Clinical Trial 23674

• Overview

  Study Title:

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

  Summary:

  The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept works in reducing the need for RBC transfusions over longer periods of time or in adults with high transfusion needs. The study will also check on how safe elritercept is and how well it is tolerated.

  Objective:

  Primary: *To evaluate the efficacy of elritercept in reducing RBC transfusions. Secondary: *To evaluate the efficacy of elritercept in reducing RBC transfusions over longer intervals and/or in participants with HTB. *To assess the safety and tolerability of elritercept.

• Treatments

  Therapies:

  Activin Receptor Type IIA (ActRIIA) ligand trap

  Medications:

  Elritercept/Placebo ()

• Inclusion Criteria

  Key Inclusion Criteria:
  • Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information and/or protected personal data in accordance with national and local study participant data protections and privacy regulations.
  • Male or female greater than or equal to (≥)18 years of age at the time of signing informed consent.
  • Diagnosis of MDS with or without RS (as determined in an evaluable bone marrow aspirate, read by an independent central reader to confirm diagnosis at Screening) according to the World Health Organization 2016 classification that meets the International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.
  • Transfusion dependence assessed in the 16 weeks immediately preceding randomization in two 8-week blocks.
  • Refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) treatment (discontinued ≥4 weeks before randomization), or unlikely to respond to ESA treatment.
  • Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening, read by an independent central reader.
  • Eastern Cooperative Oncology Group performance status of 0 to 2.
  • Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.
  • 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).
  • Additional criteria may apply.

• Exclusion Criteria

  Key Exclusion Criteria:
  • Del(5q) MDS or therapy-related (secondary) MDS.
  • Anemia due to any other known cause (e.g., thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and/or folate).
  • Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks before randomization.
  • Clinically significant cardiovascular disease.
  • Known ejection fraction > Child-Pugh class C hepatic impairment.
  • Stroke, deep vein thrombosis, or pulmonary embolism within 6 months before Screening.
  • Any known history of acute myeloid leukemia (AML).
  • Prior history of malignancies, other than MDS, unless participant has been free of the disease (including completion of any treatment, including maintenance, for prior malignancy) for ≥ 5 years.
  • History of solid organ or bone marrow transplantation.
  • Active infection requiring intravenous treatment (e.g., antibiotics, antifungals, or antivirals) within 28 days, or oral treatment within 14 days before randomization.
  • History of or known active chronic infection with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV). Participants without known positive history of HIV, HBV, and/or HCV do not require further testing, unless testing is mandated per local guidelines.
  • Body mass index ≥ 40 kilograms per meter square (kg/m^2).
  • Major surgery within 28 days before randomization.
  • History of allergy/anaphylaxis to investigational medicinal product (IMP) excipients (refer to the current elritercept IB for a list of excipients) or recombinant proteins.
  • Prior use of elritercept, luspatercept, or sotatercept.
  • Prior use of hypomethylating agents (HMAs), isocitrate dehydrogenase inhibitor, lenalidomide, imetelstat, or immunosuppressive therapy given for treatment of MDS.
  • Iron chelation therapy initiated within 8 weeks before randomization. Participants on stable doses of iron chelation therapy for ≥ 8 weeks are allowed.
  • Vitamin B12 or folate therapy initiated within 4 weeks before randomization. Participants on stable replacement doses for ≥ 4 weeks and without ongoing concurrent vitamin B12 or folate deficiency are allowed.
  • Androgen use within 8 weeks before randomization. Participants on stable androgen dosing for hypogonadism for ≥ 8 weeks are allowed.
  • High-dose corticosteroid use within 4 weeks before randomization. Participants on stable chronic steroid doses of prednisone lesser than or equal to (≤) 10 mg/day or corticosteroid equivalent for ≥ 4 weeks are allowed.10 mg/day or corticosteroid equivalent for ≥ 4 weeks are allowed.
  • Treatment with any investigational drug within 28 days before Screening or, if the half-life of the product is known, within 5 times the half-life before Screening, whichever is longer.
  • Ongoing participation in another interventional clinical study.
  • Serum EPO level >500 U/L.
  • Platelet count ≥450 × 10^9/L or ≤25 × 10^9/L.
  • Absolute neutrophil count ≤ 500/µL.
  • Serum aspartate aminotransferase or alanine aminotransferase ≥3 × the upper limit of normal (ULN).
  • Total bilirubin ≥2 × ULN unless attributable to Gilbert's syndrome.
  • Ferritin ≤ 50 micrograms per litre (μg/L).
  • Folate ≤2.0 nanograms per milliliter (ng/mL).
  • Vitamin B12 ≤200 picograms per milliliter (pg/mL).
  • Additional criteria may apply.

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