Clinical Trial 21405

Cancer Type: Malignant Hematology
Study Type: Treatment
NCT#: NCT05732961

Phase: Phase II
Principal Investigator: Komrokji, Rami

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Overview

Study Title

Phase 2, Single Arm Study of Luspatercept for the Treatment of Anemia in lower Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Myelodysplastic Syndromes/Myeloproliferative Neoplasms (MDS/MPN)

Summary

The purpose of the study is to see if participants with anemia due to their type of MDS or MDS/MPN will experience a more decreased need for regular blood transfusions if they take luspatercept plus best supportive care, and what effect, good and/or bad, luspatercept has on them and their anemia due to MDS or MDS/MPN. The safety and tolerability of luspatercept will also be evaluated in this study.

Objective

1. To evaluate RBC transfusion independence (RBC-TI) for the treatment of anemia due to IPSS-R very low, low, or intermediate risk MDS or non-proliferative MDS/MPN who require RBC transfusions; 2. To assess the safety and tolerability of luspatercept in this population; 3. To evaluate the effect of luspatercept on reduction in RBC transfusions, increase in hemoglobin, duration of RBC-TI, increase in neutrophils, and increase in platelets; 4. To evaluate changes in ASC specks in peripheral blood plasma as a biomarker of drug response, and relationship to NLRP3 inflammasome activation in bone marrow cells.

Treatments

Therapies

Therapy (NOS)

Medications

ACE-536 (Luspatercept); Luspatercept ()

Inclusion Criteria

Participant is >18 years at the time of signing the informed consent form

Participant is willing and able to adhere to the study visit schedule and other protocol requirements

Documented diagnosis of MDS or non-proliferative MDS/MPN (WBC > Documented acquired splicing gene mutation: Cohort 1: detectable splicing mutation other than SF3B1; Cohort 2: SF3B1 mutation with prior treatment with hypomethylating agent and or lenalidomide

> Refractory, intolerant to, or ineligible for, prior ESA treatment, as defined by protocol

Discontinuation of ESAs, G-CSF, GM-CSF ≥ 4 weeks prior to start of study treatment

Require RBC transfusions

Applies to on treatment subjects only - females of childbearing potential (FCBP) defined as a sexually mature woman who: has achieved menarche at some point, has not undergone a hysterectomy or bilateral oophorectomy, or has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must: Have two negative pregnancy tests 48 hours apart as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence* from heterosexual contact. Either commit to true abstinence*from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy

Applies to on treatment subjects only - Male subjects must: Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy. * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception).

Exclusion Criteria

Prior allogeneic or autologous stem cell transplant

MDS associated with del 5q cytogenetic abnormality if no prior lenalidomide treatment

Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment

ANC > Platelet count ˂50,000/μL (50 x 109/L)

Active other malignancies

Severe renal impairment (eGFR >ALT or AST ≥ 3 × ULN

Prior treatment with Luspatercept or Sotatercept

Pregnant or breastfeeding females

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