Loading clinical trials...

Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts | Clinical Trials | Clareo Health

TERMINATEDPHASE2

Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts

Phase II Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Patients With Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis (MDS/MPN-RS-T) and Myelodysplastic/Myeloproliferative Neoplasms, Unclassifiable With Ring Sideroblasts (MDS/MPN-U With RS)

NCT05005182•Mayo Clinic

View on ClinicalTrials.gov

Summary

This phase II trial studies the effects of luspatercept with or without hydroxyurea in treating patients with myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis or unclassifiable with ring sideroblasts. Biological therapies, such as luspatercept, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop cancer cells from growing. Hydroxyurea may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving luspatercept with or without hydroxyurea may help doctors determine what doses of the combination is safe for patients to take and how the disease responds to the treatment.

Detailed Description

PRIMARY OBJECTIVE: I. To document the erythroid response rate assessed as per the 2015 International Working Group (IWG) myelodysplastic (MDS)/myeloproliferative neoplasms (MPN) response criteria. SECONDARY OBJECTIVES: I. To document response duration, time to acute myeloid leukemia (AML) transformation, AML-free survival (LFS) and overall survival (OS) in patients with MDS/MPN-with ring sideroblasts (RS) and thrombocytosis (T) and MDS/MPN-unclassifiable (U)U with RS. II. To document safety of luspatercept (luspatercept-aamt) in patients with MDS/MPN-RS-T and MDS/MPN-U with RS. EXPLORATORY OBJECTIVE: I. To assess overall health-related quality of life as measured by Hematological Malignancy Specific Patient-Reported Outcome Measure (HM-PRO). CORRELATIVE RESEARCH OBJECTIVE: I: To find an effective biomarker of response to luspatercept-aamt. OUTLINE: Patients are assigned to 1 of 2 cohorts. COHORT A: Patients receive luspatercept subcutaneously (SC) on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection, bone marrow biopsy and aspirate at baseline and on study. COHORT B: Patients receive luspatercept SC on day 1 and hydroxyurea orally (PO) on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection, bone marrow biopsy and aspirate at baseline and on study. After completion of study treatment, patients are followed up every 3 months.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Age \>= 18 years
•Patients with a World Health Organization(WHO)-defined diagnosis of MDS/MPN-RS-T or MDS/MPN-U with \>= 15% RS
•Prior treatment with lenalidomide, hypomethylating agents, immunosuppressive therapy, erythropoietin stimulating agents (ESA) or investigational agent is allowed as long as patients have not received luspatercept-aamt or sotatercept. If there is prior history of investigational agent, there should be an interval equivalent to at least four elimination half-lives of the agent prior to enrollment. Note: For patients who have received prior lenalidomide, hypomethylating agents, or immunosuppressive therapy, there must be \>= 6 weeks since the last dose before luspatercept-aamt treatment is started
•Eastern Cooperative Oncology Group (ECOG) performance score of 0, 1 or 2
•Requirement of red blood cell transfusions (\>= 2 unit =\< 8-weeks prior to registration) OR symptomatic anemia with hemoglobin \< 9.5 g/dL OR hematocrit \< 30% (as long as there is documentation of adequate iron stores (ferritin \> 50 mg/L) =\< 5 weeks prior to registration). Symptomatic anemia is defined as fatigue with or without exertion, shortness of breath with or without exertion, or decrease in exercise tolerance
•Hemoglobin =\< 9.5 g/dL (obtained =\< 14 days prior to registration)
•Total bilirubin =\< 1.5 x upper limit of normal (ULN) (obtained =\< 14 days prior to registration)
•Alanine aminotransferase (ALT) and aspartate transaminase (AST) =\< 3 x ULN (=\< 5 x ULN for patients with liver involvement) (obtained =\< 14 days prior to registration)
•Calculated creatinine clearance \>= 30 ml/min using the Cockcroft-Gault (obtained =\< 14 days prior to registration)
•Females of childbearing potential (FCBP) defined as a sexually mature woman who:
+12 more criteria

Exclusion Criteria

•Any of the following because this study involves an agent that has known genotoxic, mutagenic and teratogenic effects:
•* Pregnant persons
•* Nursing persons
•* Persons of childbearing potential who are unwilling to employ highly effective contraception
•Any of the following prior therapies:
•* Surgery =\< 3 weeks prior to registration
•* Chemotherapy or other agents =\< 2 weeks prior to registration
•Uncontrolled intercurrent non-cardiac illness including, but not limited to:
•* Ongoing or active infection
•* Uncontrolled hypertension (defined as systolic blood pressure \>= 140 mmHg or diagnostic blood pressure \>= 90 mmHg despite use of \>= 3 anti-hypertensive drugs at optimal doses)
+10 more criteria

Locations (3)

Mayo Clinic Hospital in Arizona

Phoenix, Arizona, United States

Mayo Clinic in Florida

Jacksonville, Florida, United States

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Key Dates

Start Date

February 20, 2023

Primary Completion Date

April 17, 2023

Completion Date

June 17, 2023

Last Updated

September 19, 2024

Enrollment

ACTUAL participants

Conditions

Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis, Not Otherwise SpecifiedMyelodysplastic/Myeloproliferative Neoplasm, Not Otherwise Specified

Interventions

Biospecimen Collection

PROCEDURE

Bone Marrow Aspiration

PROCEDURE

Bone Marrow Biopsy

PROCEDURE

Hydroxyurea

DRUG

Luspatercept

BIOLOGICAL

Questionnaire Administration

OTHER

Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia

NCT07468916

Atypical Chronic Myeloid LeukemiaChronic Myelomonocytic Leukemia+3 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 19, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions