MyelofibrosisPhase 2NCT05037760
A Study of Elritercept Alone or Together With Ruxolitinib in Adults With Myelofibrosis
A Phase 2 study testing Elritercept and Ruxolitinib in people with Myelofibrosis, run by Takeda at 46 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- Elritercept and Ruxolitinib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 135 participants
- Where
- 46 sitesin 7 countries
- Study dates
- 16 Dec 2021to 28 Feb 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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)
- Refractory to treatment with JAK inhibitor(s)
- Intolerance to treatment with JAK inhibitor(s)
- Participant no longer met risk/benefit ratio to continue JAK inhibitor(s) OR
- Participant with prognostic score of intermediate-1 or higher per Dynamic International Prognostic Scoring System (DIPSS) and is ineligible for JAK inhibitor(s) in the opinion of the Investigator
- Participants previously treated with JAK inhibitor(s) must have discontinued JAK inhibitor therapy ≥8 weeks before C1D1
- Arms 1B and 2B:
- Has been receiving ruxolitinib prescribed for a diagnosis of PMF (prefibrotic or overtly fibrotic), post-PV MF, or post-ET MF for ≥8 weeks prior to C1D1 and on a stable dose for ≥4 weeks prior to C1D1. In Arm 2B only, at least 10 participants should have been on ruxolitinib for <6 months prior to C1D1.
- Meets ≥1 of the following criteria in the opinion of the Investigator:
- Current ruxolitinib treatment is considered to be providing insufficient control of the disease
- The participant's cytopenias are limiting the participant's ruxolitinib dose intensity
- The participant's disease is symptomatic and warrants additional therapy
- Arm 2C (Brazil only):
- No prior treatment with JAK inhibitor(s) and no access to JAK inhibitor therapy as determined by the Investigator
- Spleen volume ≥ 450 cubic centimeter (cm^3) as assessed by CT or MRI collected during the pretreatment period and/or
- Myelofibrosis Symptom Assessment Form Total Symptom Score (MF-SAF-TSS) meeting at least one of the following criteria during the pretreatment period:
- 2 symptoms with average score ≥ 3
- Average total symptom score ≥ 10
- Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception as described in the protocol.
Who cannot join
- 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)
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- 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.
- History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the investigational drug, or ruxolitinib for participants enrolling in Arm 1B or 2B.
- Diagnosis of hemolytic anemia, active bleeding, hemoglobinopathies, or congenital disorders as a cause of the participant's anemia.
- History of intracranial hemorrhage (any grade).
- National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade ≥2 bleeding events within the 3 months prior to C1D1.
- Receipt of an RBC or platelet transfusion for any reason(s) or combination of reasons other than underlying MF within the 12 weeks prior to C1D1. If a participant requires a transfusion for an unanticipated reason during the Pretreatment Period, a prolonged screening period may be considered after discussion with the Medical Monitor.
- Treatment History:
- Prior treatment with luspatercept, sotatercept, or other commercially available or investigational transforming growth factor-beta (TGF-β) inhibitors (all arms).
- Treatment within 28 days prior to C1D1 with:
- Erythropoiesis-stimulating agent (ESA)
- Granulocyte colony-stimulating factor (G-CSF)
- Granulocyte-macrophage colony-stimulating factor (GM-CSF)
- Thrombopoietin (TPO) agonists
- Immunomodulator imide drugs (IMiDs) (e.g., thalidomide, pomalidomide, lenalidomide)
- Interferon
- Hydroxyurea
- Steroids at doses exceeding corticosteroid equivalent of 10 mg/day prednisone
- Newly initiated iron chelation therapy within the 8 weeks prior to C1D1. Stable doses of iron chelators are allowed if prescribed per label.
- Vitamin B12 and/or folate therapy initiated within 4 weeks before randomization. Participants on stable replacement doses for ≥4 weeks and without concurrent vitamin B12 or folate deficiency are allowed.
- Treatment with another investigational drug or device or approved therapy for the treatment of MF or anemia in MF ≤28 days prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.
- For Arms 1B and 2B (participants receiving ruxolitinib), initiation of treatment with strong cytochrome P450 (CYP)3A4 inhibitors within 2 weeks prior to C1D1. Participants receiving CYP3A4 inhibitors/inducers as concomitant therapy with ruxolitinib in accordance with ruxolitinib local prescribing information may continue to receive such therapies in this study.
- Laboratory Exclusions (during screening):
- Bone marrow aspirate blast percentage >5 percent (%)
- In the event of a non-evaluable pretreatment bone marrow aspirate expected to be due to marrow fibrosis, participants may be enrolled without bone marrow aspirate blast percentage data if all other eligibility criteria are met. Historical bone marrow data may be requested to support confirmation of diagnosis.
- Peripheral blood blast percentage ≥10%
- Platelet count <25 × 10^9 per liter (10^9/)L or >450 × 10^9/L
- Persistent Hgb <7 g/dL despite RBC transfusions
- Transferrin saturation <15%
- Ferritin <50 nanograms per mililiters (ng/mL)
- Folate <4.5 nanomoles per liter (nmol/L) (<2.0 picograms per liter (pg/L))
- Vitamin B12 <148 picomoles per liter (pmol/L) (<200 picograms per milliliter (pg/mL))
- Estimated glomerular filtration rate <30 milliliters per minute per 1.73 square meter (mL/min/1.73 m^2) (as determined by the Chronic Kidney Disease Epidemiology Collaboration equation)
- Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) >3 × upper limit of normal (ULN)
- Total bilirubin >2 × ULN
- International normalized ratio (INR) >1.2 × ULN, unless participant is receiving anticoagulation, in which instance the INR must fall within the participant's designated therapeutic range.
- Miscellaneous:
- Pregnant or lactating females.
- Any other condition not specifically noted above that, in the opinion of the Investigator or Sponsor, would preclude the participant from participating in the study.
- Participants who are investigational site staff members directly involved in the conduct of the study and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Keros or contract research organization (CRO) employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
Trial locations and contacts
46 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
46 sites in 7 countries
Italy12 sites, 11 recruiting
- Recruiting
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari
Site Contact
Pellegrino Musto
- Recruiting
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi
Bologna
Site Contact
Francesca Palandri
- Recruiting
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Brescia
Site Contact
Mariella D'Adda
- Recruiting
Azienda Ospedaliera Universitaria Careggi
Florence
Site Contact
Alessandro Maria Vannucchi
- Recruiting
Ospedale Policlinico San Martino
Genova
Site Contact
Marica Laurino
- Recruiting
ASST Grande Ospedale Metropolitano Niguarda, Niguarda Cancer Center
Milan
Site Contact
Marianna Caramella
- Recruiting
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan
Site Contact
Alessandra Iurlo
- Completed
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Milan
- Recruiting
Arcispedale S. Maria Nuova Azienda Ospedaliera di Reggio Emilia
Reggio Emilia
Site Contact
Alessia Tieghi
- Recruiting
Azienda Ospedaliera Universitaria Policlinico Umberto I
Roma
Site Contact
Massimo Breccia
- Recruiting
Azienda Socio Sanitaria Territoriale Sette Laghi
Varese
Site Contact
Marco Brociner
- Recruiting
Azienda Ospedaliera Universitaria Integrata Verona
Verona
Site Contact
Massimiliano Bonifacio
Spain7 sites, 7 recruiting
- Recruiting
ICO Badalona - Hospital Universitari Germans Trias i Pujol
Badalona
Site Contact
Bianca Xicoy
- Recruiting
Hospital Universitari Vall d'Hebron
Barcelona
Site Contact
Maria Laura Fox
- Recruiting
Hospital Universitario La Paz
Madrid
Site Contact
Mercedes Gasior Kabat
- Recruiting
Hospital Universitario La Princesa
Madrid
Site Contact
Javier Loscertales
- Recruiting
Hospital Universitario de Salamanca
Salamanca
Site Contact
Jesus Maria Hernandez Rivas
- Recruiting
Hospital Clinico Universitario de Valencia
Valencia
Site Contact
Juan Carlos Juan Carlos Hernandez Boluda
- Recruiting
Hospital QuironSalud de Zaragoza
Zaragoza
Site Contact
Pilar Giraldo
Australia6 sites, 6 recruiting
- Recruiting
Concord Hospital
Concord, New South Wales
Site Contact
Ilona Cunningham
- Recruiting
St. Vincents Hospital Melbourne
Fitzroy, Victoria, 3355
Site Contact
Shuhying Tan
- Recruiting
Royal Melbourne Hospital
Melbourne, Victoria, 3050
Site Contact
Lynette Chee
- Recruiting
The Tweed Hospital
Tweed Heads, New South Wales, 2485
Site Contact
Steven Wong
- Recruiting
Ballarat Oncology & Haematology Service
Wendouree, Victoria, 3355
Site Contact
George Kannourakis
- Recruiting
Flinders Medical Centre
Woodville South, South Australia, 5042
Site Contact
David Ross
Brazil6 sites, 6 recruiting
- Recruiting
Hospital de Clinicas de Porto Alegre
Porto Alegre
Site Contact
Laura Maria Fogliatto
- Recruiting
IMV-Pesquisa Cardiologica Sociedade Simples
Porto Alegre
Site Contact
Marcelo Capra
- Recruiting
Albert Einstein Sociedade Beneficente Israelita Brasiliera
São Paulo
Site Contact
Nelson Hamerschlak
- Recruiting
Hospital Beneficencia Portuguesa de Sao Paulo
São Paulo
Site Contact
Fabio Pires de Souza Santos
- Recruiting
Hospital Das Clinicas Da Faculdade de Medicina Da U S P
São Paulo
Site Contact
Fernanda Salles Seguro
- Recruiting
Instituto de Ensino e Pesquisas Sao Lucas
São Paulo
Site Contact
Rodrigo Santucci Alves da Silva
France6 sites, 2 recruiting
- Completed
CHU Amiens - Hopital Sud
Amiens
- Completed
Hopital Morvan
Brest
- Completed
Hopital Prive Sevigne
Cesson-Sévigné
- Recruiting
Centre Hospitalier Lyon Sud
Lyon
Site Contact
Fiorenza Barraco
- Recruiting
Institut de Cancerologie du Gard
Nîmes
Site Contact
Stefan Wickenhauser
- Completed
Hopital de la Source - CHR Orleans
Orléans
United Kingdom5 sites, 5 recruiting
- Recruiting
United Lincolnshire Hospitals NHS Trust - Pilgrim Hospital
Boston
Site Contact
Ciro Rinaldi
- Recruiting
St James Hospital,Leeds
Leeds
Site Contact
Chun Huat Teh
- Recruiting
Guys Hospital
London
Site Contact
Jennifer O'Sullivan
- Recruiting
Hammersmith Hospital
London
Site Contact
Simone Claudiani
- Recruiting
University College London
London
Site Contact
Andrew Wilson
South Korea4 sites, 2 recruiting
- Completed
Gachon University Gil Medical Center
Incheon
- Completed
Samsung Medical Center
Seoul
- Recruiting
Seoul St. Marys Hospital, The Catholic University of Korea
Seoul
Site Contact
Sung-Eun Lee
- Recruiting
Soonchunhyang University Seoul Hospital
Seoul
Site Contact
Jong-Ho Won
About this study
Official title: A Phase 2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KER-050 as Monotherapy or in Combination With Ruxolitinib in Participants With Myelofibrosis
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.
Read the detailed description
Elritercept is an investigational therapeutic protein designed to increase red blood cell and platelet production by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family of proteins to promote hematopoiesis. It is being developed for the treatment of low blood cell counts, or cytopenias including anemia and thrombocytopenia in participants with Myelodysplastic Syndrome (MDS) and Myelofibrosis (MF).
- Conditions
- Myelofibrosis
- Being tested
- Elritercept, Ruxolitinib
- Keywords
- Thrombocytopenia, Anemia, Red blood cells, Bone marrow, KER-050, drug therapy, elritercept
- Medical subject headings
- Primary Myelofibrosis, Thrombocytopenia, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElriterceptDrug
Also called KER-050, TAK-226
Elritercept SC injection.
RuxolitinibDrug
Ruxolitinib tablet.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: Drug: Elritercept; Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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)
Measured: 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
Measured: 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
Participants with anemia requiring RBC transfusions will be assessed for this outcome measure.
Measured: Up to Week 24
Percentage of Participants with RBC Transfusions From Baseline Over a Period of ≥12 Consecutive Weeks
Participants with anemia requiring RBC transfusions will be assessed for this outcome measure and percentage of participants with ≥50% decrease in number of RBC transfusions will be represented.
Measured: Up to Week 24
Percentage of Participants with Mean Haemoglobin (Hgb) Increase From Baseline Over a Period of ≥12 Consecutive Weeks
Participants with transfusion-independence will be assessed for this outcome measure for mean Hgb ≥1.5 grams per deciliter (g/dL) and ≥2.0 g/dL.
Measured: Up to Week 24
Percentage of Participants with Improvement in the Myelofibrosis Symptom Assessment Form Version 4.0 Total Symptom Score (MF-SAF(v4.0)-TSS) From Baseline at Week 24
Percentage of participants with MF-SAF(v4.0)-TSS of ≥50% will be presented for this assessment. MF-SAF v4.0 captures participant self-report of 7 core MF symptoms (this includes fatigue, night sweats, itching, abdominal discomfort, pain under ribs on left side, early satiety, and bone pain) over the past 7 days. Response options range from 0 (absent) to 10 (worst imaginable). The TSS is computed by averaging the individual item responses and multiplying by 7 for a weekly total score ranging from 0 to 70. Higher scores represent higher symptom burden.
Measured: Week 24
Percentage of Participants with Decrease in Spleen Volume From Baseline as Measured by Computed Tomography/Magnetic Resonance Imaging (CT/MRI) at Week 24
Percentage of participants with decrease in spleen volume of ≥35% will be presented for this outcome measure.
Measured: Week 24
Plasma Concentrations of Elritercept
Measured: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Maximum Observed Serum Concentration (Cmax) of Elritercept
Measured: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Time to Maximum Observed Concentration (Tmax) of Elritercept
Measured: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Area Under the Concentration-time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Elritercept
Measured: Through Cycle 1 (each cycle=28 days)
Minimum Observed Serum Concentration (Cmin) of Elritercept
Measured: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Accumulation Rate (Rac) of Elritercept
Measured: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Change From Baseline in Red Cell Parameters
The red cell parameters including reticulocyte count, Hgb, mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), and reticulocyte cell Hgb will be assessed.
Measured: At multiple timepoints from pre-treatment period up to 8 weeks after end of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2021
- Study start16 Dec 2021
- Primary completion28 Feb 2028 (estimated)
- Study completion28 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 135 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05037760
- Sponsor's protocol ID
- KER050-MF-301
- Other IDs
- 2023-507468-38-00, 2023
- Status last verified
- Feb 2026
- First submitted
- 23 Aug 2021
- Last updated
- 5 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KER-050 as Monotherapy or in Combination With Ruxolitinib in Participants With Myelofibrosis
- ClinicalTrials.gov ID
- NCT05037760
- Acronym / sponsor ID
- KER050-MF-301
- Conditions
- Myelofibrosis
- Keywords
- Thrombocytopenia, Anemia, Red blood cells, Bone marrow, KER-050, drug therapy, elritercept
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 135 participants (estimated)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2021 (actual)
- Primary completion
- 28 Feb 2028 (estimated)
- Study completion
- 28 Feb 2030 (estimated)
- First posted
- 8 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Elritercept is an investigational therapeutic protein designed to increase red blood cell and platelet production by inhibiting the signaling of a subset of the transforming growth factor beta (TGF-ß) family of proteins to promote hematopoiesis. It is being developed for the treatment of low blood cell counts, or cytopenias including anemia and thrombocytopenia in participants with Myelodysplastic Syndrome (MDS) and Myelofibrosis (MF).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Arm 1A: Elritercept | Experimental | Drug: ElriterceptParticipants 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. |
| Arm 1B: Elritercept + Ruxolitinib | Experimental | Drug: Elritercept, Drug: RuxolitinibParticipants 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. |
| Arm 2A: Elritercept | Experimental | Drug: ElriterceptParticipants 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. |
| Arm 2B: Elritercept + Ruxolitinib | Experimental | Drug: Elritercept, Drug: RuxolitinibParticipants 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. |
| Experimental: Arm 2C: Elritercept (Brazil Only) | Experimental | Drug: ElriterceptParticipants 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. |
| Long-Term Extension | Experimental | Drug: Elritercept, Drug: RuxolitinibParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- 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.
