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    Summary
    EudraCT Number:2021-000348-22
    Sponsor's Protocol Code Number:KER047-IR-201
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-04-20
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-000348-22
    A.3Full title of the trial
    A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    IRIDA Study of KER-047
    A.4.1Sponsor's protocol code numberKER047-IR-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 pointClinical Project Manager
    B.5.3 Address:
    B.5.3.1Street Address1050 Waltham Street, Suite 302​
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16178931255
    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-047
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNPRD8828729
    D.3.9.2Current sponsor codeKER-047 (Keros)
    D.3.9.3Other descriptive name7-Fluoro-6-methoxy-4-{6-[4-(4-methyl-piperazin-1-yl)-piperidin-1-yl]-pyrazolo[1,5-a]pyrimidin-3-yl}-quinoline
    D.3.9.4EV Substance CodeSUB222567
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number25 to 75
    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
    Iron-Refractory Iron-Deficiency Anemia (IRIDA)
    E.1.1.1Medical condition in easily understood language
    IRIDA is rare, inherited form of iron deficiency anemia that is characterized by high hepcidin levels.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of ascending doses of KER-047 in participants with iron-refractory iron deficiency anemia (IRIDA)
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    • To evaluate the pharmacodynamic (PD) effects of KER-047 on iron metabolism in participants with IRIDA
    • To evaluate plasma accumulation of KER-047 across the treatment period
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants are eligible for the study if all of the following criteria apply:
    1. Male or female ≥ 18 years of age, at the time of signing informed consent.
    2. Confirmed diagnosis of IRIDA based on the following:
    - Documented homozygous or compound heterozygous TMPRSS6 gene variant(s) of Class 3 or greater (variant of uncertain significance, likely pathogenic) per the Association for Clinical Genomic Science (ACGS) guideline.
    3. Serum TSAT at screening <15%.
    4. Participants receiving oral iron supplementation must be on a stable dose for ≥4 weeks prior to Day 1, with a maximum of 60 mg/day of oral elemental iron. Intravenous (IV) iron is not permitted during the study.
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    Medical History
    1. Body mass index >35 kg/m squared
    2. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Day 1 or oral antibiotics within 14 days of Day 1. Any infection with >5 days of fever (>38.5 degrees C) within 28 days prior to Day 1.
    3. Presence of uncontrolled heart disease or New York Heart Association Class 3 or 4 heart failure.
    4. History or presence at screening of an uncontrolled chronic disease.
    5. History of drug or alcohol abuse, as defined by the investigator, within the past 2 years.
    6. History of stroke, arterial embolism, or unresolved deep venous thrombosis within 6 months prior to Day 1.
    7. Major surgery within 28 days prior to Day 1. Participants who had surgery more than 28 days prior to Day 1 must have recovered satisfactorily to participate in the study, in the opinion of the Investigator.
    8. Known positive for human immunodeficiency virus, active infectious hepatitis B, or active infectious hepatitis C.
    9. Any malignancy that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery within the last year prior to Day 1.
    10. History of solid organ or hematological transplantation.
    11. Have had a fracture within 4 weeks of D1.
    12. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the IMP.

    Treatment History
    13. Treatment with IV iron within 28 days prior to Day 1.
    14. Receiving treatment with proton pump inhibitors (PPIs). Participants receiving PPIs who discontinue use at least 7 days prior to Day 1 are permitted to enroll.
    15. Receiving and plan to continue any disallowed medications.
    16. Treatment with another investigational drug or device, or approved therapy for investigational use ≤ 28 days prior to Day 1, or, if the half-life of the previous product is known, within 5 times the half-life prior to Day 1, whichever is longer.

    Laboratory Exclusions
    17. Hemoglobin level ≥13.8 g/dL (8.56 mmol/L) (males) or ≥12.1 g/dL (7.51 mmol/L) (females)
    18. Serum ferritin <50 or >700 µg/L
    19. Absolute lymphocyte count <1.00 x 10^9/L
    20. Absolute neutrophil count <1.50 x 10^9/L
    21. Estimated glomerular filtration rate by Chronic Kidney Disease-Epidemiology Collaboration creatinine equation < 45 mL/min/1.73 m2
    22. Alanine transaminase or aspartate transaminase >2x upper limit of normal (ULN)
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    - Safety and tolerability as determined by the incidence of treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), treatment-related adverse events (AEs), and
    discontinuations due to AEs; and change from baseline in clinical laboratory values, vital signs, and electrocardiogram (ECG)

    Pharmacodynamic Endpoints
    - Hepcidin concentration in plasma
    - Serum iron
    - Ferritin
    - Total iron binding capacity (TIBC)
    - Transferrin saturation (TSAT)
    - Reticulocyte hemoglobin content (RET-He/CHr)
    - Soluble transferrin receptor (sTfR) and sTfR/log ferritin index
    - TSAT/hepcidin; ferritin/hepcidin

    Pharmacokinetic Endpoints
    The following parameters for KER-047 and any metabolites of interest will be assessed:
    - Trough plasma concentration (Ctrough)
    - Plasma accumulation (Rac)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety assessments will be summarized at Baseline and at each observed time point.
    Pharmacodynamic and exploratory endpoints will be evaluated as change from baseline after 28 days of treatment in Part 1 and after 28 or 56 days of treatment in Part 2.
    Pharmacokinetic endpoints will be assessed in Part 1 on Days 8, 15, 22 and 29 , and in Part 2 on Days 8, 15, 22, and 29 if treatment was 28 days or Days 8, 15, 22, 29, 36, 43, 50, and 57 if treatment was 56 days.
    E.5.2Secondary end point(s)
    Please see above
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please see 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 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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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 years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days29
    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: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 12
    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
    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 Decision2021-04-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-10
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-06-09
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