RecruitingPhase 2

Luspatercept + Darbepoetin in MDS

United States60 participantsStarted 2025-12-16

Plain-language summary

This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in non-mutated SF3B1 , lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level \< 500 IU/L.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Ability to understand and the willingness to sign a written informed consent document.
✓. Participant is 18 years or older at the time of signing informed consent.
✓. Participant has lower-risk myelodysplastic syndrome (MDS) defined as very low, low and intermediate risk by International Prognostic Scoring System-Revised (IPSS-R) criteria (3).
✓. Bone marrow biopsy within 90 days of screening demonstrated less than 5% blasts in the aspirate and/or core biopsy. If no bone marrow biopsy was done within 90 days of screening it is mandatory to repeat it at screening. Otherwise, bone marrow biopsy is optional at screening to obtain correlative study samples.
✓. Absence of SF3B1 mutation and del5q.
✓. Endogenous serum erythropoietin alfa (EPO) level \< 500 IU/L.
✓. Participant is transfusion dependent defined as ≥ 2 packed red blood cell (PRBC) units/8 weeks for a minimum of eight weeks immediately prior to screening. The maximum consecutive timeframe participants may be RBC transfusion-free within this 8-week time period is six weeks.
✓. Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0-3 (Appendix 1. ECOG Performance Status Scale).

Exclusion criteria

✕. Prior treatment with ESA, luspatercept, hypomethylating agents or lenalidomide/thalidomide/other immunomodulating drug (IMiDs).
✕. Exception 1: Participants can have received ≤ 2 doses of prior epoetin alfa or ≤ 1 dose darbepoetin alfa if ≥ 8 weeks from date of consent.
✕. Exception 2: Participants can have received ≤ 1 week of treatment with lenalidomide ≥ 8 weeks from the date of consent, at the sponsor-investigator's discretion.
✕. After signing consent, the participants are not allowed to receive any of these drugs: other RBC hematopoietic growth factors (e.g., Interleukin-3), granulocyte colony stimulating factors (i.e., G-CSF, GM-CSF), except in cases of neutropenic fever, cytotoxic, chemotherapeutic, targeted or investigational agents/therapies, azacitidine, decitabine or other hypomethylating agents, lenalidomide, thalidomide and other immunomodulating drugs (IMiDs), hydroxyurea, androgens, unless to treat hypogonadism, oral retinoids (topical retinoids are permitted), arsenic trioxide, interferon and interleukins.
✕. Participants with history of seizures at any time.
✕. Participants with any of the following conditions within six months prior to screening:
✕. Stroke.
✕. Thrombosis/thromboembolism.

What they're measuring

Mean Rate of Red Blood Cell Transfusion Independence

Timeframe: 24 weeks

Mean Rate of Hemoglobin Increase

Timeframe: 24 weeks

Trial details

NCT IDNCT07096297

SponsorYale University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2030-08

Contact for this trial

Stephanie Ladd

9548950576 stephanie.ladd@yale.edu

View on ClinicalTrials.gov More MDS (Myelodysplastic Syndrome) trials