Key exclusion criteria (53 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ElriterceptAlso: KER-050, TAK-226Elritercept SC injection. | Drug | Arm 1A: Elritercept, Arm 1B: Elritercept + Ruxolitinib, Arm 2A: Elritercept, Arm 2B: Elritercept + Ruxolitinib, Experimental: Arm 2C: Elritercept (Brazil Only), Long-Term Extension |
| RuxolitinibRuxolitinib tablet. | Drug | Arm 1B: Elritercept + Ruxolitinib, Arm 2B: Elritercept + Ruxolitinib, Long-Term Extension |
Primary outcome
Part 1: Number of Participants with Adverse Events (AEs), Dose Limiting Toxicities (DLTs), Severe Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From signing of the informed consent form (ICF) through 30 days after the last dose of study drug (approximately 8 years)
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.
Part 2 and Long-Term Extension: Number of Participants with Adverse Events (AEs), Severe AEs and SAEs
Time frame: From signing of the ICF through 30 days after the last dose of study drug, (approximately 8 years)
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.
14 secondary outcomes
Percentage of Participants with Progression to Acute Myeloid Leukemia (AML)
Time frame: 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
Time frame: 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
Time frame: Up to Week 24
Percentage of Participants with RBC Transfusions From Baseline Over a Period of ≥12 Consecutive Weeks
Time frame: Up to Week 24
Percentage of Participants with Mean Haemoglobin (Hgb) Increase From Baseline Over a Period of ≥12 Consecutive Weeks
Time frame: Up to Week 24
Percentage of Participants with Improvement in the Myelofibrosis Symptom Assessment Form Version 4.0 Total Symptom Score (MF-SAF(v4.0)-TSS) From Baseline at Week 24
Time frame: Week 24
Percentage of Participants with Decrease in Spleen Volume From Baseline as Measured by Computed Tomography/Magnetic Resonance Imaging (CT/MRI) at Week 24
Time frame: Week 24
Plasma Concentrations of Elritercept
Time frame: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Maximum Observed Serum Concentration (Cmax) of Elritercept
Time frame: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Time to Maximum Observed Concentration (Tmax) of Elritercept
Time frame: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Area Under the Concentration-time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Elritercept
Time frame: Through Cycle 1 (each cycle=28 days)
Minimum Observed Serum Concentration (Cmin) of Elritercept
Time frame: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Accumulation Rate (Rac) of Elritercept
Time frame: Within 2 hours before administration of elritercept at multiple timepoints up to 8 weeks after the end of treatment
Change From Baseline in Red Cell Parameters
Time frame: At multiple timepoints from pre-treatment period up to 8 weeks after end of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2026.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
Takeda
46 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Elritercept and Ruxolitinib in people with Myelofibrosis, run by Takeda at 46 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 33 inclusion and 53 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in Italy, Spain, Australia, Brazil, France and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Number of Participants with Adverse Events (AEs), Dose Limiting Toxicities (DLTs), Severe Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From signing of the informed consent form (ICF) through 30 days after the last dose of study drug (approximately 8 years)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2026.