A global clinical study to test if the medicine Elritercept (KER-050) is effective and safe for treating adults with a type of blood disorder called Myelodysplastic Syndromes (MDS)
Scientific Title of Study
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)
Australia Brazil Bulgaria Canada Chile Czech Republic France Germany Hungary India Ireland Israel Italy Lithuania Peru Poland Republic of Korea South Africa Spain Sweden Taiwan Thailand Turkey United Kingdom United States of America
All India Institute of Medical Sciences, Room No.10, Porta Cabin, 5th floor Teaching block, New Delhi 110029 New Delhi DELHI
919811262092
drtulikaseth@gmail.com
Dr Neeraj Sidharthan
Amrita Institute of Medical Sciences
Amrita Institute of Medical Sciences, AIMS Ponekkara P O, Kochi-682041 Ernakulam KERALA
919946047464
neerajsidharthan@aims.amrita.edu
Dr Velu Nair
Apollo Hospitals International Limited
Apollo Hospitals International Limited, Plot no 1A, Bhat GIDC, Gandhinagar, Ahmedabad, 382428 Ahmadabad GUJARAT
9107966701800
nairvelu2000@yahoo.com
Dr Biju George
Christian Medical College
Christian
Medical College
Vellore- Ranipet
campus, Kilminnal
Village & Post,
Ranipet District-
632 517 Vellore TAMIL NADU
914172224553
biju@cmcvellore.ac.in
Dr M Joseph John
Christian Medical College
Christian Medical College, and Hospital/Brown Road, Ludhiana 141008 Ludhiana PUNJAB
91805495952
mjosephjohn@cmcludhiana.in
Dr Sandip Shah
HOC Vedanta (Cellcure Care Cancer Pvt. Ltd.)
HOC Vedanta (Cellcure Care Cancer Pvt. Ltd.), Ground Floor & First Floor, Vedanta Institute of Medical Sciences, Near Samved Hospital, Navrangpura, Ahmedabad- 380009 Ahmadabad GUJARAT
919824041170
sandip60@ yahoo.com
Dr Pradeep Kumar
Max Super Specialty Hospital, Vaishali A unit of Crosslay Remedies Limited
Nehru Hospital, Postgraduate Institute of Medical Education and research
Department of Clinical Hematology and Medical Oncology, 4th floor, F block, Nehru Hospital, Postgraduate Institute of Medical Education and research., Secotor 12, Chandigarh Chandigarh CHANDIGARH
917087009680
malhotrapankaj@hotmail.com
Dr Tuphan Kanti Dolai
NRS Medical College and Hospital
NRS Medical College and Hospital 138, A.J.C Bose Road Kolkat– 700014 Kolkata WEST BENGAL
91 9874890275
tkdolai@hotmail.com
Dr Dinesh Bhurani
Rajiv Gandhi Cancer Institute & Research Centre
Rajiv Gandhi Cancer Institute & Research Centre, Sector 5, Rohini, New Delhi, 110085 New Delhi DELHI
919971500861
bhurani@g mail.com
Dr Arijit Nag
Tata Medical Center
Tata Medical
Center
14, MAR
E/W, Newtown
Rajarhat
Kolkata-700160 Kolkata WEST BENGAL
9190511 21161
arijit.nag@tmckolkata.com
Dr Alok Shetty
Tata Memorial Hospital
Tata Memorial Hospital, Ernst Borges Road, Parel, Mumbai 400012 Mumbai MAHARASHTRA
Dose: Liquid solution for subcutaneous (SC) injection
Frequency:13 doses per year
Route of administration: subcutaneous (SC) injection
Duration: up to 5 years of treatment.
Comparator Agent
Placebo
Dose: Liquid solution for subcutaneous (SC) injection
Route of administration: subcutaneous (SC) injection
Frequency: 13 doses per year
Route of administration:
Duration: up to 3 years of treatment.
Inclusion Criteria
Age From
18.00 Year(s)
Age To
99.00 Year(s)
Gender
Both
Details
1. 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.
2. Male or female greater than or equal to 18 years of age at the time of signing informed consent.
3. Diagnosis of MDS with or without RS according to WHO 2016 classification that meets the IPSS-R classification of very low-, low-, or intermediate-risk MDS.
4. Transfusion dependence assessed in the 16 weeks immediately preceding randomization in two 8-week blocks, classified as either
a. LTB, defined as 4 to 7 RBC units per 16 weeks
b. HTB, defined as greater than or equal to 8 RBC units per 16 weeks
c. For all participants
i. Only transfusion events for a pretransfusion Hgb less than 10 g per dL are counted toward eligibility
ii. At least 1 transfusion event in each 8-week period and a minimum of 2 transfusion events separated by greater than or equal to 7 days within the 16-week period immediately preceding randomization
iii. No consecutive 56-day period can be RBC transfusion-free during the 16-week period immediately preceding randomization.
5. Refractory or intolerant to prior ESA treatment (discontinued greater than or equal to 4 weeks before randomization), or unlikely to respond to ESA treatment, defined as follows
a. Refractory to prior ESA treatment
documentation of nonresponse or a response that was no longer maintained with a prior ESA-containing regimen, either as a single agent or combination (e.g., with granulocyte colony-stimulating factor (G CSF)) ESA regimen must have been either
i. Recombinant human EPO greater than or equal to 40,000 IU per week for greater than or equal to 8 doses or equivalent
ii. Darbepoetin alpha greater than or equal to 500 mcg every 3 weeks for greater than or equal to 4 doses or equivalent.
b. Intolerant to prior ESA treatment documentation of discontinuation of a prior ESA-containing regimen, either as a single agent or combination (e.g., with G-CSF), at any time after introduction due to intolerance or an AE.
c. Unlikely to respond to ESA treatment low chance of response to ESA based on an endogenous serum EPO level greater than 200 U per L.
6. Less than 5 percent blasts in an evaluable bone marrow aspirate collected at Screening, read by an independent central reader.
7. ECOG performance status of 0 to 2
8. Females of childbearing potential and sexually active males must agree to use adequate contraception methods
9. 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).
ExclusionCriteria
Details
1. Del(5q) MDS or therapy-related (secondary) MDS.
2. Anemia due to any other known cause (e.g., thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and/or folate).
3. Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks before randomization.
4. Clinically significant cardiovascular disease defined as
a. New York Heart Association heart disease class III or IV
b. Fridericia corrected QT (QTcF) interval greater than 500 milliseconds during Screening
c. Presence of uncontrolled hypertension defined as mean systolic blood pressure greater than or equal to 160 mm Hg or diastolic blood pressure greater than or equal to 100 mm Hg during Screening
d. Uncontrolled arrhythmia, myocardial infarction, or unstable angina within 6 months before Screening.
5. Known ejection fraction less than 35 percent, confirmed by a local echocardiogram performed during Screening, or a previously performed echocardiogram if collected within 6 months before Screening.
6. Child-Pugh class C hepatic impairment.
7. Stroke, deep vein thrombosis, or pulmonary embolism within 6 months before Screening.
8. Any known history of AML.
9. Prior history of malignancies, other than MDS, unless the participant has been free of the disease (including completion of any treatment, including maintenance, for prior malignancy) for greater than or equal 5 years. However, participants with a history or concurrent diagnosis of the following conditions are allowed if not requiring systemic therapy
a. Basal or squamous cell carcinoma of the skin
b. Carcinoma in situ of the cervix
c. Carcinoma in situ of the breast
d. Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis (TNM) clinical staging system).
10. History of solid organ or bone marrow transplantation.
11. Active infection requiring intravenous treatment (e.g. antibiotics, antifungals, or antivirals) within 28 days, or oral treatment within 14 days before randomization.
12. History of or known active or 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.
13. Body mass index greater than or equal 40 kg per m2.
14. Major surgery within 28 days before randomization.
15. History of allergy or anaphylaxis to investigational medicinal product (IMP) excipients (refer to the current elritercept IB for a list of excipients) or recombinant proteins.
Treatment History
16. Prior use of elritercept, luspatercept, or sotatercept.
17. Prior use of hypomethylating agents (HMAs), isocitrate dehydrogenase inhibitor, lenalidomide, imetelstat, or immunosuppressive therapy given for treatment of MDS.
