A Study of Elritercept Alone or Together With Ruxolitinib in Adults With Myelofibrosis

NCT05037760 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: KER050-MF-3012023-507468-38-00
• Study Type: interventional
• Target: 135
• Locations: 7 countries
• Sites: 46

Brief Summary

The main aim of this study is to learn how safe elritercept is and how well it is tolerated when taken alone and in combination with the JAK inhibitor, ruxolitinib. Other aims are to learn about the effects of elritercept on the signs and symptoms of MF when taken with or without ruxolitinib and to learn how elritercept affects the body, how the body processes elritercept, and the effects of elritercept on anemia when taken with or without ruxolitinib The study will also check on how safe elritercept is and how well it is tolerated.

Conditions

Myelofibrosis

Keywords

Thrombocytopenia; Anemia; Red blood cells; Bone marrow; KER-050; drug therapy; elritercept

Outcome Measures

Primary Outcomes (2)

• Part 1: Number of Participants with Adverse Events (AEs), Dose Limiting Toxicities (DLTs), Severe Adverse Events (AEs) and Serious Adverse Events (SAEs)

  AE:any untoward medical occurrence (MO) in clinical study participant administered medicinal product not necessarily having causal relationship with treatment.Severe AE medically significant AE but not immediately life-threatening;may result in hospitalization/ prolonged hospital stay;disability.SAE:any untoward MO that,at any dose results in death,is life-threatening,requires in-patient hospitalization/ prolongation of existing hospitalization,results in persistent or significant disability/incapacity,is congenital anomaly/birth defect,is medically important event.DLT: any following safety events-death,Grade 4 neutropenia/ thrombocytopenia \>7 days,Grade 3 thrombocytopenia with bleeding,Neutropenic fever,assessments meeting Hy's law,Grade ≥3 nonhematologic treatment-emergent adverse events(TEAEs),elevated hemoglobin(Hgb)/ platelet count requiring dose modification, other safety event considered by Safety Review Committee(SRC) to be significant to warrant categorization as DLT.

  From signing of the informed consent form (ICF) through 30 days after the last dose of study drug (approximately 8 years)

• Part 2 and Long-Term Extension: Number of Participants with Adverse Events (AEs), Severe AEs and SAEs

  An AE is defined as any untoward medical occurrence in a clinical study participant administered a medicinal product that does not necessarily have a causal relationship with this treatment. Severe AE is defined as an AE which is medically significant but not immediately life-threatening; may result in hospitalization or prolonged hospital stay; disabling. An SAE is any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event.

  From signing of the ICF through 30 days after the last dose of study drug, (approximately 8 years)

Secondary Outcomes (14)

• Percentage of Participants with Progression to Acute Myeloid Leukemia (AML)

  From signing of the ICF through 30 days after the last dose of study drug, (approximately 8 years)

• Percentage of Participants with Progression to Accelerated MF

  From signing of the ICF through 30 days after the last dose of study drug, (approximately 8 years)

• Percentage of Participants with Red Blood Cell (RBC) Transfusion Independence Over a Period of ≥12 Consecutive Weeks

  Up to Week 24

• Percentage of Participants with RBC Transfusions From Baseline Over a Period of ≥12 Consecutive Weeks

  Up to Week 24

• Percentage of Participants with Mean Haemoglobin (Hgb) Increase From Baseline Over a Period of ≥12 Consecutive Weeks

  Up to Week 24

Study Arms (6)

Arm 1A: Elritercept

EXPERIMENTAL

Participants with anemia who have discontinued Janus Kinase (JAK) inhibitor(s) or are intolerant or ineligible for JAK inhibitor(s) will be administered escalating doses of elritercept, starting at 0.75 milligrams per kilograms (mg/kg) followed by 1.5 mg/kg and 4.5 mg/kg subcutaneously (SC), every 4 weeks for 13 cycles for a total Treatment Period of 52 weeks. Each cycle is 28 days.

Drug: Elritercept

Arm 1B: Elritercept + Ruxolitinib

EXPERIMENTAL

Participants with anemia who have been receiving ruxolitinib for ≥8 weeks prior to Cycle 1 Day 1 (C1D1) and are on a stable dose for ≥4 weeks prior to C1D1 will be administered escalating doses of elritercept, starting at 0.75 mg/kg and followed by 1.5 mg/kg and 4.5 mg/kg, SC, every 4 weeks for 13 cycles in combination with ruxolitinib therapy for a total Treatment Period of 52 weeks. Each cycle is 28 days.

Drug: Elritercept; Drug: Ruxolitinib

Arm 2A: Elritercept

EXPERIMENTAL

Participants with with anemia who have discontinued JAK inhibitor(s) or are intolerant or ineligible for JAK inhibitor(s) will be administered elritercept, 3.75 mg/kg, SC, every 4 weeks for 13 cycles for a total Treatment Period of 52 weeks. Each cycle is 28 days.

Drug: Elritercept

Arm 2B: Elritercept + Ruxolitinib

EXPERIMENTAL

Participants with anemia who have been receiving ruxolitinib for ≥8 weeks prior to C1D1 and are on a stable dose for ≥4 weeks prior to C1D1 will be administered elritercept, 3.75 mg/kg, SC, every 4 weeks for 13 cycles in combination with ruxolitinib therapy for a total Treatment Period of 52 weeks. Each cycle is 28 days.

Drug: Elritercept; Drug: Ruxolitinib

Experimental: Arm 2C: Elritercept (Brazil Only)

EXPERIMENTAL

Participants from Brazil with anemia who have received no prior treatment with JAK inhibitor(s) and have no access to JAK inhibitor therapy will be administered elritercept, 3.75 mg/kg, SC, every 4 weeks for 13 cycles for a total Treatment Period of 52 weeks. Each cycle is 28 days.

Drug: Elritercept

Long-Term Extension

EXPERIMENTAL

Participants from Arms 1A, 1B, 2A, 2B and 2C benefiting from the continued elritercept treatment as a monotherapy or in combination with ruxolitinib can continue to receive elritercept in this long-term extension phase until elritercept becomes commercially available or until elritercept is no longer being developed for the treatment of MF.

Drug: Elritercept; Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib tablet.

Arm 1B: Elritercept + Ruxolitinib; Arm 2B: Elritercept + Ruxolitinib; Long-Term Extension

Elritercept DRUG

Elritercept SC injection.

Also known as: KER-050, TAK-226

Arm 1A: Elritercept; Arm 1B: Elritercept + Ruxolitinib; Arm 2A: Elritercept; Arm 2B: Elritercept + Ruxolitinib; Experimental: Arm 2C: Elritercept (Brazil Only); Long-Term Extension

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.
• In the opinion of the Investigator, the participant is able and willing to comply with the requirements of the protocol (e.g., all study procedures, return for follow-up visits).
• Male or female greater than equal to (≥)18 years of age, at the time of signing informed consent.
• Eastern Cooperative Oncology Group (ECOG) performance score lesser than equal to (≤)2.
• Life expectancy ≥12 months per Investigator assessment.
• Confirmed diagnosis of primary myelofibrosis (PMF) (prefibrotic or overtly fibrotic) according to the 2016 World Health Organization (WHO) criteria, post-polycythemia vera myelofibrosis (PV MF), or post-essential thrombocythemia myelofibrosis (ET MF) according to the 2008 International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria.
• Anemia, defined as:
• Having received ≥6 units of RBC transfusion for Hgb ≤8.5 g/dL in the 12 weeks prior to the planned C1D1, including ≥1 unit of RBC transfusion in the 28 days prior to C1D1; or
• Having ≥3 evaluable Hgb measurements at less than (\<)10.0 g/dL including ≥1 evaluable Hgb measurement assessed 8 to 13 weeks prior to C1D1. Participants receiving RBC transfusions but not meeting criterion "a." may enroll under criterion "b." following the below parameters:
• All pre-transfusion Hgb values (defined as a Hgb assessed within the 3 days prior to a transfusion) should be recorded, and ≥1 pre-transfusion Hgb value is required.
• Hgb values collected within the 28 days following a transfusion will not be considered evaluable unless qualifying as a pre-transfusion Hgb; in cases where multiple transfusions are given in succession due to poor Hgb response, only the first pre-transfusion Hgb will be considered evaluable.
• Arm-specific criteria:
• Arms 1A and 2A:
• Previously treated with JAK inhibitor(s) and, per the Investigator, discontinued due to one of the following reasons:
• Relapsed disease following treatment with JAK inhibitor(s)

You may not qualify if:

• Medical History:
• 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.
• Presence of the following cardiac conditions:
• New York Heart Association Class 3 or 4 heart failure
• QTcF (QT interval corrected by Fridericia's formula) \>500 milliseconds (msec) on the screening or C1D1 electrocardiogram (ECG; mean of 3 measurements)
• Uncontrolled clinically significant arrhythmia (participants with rate-controlled atrial fibrillation are not excluded)
• Acute myocardial infarction or unstable angina pectoris ≤6 months prior to C1D1
• Body mass index (BMI) ≥40 kilograms per meter square (kg/m\^2).
• Presence of uncontrolled hypertension, defined as systolic blood pressure ≥160 millimeters of mercury (mmHg) or diastolic blood pressure ≥100 mmHg despite adequate treatment.
• History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.
• History of stroke, deep venous thrombosis, or arterial embolism within 6 months prior to C1D1.
• Major surgery within 28 days prior to C1D1. Participants must have completely recovered from any previous surgery prior to C1D1 in the opinion of the Investigator.
• Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B with positive viral load (hepatitis B virus \[HBV\] deoxyribonucleic acid \[DNA\]), or active infectious hepatitis C with positive viral load (hepatitis C virus \[HCV\] ribonucleic acid \[RNA\]). Participants without a known positive history of HIV, HBV, and/or HCV do not require further testing, unless testing is mandated per local guidelines.
• Any malignancy other than PMF, post-ET MF, or post-PV MF that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or biologic therapy, within 1 year prior to C1D1. In situ cancers, squamous cell and basal cell carcinomas, and monoclonal gammopathy of unclear significance are allowed at the discretion of the Investigator.
• History of solid organ or hematological transplantation.

