 8 und ≥ 12 Wochen
    - Häufigkeit von RBC-Transfusionen bei transfusions-abhängigen Patienten
    - Neutrophilen- und Thrombozytenanprechen (HI-N und HI-P)
    - Krankheitsprogression nach den Kriterien der IWG 2018
    - Sicherheits- und Toxizitätsprofil von CA-4948
    - Lebensqualität (QoL)
    - Mutationsmuster und Belastung ausgewählter Gene und deren Einfluss auf das Ansprechen
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - MDS OR MDS/MPN including MDS/MPN-RS-T, MDS/MPNu, aCML or CMML
    - IPSS-R up to 3.5: very low/low/intermediate risk disease (MDS); MDS/MPN < 10% bone marrow blasts; CPSS-Score low/intermediate for CMML
    - Symptomatic anemia
    - Defined transfusion strategy
    - No available option of an approved MDS therapy and classification of prior erythropoiesis-stimulating agent (ESA) treatment as follows:
    + Cohort A: ESA exposed (and refractory or intolerant);
    + Cohort B: ESA naive AND serum erythropoietin level >200 U/L
    - MDS oder MDS/MPN einschließlich MDS/MPN-RS-T, MDS/MPNu, aCML oder CMML
    - IPSS-R bis zu 3,5: sehr niedriges, niedriges oder mittleres Risiko (MDS); MDS/MPN < 10% Knochmarksblasten; CPSS-Score bei CMML: niedrig, intermediär
    - Symptomatische Anämie
    - Definierte Transfusionsstrategie
    - Keine verfügbare Option einer zugelassenen MDS-Therapie und Einstufung der vorherigen Behandlung mit Erythropoiese-stimulierenden Agenzien (ESA) wie folgt:
    + Kohorte A: ESA-exponiert (und refraktär oder intolerant);
    + Kohort B: ESA naïve UND Serum Erythropoietinlevel >200 U/L

    E.4Principal exclusion criteria
    Compliance with major study procedures
    1. Inability to swallow and retain oral medications (> 10 pills)
    2. Patient does not accept bone marrow sampling
    3. Patient does not accept up to weekly peripheral blood sampling

    Safety
    4. Eastern Cooperative Oncology Group (ECOG) Performance Status of ≥3
    5. Inacceptable organ function:
    a. Serum creatinine >2 × ULN or calculated creatinine clearance < 30 ml/min
    b. Aspartate aminotransferase (AST) > 2 × ULN OR alanine aminotransferase (ALT) > 2 × ULN
    c. Total bilirubin >2 × ULN
    i. exception >3 × ULN in patients with documented Gilbert’s syndrome

    Interfering treatments
    6. Prior treatment with azacitidine (injectable or oral) or decitabine.
    7. The patient medically requires treatment with the following drugs that are forbidden during the trial or was exposed to one of these 14 days before registration:
    a) Erythropoiesis stimulating agent (ESA) or luspatercept,
    b) Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony stimulating factor (GM-CSF)
    c) Lenalidomide
    d) Another investigational drug or device, or approved therapy for investigational use
    8. Iron chelation therapy
    9. Major surgery within 28 days prior to registration.

    Concomitant diseases
    10. Known human immunodeficiency virus (HIV) infection
    11. Active infectious hepatitis (Type B (HBV) or Type C (HCV)
    12. Hepatitis virus detectable within 6 months before registration in patients with a history of hepatitis
    13. History of other invasive malignancy, unless definitively treated with curative intent, provided it is deemed to be at low risk for recurrence by the treating physician
    14. Presence of an acute or chronic toxicity resulting from prior anti-cancer therapy that has not resolved to Grade ≤1 (except anemia and alopecia), as determined by NCI CTCAE v 5.0
    15. Known allergy or hypersensitivity to any component of the formulation of CA-4948 used in this study
    16. Severe cardiovascular disease

    Formal requirements
    17. Positive serum pregnancy test in women of childbearing potential
    18. Women of childbearing potential and men who partner with a woman of childbearing potential unwilling to use highly effective contraceptive methods for the duration of the study and for 90 days after the last dose of CA-4948
    19. Age under 18 years at registration
    20. Inability to provide written informed consent (Subject without legal capacity who is unable to understand the nature, scope, significance and consequences of this clinical trial)
    21. Simultaneous participation in another interventional clinical trial or participation in any clinical trial involving administration of an investigational medicinal product within 28 days prior registration

    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is erythroid response (HI-E) according to IWG 2018 criteria
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 4 cycles CA-4948 treatment.
    E.5.2Secondary end point(s)
    • HI-E response duration
    • Time to HI-E
    • Frequency of RBC transfusions in transfusion dependent patients
    • Neutrophil and platelet (HI-N and HI-P) responses according to IWG 2018 criteria
    E.5.2.1Timepoint(s) of evaluation of this end point
    - HI-E response duration: from time point of response determination (after start of treatment) until loss of response
    Time to HI-E: time from start of treatment until HI-E determination
    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 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 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 concerned15
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months42
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months42
    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: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 60
    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 state84
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 84
    F.4.2.2In the whole clinical trial 84
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Patients will be treated as usual after the individual's trial completion.
    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 Decision2021-08-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-06
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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