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    Summary
    EudraCT Number:2021-001838-19
    Sponsor's Protocol Code Number:KER050-MD-201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-11-17
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-001838-19
    A.3Full title of the trial
    A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
    Estudio de fase II, abierto, de aumento escalonado de la dosis de KER-050 para el tratamiento de la anemia en pacientes con síndromes mielodisplásicos (SMD) de riesgo muy bajo, bajo o intermedio
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
    Estudio de fase II, abierto, de aumento escalonado de la dosis de KER-050 para el tratamiento de la anemia en pacientes con síndromes mielodisplásicos (SMD) de riesgo muy bajo, bajo o intermedio
    A.4.1Sponsor's protocol code numberKER050-MD-201
    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 SponsorKeros 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 supportKeros Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKeros Therapeutics, Inc.
    B.5.2Functional name of contact pointCentral Contact Person
    B.5.3 Address:
    B.5.3.1Street Address99 Hayden Avenue, Suite 120, Building E
    B.5.3.2Town/ cityLexington
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicalstudies@kerostx.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 nameKER-050
    D.3.2Product code PRD8997233
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPRD8997233
    D.3.9.2Current sponsor codeKER-050
    D.3.9.3Other descriptive nameKER-050
    D.3.9.4EV Substance CodeSUB224243
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number67 to 83
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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).
    La anemia en pacientes con síndromes mielodisplásicos (SMD) de riesgo muy bajo, bajo o intermedio
    E.1.1.1Medical condition in easily understood language
    Myelodysplastic syndromes (MDS) are conditions that are observed when the blood-forming cells in the bone marrow become abnormal.
    Los síndromes mielodisplásicos (SMD) son afecciones que se observan cuando las células productoras de sangre en la médula ósea se vuelven anormales.
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002272
    E.1.2Term Anemia
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1: Dose Escalation
    • To evaluate the safety and tolerability of ascending doses of KER-050 in participants with very low, low, or intermediate risk MDS in order to determine the dose(s) that will be evaluated in Part 2 of the study.
    Part 2: Dose Confirmation
    • To confirm the safety and tolerability of the dose(s) selected in Part 1.
    Parte 1 de aumento gradual de la dosis:
    •Evaluar la seguridad y tolerabilidad del aumento escalonado de la dosis de KER-050 en participantes con SMD de riesgo muy bajo, bajo o intermedio para determinar las dosis que se evaluarán en la parte 2 del estudio.

    Parte 2 de confirmación de la dosis:
    •Confirmar la seguridad y tolerabilidad de las dosis seleccionadas en la parte 1.
    E.2.2Secondary objectives of the trial
    Secondary Objectives (Part 1 and Part 2)
    • To evaluate the PD effects of KER-050 on erythropoiesis in participants with very low, low, or intermediate risk MDS, separately for ring sideroblast (RS) positive and non-RS populations.
    • To evaluate the efficacy of KER-050 on anemia in participants with very low, low or intermediate risk MDS, separately for RS positive and non-RS populations.
    Objetivos secundarios (parte 1 y parte 2):
    •Evaluar los efectos farmacodinámicos (FD) de KER-050 en la eritropoyesis en participantes con SMD de riesgo muy bajo, bajo o intermedio, por separado para las poblaciones positivas para sideroblastos en anillo (SA) y sin sideroblastos en anillo (SSA).

    •Evaluar la eficacia de KER-050 en la anemia en participantes con SMD de riesgo muy bajo, bajo o intermedio, por separado para las poblaciones positivas para SA y SSA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease.
    2. < 5% blasts in bone marrow.
    3. Peripheral blood white blood cell (WBC) count < 13,000/µL.
    4. Anemia defined as:
    a. In LTB participants (defined as having received < 4 units of RBCs within 8 weeks), mean hemoglobin concentration < 10.0 g/dL of two measurements (one performed within 1 day prior to Cycle 1 Day 1 and the other performed 7-28 days prior, not influenced by RBC transfusion within 7 days of measurement) OR
    b. In HTB participants (defined as requiring > or = 4 units of RBCs for hemoglobin < or = 9.0 g/dL within 8 weeks)
    5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
    6. Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.
    E.4Principal exclusion criteria
    1. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
    2. Diagnosis of secondary MDS (ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
    3. Vitamin B12 deficiency.
    4. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
    5. Treatment within 28 days prior to Cycle 1 Day 1 with:
    a. Erythropoiesis stimulating agent (ESA) OR
    b. Granulocyte colony-stimulating factor (G-CSF) OR
    c. Granulocyte-macrophage colony-stimulating factor (GM-CSF)
    6. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
    7. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
    8. Treatment with another investigational drug or device or approved therapy for investigational use < or = 28 days prior to Cycle 1 Day 1, or if the half-life of the previous product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
    9. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
    10. Transferrin saturation < 15%.
    11. Ferritin < 50 µg/L.
    12. Folate < 4.5 nmol/L (< 2.0 ng/mL).
    13. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
    14. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
    15. Pregnant or lactating females.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint:
    Safety and tolerability as determined by the incidence of adverse events (AEs) and serious AEs (SAEs).
    E.5.1.1Timepoint(s) of evaluation of this end point
    105 Weeks
    105 semanas
    E.5.2Secondary end point(s)
    1. Maximum concentrations of KER-050 [Time Frame: Measured at multiple timepoints over the course of treatment from study day 1 to approximately 15 weeks]
    2. Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants. [Time Frame: Measured over the course of study, up to approximately 25 weeks from study day 1]
    a. In LTB participants, the proportion of participants who have a hemoglobin increase of ≥ 1.5 g/dL from Baseline for ≥ 14 days (in the absence of RBC transfusions).
    b. In HTB participants, the proportion of participants having a reduction of ≥ 50% or ≥ 4 RBC units transfused compared to pretreatment over an 8-week period.
    3. Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response [Time Frame: Measured over the course of study, up to approximately 25 weeks from study day 1]
    a. In LTB participants, the proportion of participants with an increase of ≥ 1.5 g/dL from pretreatment hemoglobin during any 8-week period on study compared to Baseline.
    b. In HTB participants, the proportion of participants having a reduction by ≥ 4 units of RBCs transfused (for a hemoglobin ≤ 9.0 g/dL) during any 8-week period on study, compared with the 8-week period prior to study Cycle 1 Day 1.
    4. Mean change from baseline in hemoglobin [Time Frame: Measured over the course of study, up to approximately 25 weeks from study day 1]
    5. Time to erythroid response and modified 2006 IWG HI-E response [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks)]
    6. Duration of erythroid response and modified 2006 IWG HI-E response [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks)]
    7. Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence ≥ 8 weeks [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks)]
    8. Change from Baseline in RBC counts and reticulocytes [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks)]
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints indicated above.
    Puntos de tiempo indicados arriba.
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tolerabilidad
    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 Yes
    E.8.1.7.1Other trial design description
    Dosis ascendente de 2ª parte
    2-part Ascending Dose
    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 concerned6
    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
    Australia
    Israel
    New Zealand
    Czechia
    France
    Germany
    Spain
    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 end of the study is defined as completion of the last participant completing the last visit or last procedure.
    El final del estudio se define como la finalización del último participante que completó la última visita o el último procedimiento.
    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 months9
    E.8.9.1In the Member State concerned days12
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days12
    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: 34
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 70
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 104
    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 continue with standard of care after trial participation ends.
    Después de que finalice la participación en el ensayo, los pacientes continuarán con la práctica clínica habitual.
    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 Decision2022-03-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-20
    P. End of Trial
    P.End of Trial StatusOngoing
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