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    Summary
    EudraCT Number:2021-000348-22
    Sponsor's Protocol Code Number:KER047-IR-201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-08-10
    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 number2021-000348-22
    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
    Estudio de fase II, abierto, de aumento gradual y expansión de la dosis de KER‐047 para el tratamiento de la anemia ferropénica refractaria al hierro (IRIDA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    IRIDA Study of KER-047
    Estudio IRIDA de 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 Address99 Hayden Avenue, Bldg. E, Suite 120,
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 617 3146297
    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 succinate
    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)
    Anemia ferropénica refractaria al hierro (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.
    La IRIDA es una forma rara y hereditaria de anemia ferropénica que se caracteriza por niveles elevados de hepcidina.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10022972
    E.1.2Term Iron deficiency anaemia
    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
    To evaluate the safety and tolerability of ascending doses of KER-047 in participants with iron-refractory iron deficiency anemia (IRIDA)
    Evaluar la seguridad y la tolerabilidad de dosis ascendentes de KER-047 en participantes con anemia ferropénica refractaria al hierro (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
    Objetivos secundarios:
    • Evaluar los efectos farmacodinámicos (FD) de KER-047 sobre el metabolismo del hierro en participantes con IRIDA.
    • Para evaluar la acumulación plasmática de KER-047 a lo largo del periodo de tratamiento.
    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 > or = 18 years of age, at the time of signing informed consent.
    2. 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 (VUS), likely pathogenic, pathogenic) per the Association for Clinical Genomic Science (ACGS).
    3. Serum TSAT at screening <15%.
    4. Participants receiving oral iron supplementation must be on a stable dose for > or = 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.
    Los participantes son elegibles para el estudio si se aplican todos los criterios siguientes:
    1. Hombre o mujer > o = 18 años de edad, en el momento de firmar el consentimiento informado.
    2. Diagnóstico de IRIDA basado en lo siguiente
    - Variante(s) documentada(s) del gen TMPRSS6 homocigótica(s) o heterocigótica(s) compuesta(s) de clase 3 o superior (variante de significado incierto (VUS), probablemente patogénica, patogénica) según la Association for Clinical Genomic Science (ACGS).
    3. TSAT sérico en el momento del cribado <15%.
    4. Los participantes que reciban suplementos de hierro por vía oral deben estar en una dosis estable durante > o = 4 semanas antes del Día 1, con un máximo de 60 mg/día de hierro elemental oral. No se permite el uso de hierro intravenoso (IV) durante el estudio.
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    Medical History
    1. Body mass index (BMI) >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 (NYHA) Class 3 or 4 heart failure.
    4. History of uncontrolled hyper- or hypothyroidism.
    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 (DVT) 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 (HIV), active infectious hepatitis B (HBV), or active infectious hepatitis C (HCV).
    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. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug.
    12. History of corneal dysplasia.
    13. History of any known inflammatory or immunodeficiency disorder (e.g., lupus, common variable immunodeficiency [CVID]).

    Treatment History
    14. Treatment with IV iron within 28 days prior to study entry
    15. 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.
    16. Receiving and plan to continue any disallowed medications listed in the protocol.
    17. 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
    18. Hemoglobin level ≥13.8 g/dL (8.56 mmol/L) (males) or > or = 12.1 g/dL (7.51 mmol/L) (females)
    19. Serum ferritin <50 or >700 µg/L
    20. Absolute lymphocyte count <1.00 x 10^9/L
    21. Absolute neutrophil count <1.50 x 10^9/L
    22. Estimated glomerular filtration rate (eGFR) by Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine equation < 45 mL/min/1.73 m2
    23. Alanine transaminase (ALT) or aspartate transaminase (AST) >2x upper limit of normal (ULN)
    24. C-reactive protein (CRP) > or =10 mg/L
    Los participantes están excluidos del estudio si se aplica alguno de los siguientes criterios:
    Historial médico
    1. Índice de masa corporal (IMC) >35 kg/m al cuadrado
    2. Cualquier infección activa que requiera terapia antibiótica parenteral en los 28 días anteriores al Día 1 o antibióticos orales en los 14 días posteriores al Día 1. Cualquier infección con >5 días de fiebre (>38,5 grados C) dentro de los 28 días anteriores al Día 1.
    3. Presencia de enfermedad cardíaca no controlada o insuficiencia cardíaca de clase 3 o 4 de la New York Heart Association (NYHA).
    4. Antecedentes de hiper o hipotiroidismo no controlado.
    5. Antecedentes de abuso de drogas o alcohol, según la definición del investigador, en los últimos 2 años.
    6. Antecedentes de accidente cerebrovascular, embolia arterial o trombosis venosa profunda (TVP) no resuelta en los 6 meses anteriores al día 1.
    7. Cirugía mayor en los 28 días anteriores al Día 1. Los participantes que hayan sido operados más de 28 días antes del Día 1 deben haberse recuperado satisfactoriamente para participar en el estudio, a juicio del investigador.
    8. Positividad conocida para el virus de la inmunodeficiencia humana (VIH), hepatitis infecciosa B (VHB) activa o hepatitis infecciosa C (VHC) activa.
    9. Cualquier tumor maligno que no haya estado en remisión y/o que haya requerido terapia sistémica, incluyendo radiación, quimioterapia, terapia hormonal o cirugía en el último año antes del Día 1.
    10. Antecedentes de trasplante de órganos sólidos o hematológicos.
    11. Antecedentes de reacciones alérgicas o anafilácticas graves o de hipersensibilidad a las proteínas recombinantes o a los excipientes del medicamento en investigación.
    12. Antecedentes de displasia corneal.
    13. Antecedentes de cualquier trastorno inflamatorio o de inmunodeficiencia conocido (por ejemplo, lupus, inmunodeficiencia variable común [IDCV]).

    Historial de tratamiento
    14. Tratamiento con hierro intravenoso en los 28 días anteriores a la entrada en el estudio
    15. Tratamiento con inhibidores de la bomba de protones (IBP). Los participantes que reciban IBP y suspendan su uso al menos 7 días antes del Día 1 podrán inscribirse.
    16. Recibir y planear continuar con cualquier medicamento no permitido en el protocolo.
    17. Tratamiento con otro fármaco o dispositivo en investigación, o terapia aprobada para uso en investigación ≤ 28 días antes del Día 1, o, si se conoce la vida media del producto anterior, dentro de 5 veces la vida media antes del Día 1, lo que sea más largo.

    Exclusiones de laboratorio
    18. Nivel de hemoglobina ≥13,8 g/dL (8,56 mmol/L) (varones) o > o = 12,1 g/dL (7,51 mmol/L) (mujeres)
    19. Ferritina sérica <50 o >700 µg/L
    20. Recuento absoluto de linfocitos <1,00 x 10^9/L
    21. Recuento absoluto de neutrófilos <1,50 x 10^9/L
    22. Tasa de filtración glomerular estimada (TFGe) según la ecuación de creatinina de la Colaboración en Enfermedad Renal Crónica (CKD-EPI) < 45 mL/min/1,73 m2
    23. Transaminasa de alanina (ALT) o transaminasa de aspartato (AST) >2 veces el límite superior de la normalidad (ULN)
    24. Proteína C reactiva (PCR) > o =10 mg/L
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    - Safety and tolerability as determined by the incidence of adverse events (AEs) and SAEs

    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
    - mean trough plasma KER-047 concentration (Ctrough)
    - Plasma KER-047 accumulation (Rac)
    Criterios de seguridad
    - Seguridad y tolerabilidad determinadas por la incidencia de eventos adversos (EA) y EAS

    Criterios de valoración farmacodinámicos
    - Concentración de hepcidina en plasma
    - Hierro sérico
    - Ferritina
    - Capacidad total de fijación del hierro (TIBC)
    - Saturación de transferrina (TSAT)
    - Contenido de hemoglobina reticulocitaria (RET-He/CHr)
    - Receptor de transferrina soluble (sTfR) e índice sTfR/log de ferritina
    - TSAT/hepcidina; ferritina/hepcidina

    Criterios de valoración farmacocinética
    - Concentración plasmática media de KER-047 (Ctrough)
    - Acumulación plasmática de KER-047 (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 14 days of treatment in Part 1 and after 28 or 56 days of treatment in Part 2.
    Pharmacokinetic endpoints will be assessed on Days 8 and 15 in Part 1, and 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 in Part 2.
    Las evaluaciones de seguridad se resumirán en la línea de base y en cada punto de tiempo observado.
    Los criterios de valoración farmacodinámicos y exploratorios se evaluarán como cambio respecto a la situación basal después de 14 días de tratamiento en la Parte 1 y después de 28 o 56 días de tratamiento en la Parte 2.
    Los criterios de valoración farmacocinéticos se evaluarán los días 8 y 15 en la Parte 1, y los días 8, 15, 22 y 29 si el tratamiento fue de 28 días o los días 8, 15, 22, 29, 36, 43, 50 y 57 si el tratamiento fue de 56 días en la Parte 2.
    E.5.2Secondary end point(s)
    Please see above
    Véase arriba
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please see above
    Véase 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 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
    Última visita del último paciente
    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 months7
    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 months9
    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 state2
    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
    Ninguno
    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 Decision2022-11-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-23
    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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