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    Summary
    EudraCT Number:2021-001838-19
    Sponsor's Protocol Code Number:KER050-MD-201
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-11-02
    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 ConcernedCzechia - SUKL
    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)
    Otevřené klinické hodnocení fáze 2 se vzestupnou dávkou přípravku KER-050 k léčbě anémie u pacientů s myelodysplastickým syndromem (MDS) s velmi nízkým, nízkým nebo středním rizikem.
    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)
    Otevřené klinické hodnocení fáze 2 se vzestupnou dávkou přípravku KER-050 k léčbě anémie u pacientů s myelodysplastickým syndromem (MDS) s velmi nízkým, nízkým nebo středním rizikem.
    A.4.1Sponsor's protocol code numberKER050-MD-201
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1294-3670
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation PlanP/407/2022
    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 Address1050 Waltham Street, Suite 302
    B.5.3.2Town/ cityLexington
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.6E-mailker050-md-201@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 nameelritercept
    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 INNelritercept
    D.3.9.1CAS number 2727114-26-1
    D.3.9.2Current sponsor codeKER-050
    D.3.9.3Other descriptive nameelritercept
    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 number75-100
    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).
    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.
    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 27.0
    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 10005329 - Blood and lymphatic 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
    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.
    Long-Term Extension
    •To evaluate the long-term safety and tolerability of KER-050 in participants with very low, low, or intermediate risk MDS in the Long-term Extension (LTE).
    E.2.2Secondary objectives of the trial
    Secondary Objectives (All Parts):
    Safety Objective:
    •To evaluate the progression to higher risk MDS or acute myeloid leukemia (AML)

    Efficacy Objective:
    •To evaluate the efficacy of KER-050 on anemia in participants with very low, low, orintermediate risk MDS, separately for ring sideroblast (RS)-positive and non-RS populations

    Pharmacodynamic Objective:
    •To evaluate the pharmacodynamic (PD) effects of KER-050 on erythropoiesis in participantswith very low, low, or intermediate risk MDS, separately for RS-positive and non-RSpopulations
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of MDS (Parts 1 and 2) according to WHO classification that meets International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.
    2. < 5% blasts in bone marrow during the Pretreatment Period.
    3. Peripheral blood white blood cell (WBC) count < 13,000/µL during the Pretreatment Period.
    4. Anemia defined as:
    a. In LTB participants, having received 1 to 3 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks of the Pretreatment Period 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. Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and/or antifungals for neutropenia are allowed.
    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 with ESA within 56 days prior to C5D1.
    6. Prior or concurrent chronic treatment with G-CSF or GM-CSF.
    7. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
    8. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
    9. 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.
    10. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
    11. Transferrin saturation < 15%.
    12. Ferritin < 50 µg/L.
    13. Folate < 4.5 nmol/L (< 2.0 ng/mL).
    14. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
    15. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
    16. Pregnant or lactating females.

    Medical History
    •Diagnosis of MDS with deletion of chromosome 5q (Del5q).
    •Presence of uncontrolled heart disease or New York Heart Association Class III or IV heart failure.
    •Presence of uncontrolled hypertension (Grade ≥ 2 high blood pressure).
    •History of stroke, deep venous thrombosis, or arterial embolism within 6 months prior to C1D1(or C5D1 for the Part 1 Extension).
    •Any malignancy other than MDS that has not been in remission and/or has required systemictherapy 1 year prior to C1D1 (or C5D1 for the Part 1 Extension).
    •Diagnosis of secondary MDS.
    •History of solid organ or hematological transplantation
    •Body mass index ≥ 40 kg/m2.

    Treatment History
    •Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
    •Treatment with ESA within 8 weeks prior to C1D1 (or C5D1 for the Part 1 Extension).
    •Prior or concurrent chronic treatment with granulocyte colony-stimulating factor (G-CSF) orgranulocyte-macrophage colony-stimulating factor (GM-CSF), for reasons other than thetreatment of MDS.
    E.5 End points
    E.5.1Primary end point(s)
    Part 1 and Part 2:
    •Safety and tolerability as determined by the incidence of treatment-emergent adverse events(TEAEs) and serious adverse events (SAEs).