Sponsors & Collaborators

• Takeda: lead

Study Sites (46)

Concord Hospital

Concord, New South Wales, Australia

RECRUITING

The Tweed Hospital

Tweed Heads, New South Wales, 2485, Australia

RECRUITING

Flinders Medical Centre

Woodville South, South Australia, 5042, Australia

RECRUITING

St. Vincents Hospital Melbourne

Fitzroy, Victoria, 3355, Australia

RECRUITING

Royal Melbourne Hospital

Melbourne, Victoria, 3050, Australia

RECRUITING

Ballarat Oncology & Haematology Service

Wendouree, Victoria, 3355, Australia

RECRUITING

Hospital de Clinicas de Porto Alegre

Porto Alegre, Brazil

RECRUITING

IMV-Pesquisa Cardiologica Sociedade Simples

Porto Alegre, Brazil

RECRUITING

Albert Einstein Sociedade Beneficente Israelita Brasiliera

São Paulo, Brazil

RECRUITING

Hospital Beneficencia Portuguesa de Sao Paulo

São Paulo, Brazil

RECRUITING

Hospital Das Clinicas Da Faculdade de Medicina Da U S P

São Paulo, Brazil

RECRUITING

Instituto de Ensino e Pesquisas Sao Lucas

São Paulo, Brazil

RECRUITING

CHU Amiens - Hopital Sud

Amiens, France

COMPLETED

Hopital Morvan

Brest, France

COMPLETED

Hopital Prive Sevigne

Cesson-Sévigné, France

COMPLETED

Centre Hospitalier Lyon Sud

Lyon, France

RECRUITING

Institut de Cancerologie du Gard

Nîmes, France

RECRUITING

Hopital de la Source - CHR Orleans

Orléans, France

COMPLETED

Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

Bari, Italy

RECRUITING

Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi

Bologna, Italy

RECRUITING

Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

Brescia, Italy

RECRUITING

Azienda Ospedaliera Universitaria Careggi

Florence, Italy

RECRUITING

Ospedale Policlinico San Martino

Genova, Italy

RECRUITING

ASST Grande Ospedale Metropolitano Niguarda, Niguarda Cancer Center

Milan, Italy

RECRUITING

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Milan, Italy

COMPLETED

Arcispedale S. Maria Nuova Azienda Ospedaliera di Reggio Emilia

Reggio Emilia, Italy

RECRUITING

Azienda Ospedaliera Universitaria Policlinico Umberto I

Roma, Italy

RECRUITING

Azienda Socio Sanitaria Territoriale Sette Laghi

Varese, Italy

RECRUITING

Azienda Ospedaliera Universitaria Integrata Verona

Verona, Italy

RECRUITING

Gachon University Gil Medical Center

Incheon, South Korea

COMPLETED

Samsung Medical Center

Seoul, South Korea

COMPLETED

Seoul St. Marys Hospital, The Catholic University of Korea

Seoul, South Korea

RECRUITING

Soonchunhyang University Seoul Hospital

Seoul, South Korea

RECRUITING

ICO Badalona - Hospital Universitari Germans Trias i Pujol

Badalona, Spain

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, Spain

RECRUITING

Hospital Universitario La Princesa

Madrid, Spain

RECRUITING

Hospital Universitario de Salamanca

Salamanca, Spain

RECRUITING

Hospital Clinico Universitario de Valencia

Valencia, Spain

RECRUITING

Hospital QuironSalud de Zaragoza

Zaragoza, Spain

RECRUITING

United Lincolnshire Hospitals NHS Trust - Pilgrim Hospital

Boston, United Kingdom

RECRUITING

St James Hospital,Leeds

Leeds, United Kingdom

RECRUITING

Guys Hospital

London, United Kingdom

RECRUITING

Hammersmith Hospital

London, United Kingdom

RECRUITING

University College London

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis; Thrombocytopenia; Anemia

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 23, 2021
• First Posted: September 8, 2021
• Study Start: December 16, 2021
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): February 28, 2030
• Last Updated: February 5, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

ES (7); AU (6); BR (6); FR (6); KR (4); IT (12); GB (5)