A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda
About this trial
The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept. Other aims are to learn how safe elritercept is by looking at how many participants have MDS that worsens during the study and learn about the effects of elritercept on anemia linked to MDS. The study will also look to learn how elritercept affects the production of healthy RBCs.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Male or female ≥ 18 years of age, at the time of signing informed consent.
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).
Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.
Disqualifiers
Diagnosis of MDS with deletion of chromosome 5q (Del5q).
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 uncontrolled heart disease or New York Heart Association (NYHA) Class III or IV heart failure.
History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.
Trial design
Design model
Sequential
Treatments tested in this trial
Elritercept
DrugElritercept SC injection.
Treatment groups
160 Participants
are divided into 13 treatment groups13
Treatment groupsSee each treatment group below.
Group A: Part 1: Elritercept Cohort 1Experimental treatment 1 intervention
Group B: Part 1: Elritercept Cohort 2Experimental treatment 1 intervention
Group C: Part 1: Elritercept Cohort 3Experimental treatment 1 intervention
Group D: Part 1: Elritercept Cohort 4Experimental treatment 1 intervention
Group E: Part 1: Elritercept Cohort 5Experimental treatment 1 intervention
Group F: Part 2: Elritercept Dose Confirmation Cohort AExperimental treatment 1 intervention
Group G: Part 2: Elritercept Dose Confirmation Cohort BExperimental treatment 1 intervention
Group H: Part 2: Elritercept Dose Confirmation Cohort CExperimental treatment 1 intervention
Group I: Experimental: Part 2: Elritercept Dose Confirmation Cohort DExperimental treatment 1 intervention
Group J: Part 2: Elritercept Dose Confirmation Cohort EExperimental treatment 1 intervention
Group K: Part 2: Elritercept Dose Confirmation Cohort FExperimental treatment 1 intervention
Group L: Part 2: Elritercept Dose Confirmation Cohort GExperimental treatment 1 intervention
Group M: Long-term Extension CohortExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (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. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not it is related to the medicinal (investigational) product. 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.
Time frame
From treatment initiation to end of study (up to 11.2 years)Secondary outcomes
1
Number of Participants with Progression to Higher Risk MDS or Acute Myeloid Leukemia (AML)
The progression to higher risk MDS or AML will be assessed as per the World Health Organization (WHO) 2016 criteria.
Time frame
From study day 1 through end of study (up to 11.2 years)2
Percentage of Participants with Low Transfusion Burden (LTB) and High Transfusion Burden (HTB) who Achieve RBC Transfusion Independence (TI)
Participants with LTB and HTB achieving RBC TI greater than or equal to (≥) 8 weeks, overall RBC TI and based on RS status will be assessed.
Time frame
From study day 1 through end of study (up to 11.2 years)3
Percentage of Participants who Achieve Hematologic Improvement Erythroid (HI-E) Response Based on Modified 2006 International Working Group (IWG)
The response is defined as a mean hemoglobin (Hgb) increase of ≥1.5 grams per deciliter (g/dL) from Baseline during any 8-week period during the treatment period for LTB participants and participants with non-transfused anemia, and is defined as a reduction by ≥ 4 units of RBCs transfused during any 8-week period on study compared with the 8-week period prior to study cycle 1 day 1 (C1D1) (cycle length = 28 days) for HTB participants. Participants achieving overall HI-E response and based on RS status will be assessed.
Time frame
From study day 1 to end of study (up to 11.2 years)4
Percentage of Participants who Achieve Overall Erythroid Response
The response is defined as a mean hemoglobin (Hgb) increase of ≥1.5 grams per deciliter (g/dL) from Baseline during any 8-week period during the treatment period for LTB participants and participants with non-transfused anemia, and is defined as a reduction by ≥ 4 units of RBCs transfused during any 8-week period on study compared with the 8-week period prior to study cycle 1 day 1 (C1D1) (cycle length = 28 days) for HTB participants. TI is defined as RBC transfusion independence for ≥ 8 weeks. Overall erythroid response is defined as HI-E or TI over any 8-week period. Participants achieving overall erythroid response and based on RS status will be assessed.
Time frame
Up to approximately 11.2 years Source ClinicalTrials.gov