    Long-term Extension:
    •Safety and tolerability as determined by the incidence of TEAEs and SAEs over time
    E.5.1.1Timepoint(s) of evaluation of this end point
    105 Weeks unless participants elect to continue in the Long-term Extension (LTE)
    E.5.2Secondary end point(s)
    1. Incidence of progression to higher risk MDS or AML per World Health Organization (WHO) 2016 criteria.
    2. Proportion of low transfusion burden (LTB) and high transfusion burden (HTB) participants who achieve red blood cell (RBC) transfusion independence (TI) ≥ 8 weeks, overall and by RS status.
    3. Proportion of participants who achieve modified 2006 International Working Group (IWG) Hematologic Improvement-Erythroid (HI E) response overall and by RS status, where:
    - In LTB participants and participants with non-transfused anemia, response is defined as a mean hemoglobin (Hgb) increase of ≥ 1.5 g/dL from Baseline during any 8 week period during the treatment period.
    - In HTB participants, response is defined as a reduction by ≥ 4 units of RBCs transfused during any 8-week period on study compared with the 8-week period prior to study Cycle 1 Day 1 (C1D1).
    4. Proportion of participants who achieve overall erythroid response, defined as either HI-E or TI over any 8-week period, overall and by RS status.
    5. Proportion of participants who achieve erythropoietic improvement overall and by RS status, where:
    - In low-transfusion burden (LTB) participants and participants with non-transfused anemia, improvement is defined as a mean hemoglobin (Hgb) increase of ≥ 1.5 g/dL from Baseline for ≥ 14 days (in the absence of red blood cell [RBC] transfusions).
    - In high transfusion burden (HTB) participants, improvement is defined as a reduction of ≥ 50% or ≥ 4 RBC units transfused compared to pretreatment over an 8-week period.
    6. Mean change from baseline in hemoglobin [Time Frame: Measured over the course of study, up to approximately 25 weeks from study day 1 unless participants elect to continue on in the LTE].
    7. Time to HI-E response [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks unless participants elect to continue on in the LTE)].
    8. Duration of HI-E response [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks unless participants elect to continue on in the LTE)].
    9. Time to TI response.
    10. Duration of TI response.
    11. Proportion of LTB and HTB participants who achieve TI ≥ 12 weeks, 16 weeks, 24 weeks, 48 weeks.
    12. Change from baseline of red cell parameters, including reticulocyte count, mean corpuscular volume, mean corpuscular hemoglobin, and reticulocyte count, mean corpuscular volume, mean corpuscular volume, mean corpuscular hemoglobin and reticulocyte cell Hgb by visit. [Time Frame: From treatment initiation to End of Study visit (approximately 25 weeks unless participants elect to continue on in the LTE)].

    Safety Endpoint:
    •Incidence of progression to higher risk MDS or AML per World Health Organization (WHO)2016 criteria

    Efficacy Endpoints:
    •Proportion of low transfusion burden (LTB) and high transfusion burden (HTB) participants who achieve red blood cell (RBC) transfusion independence (TI) ≥ 8 weeks, overall and by RSstatus
    •Proportion of participants who achieve modified 2006 International Working Group (IWG)Hematologic Improvement-Erythroid (HI-E) response overall and by RS status, where:−In LTB participants and participants with non-transfused anemia, response is definedas a mean hemoglobin (Hgb) increase of ≥ 1.5 g/dL from Baseline during any 8-weekperiod during the treatment period
    −In HTB participants, response is defined as a reduction by ≥ 4 units of RBCstransfused during any 8-week period on study compared with the 8-week period priorto study Cycle 1 Day 1 (C1D1)

    •Proportion of participants who achieve overall erythroid response, defined as either HI-E or TIover any 8-week period, overall and by RS status
    •Proportion of participants who achieve erythropoietic improvement overall and by RS status,where:
    −In LTB participants and participants with non-transfused anemia, improvement isdefined as a mean Hgb increase of ≥ 1.5 g/dL from Baseline for ≥ 14 days (in theabsence of RBC transfusions)
    −In HTB participants, improvement is defined as a reduction of ≥ 50% or ≥ 4 RBC unitstransfused compared with pretreatment over an 8-week period
    •Mean change from Baseline in Hgb by visit
    •Time to HI-E response
    •Duration of HI-E response
    •Time to TI response
    •Duration of TI response
    •Proportion of LTB and HTB participants who achieve TI ≥ 12 weeks, 16 weeks, 24 weeks, 48weeks

    Pharmacodynamic Endpoint:
    •Change from Baseline of red cell parameters, including reticulocyte count, mean corpuscularvolume, mean corpuscular hemoglobin, and reticulocyte cell Hgb by visit.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints indicated above.
    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
    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
    2-part Ascending Dose and Long-term Extension
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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
    New Zealand
    Australia
    Israel
    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
    The End of Trial is defined as when all of the participants have completed the treatment period in Part 1, Part 2, or the LTE. Participants will continue to be followed in the Survival Follow-up Period until all participants have withdrawn consent, are declared lost to follow-up, have died, or until study closure, whichever is earliest.
    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: 76
    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 59
    F.4.2.2In the whole clinical trial 141
    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.
    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 Decision2022-03-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-03
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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