18. Iron chelation therapy initiated within 8 weeks before randomization. Participants on stable doses of iron chelation therapy for greater than or equal 8 weeks are allowed.
19. Vitamin B12 or folate therapy initiated within 4 weeks before randomization. Participants on stable replacement doses for greater than or equal 4 weeks and without ongoing concurrent vitamin B12 or folate deficiency are allowed.
20. Androgen use within 8 weeks before randomization. Participants on stable androgen dosing for hypogonadism for greater than or equal 8 weeks are allowed.
21. High-dose corticosteroid use within 4 weeks before randomization. Participants on stable chronic steroid doses of prednisone less than or equal 10 mg per day or corticosteroid equivalent for greater than or equal 4 weeks are allowed.
22. 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.
23. Ongoing participation in another interventional clinical study.
Laboratory Exclusions (during Screening)
24. Serum EPO level greater than 500 U per L.
25. Platelet count greater than or equal 450 × 109 per L or less than or equal 25 × 109 per L.
26. Absolute neutrophil count less than or equal 500 per micrograms L.
27. Serum aspartate aminotransferase or alanine aminotransferase greater than or equal to 3 × the upper limit of normal (ULN).
28. Total bilirubin greater than or equal to 2 × ULN unless attributable to Gilbert syndrome.
29. Ferritin less than or equal to 50 micrograms/L.
30. Folate less than or equal 2.0 ng/mL.
31. Vitamin B12 less than or equal 200 pg/mL.
32. Estimated glomerular filtration rate less than 30 mL per min per 1.73m2 as determined by the Chronic Kidney Disease Epidemiology (CKD-EPI) Collaboration equation
Miscellaneous
33. Pregnant or lactating female.
34. Any other condition not specifically noted above that, in the opinion of the Investigator, would preclude the participant from participating in the study or could confound interpretation of data from the study.
35. Investigational site staff members directly involved in the conduct of the study and site staff members otherwise supervised by the Investigator, employees of the Sponsor or contract research organization directly involved in the conduct of the study, or immediate family members (defined as a spouse, parent, child, or sibling, whether biological or legally adopted).
36. For Participants in France: Persons under court protection, persons not affiliated with a social security system, and protected adults (per applicable French law (Art. L. 1121-6, Art. L. 1121-8, Art. L. 1121-8-1)).
Method of Generating Random Sequence
Computer generated randomization
Method of Concealment
Centralized
Blinding/Masking
Participant and Investigator Blinded
Primary Outcome
Outcome
TimePoints
To evaluate the efficacy of elritercept in reducing red blood cell (RBC) transfusions
Endpoints
To evaluate the efficacy of elritercept in reducing red blood cell (RBC) transfusions
Endpoints
Secondary Outcome
Outcome
TimePoints
To evaluate the efficacy of elritercept in reducing RBC transfusions over longer intervals & or in participants with high-transfusion burden (HTB)
To assess the safety & tolerability of elritercep
Proportion of participants achieving TI for greater than or equal to 24 weeks from baseline through week 48
Proportion of participants with HTB achieving TI for greater than or equal to 8 weeks from baseline through week 24
Incidence of treatment-emergent adverse events & serious adverse events
Change from baseline in clinical laboratory values, vital signs, & electrocardiograms
Target Sample Size
Total Sample Size="225" Sample Size from India="30" Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials" Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials"
Phase of Trial
Phase 3
Date of First Enrollment (India)
16/03/2026
Date of Study Completion (India)
Applicable only for Completed/Terminated trials
Date of First Enrollment (Global)
04/07/2025
Date of Study Completion (Global)
Applicable only for Completed/Terminated trials
Estimated Duration of Trial
Years="5" Months="0" Days="0"
Recruitment Status of Trial (Global)
Open to Recruitment
Recruitment Status of Trial (India)
Open to Recruitment
Publication Details
N/A
Individual Participant Data (IPD) Sharing Statement
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
Brief Summary
The purpose of this study is to determine whether Elritercept is a safe and effective treatment for Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes.