Ignite Creation Date:
2026-03-26 @ 3:20 PM
Ignite Modification Date:
2026-04-05 @ 6:40 PM
Study NCT ID:
NCT07463820
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-03-25
First Post:
2026-03-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial)
Sponsor:
National Cancer Institute (NCI)
Organization:
Study Overview
Official Title:
A Phase II Study of Combination Therapy With Luspatercept in Lower Risk Myelodysplasia: A Tier 1 MyeloMATCH Substudy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2026-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This phase II MyeloMATCH treatment trial tests luspatercep with or without epoetin alfa or emavusertib for the treatment of low risk myelodysplasia and anemia. Biological therapies, such as luspatercep, use substances made from living organisms that may attack specific cancer cells and stop them from growing or kill them. Epoetin alfa is a substance that is made in the laboratory and stimulates the bone marrow to make red blood cells. Emavusertib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving luspatercep with or without epoetin alfa or emavusertib may be effective for treating patients with low risk myelodysplasia and anemia.
Detailed Description:
PRIMARY OBJECTIVE:
I. To assess the erythroid response rate at 24 weeks (Hematologic Improvement - Erythroid) using revised international working group 2018 criteria (Platzbecker 2019b), in patients with myelodysplastic syndrome (MDS) with previously untreated lower risk disease and symptomatic anemia, when treated with luspatercept based combination or mono-therapy, according to the presence or absence of specific spliceosome mutations.
SECONDARY OBJECTIVES:
I. Transfusion-free survival. II. Duration of the erythroid response. III. Depth of the erythroid response. IV. Bi- or tri-lineage responses. V. Safety and tolerability. VI. Overall and leukemia-free survival. VII. Minimal residual disease (MRD) with next generation sequencing and flow cytometry VIII. Comparison of Hematologic Improvement - Erythroid and safety between arms 2 and 3 (if both arms are positive).
IX. Comparison of Hematologic Improvement - Erythroid and safety between ring sideroblast + and - populations.
OUTLINE: This is a dose-escalation study of luspatercept in combination, dependent upon arms assignments, with fixed dose epoetin alfa or emavusertib. Patients are randomized to 1 of 3 arms.
ARM 1: Patients receive luspatercept subcutaneously (SC) on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
ARM 2: Patients receive luspatercept SC on day 1 and epoetin alfa SC once weekly (QW) for each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
ARM 3: Patients receive luspatercept SC on day 1 and emavusertib orally (PO) twice daily (BID) on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
After completion of study treatment, patients are followed up at 4 weeks and every 3 months until loss of response and/or the development of transfusion dependence then every 6 months thereafter or every 6 months for patients not in HI-E and are transfusion dependent or who have progressed/relapsed.
I. To assess the erythroid response rate at 24 weeks (Hematologic Improvement - Erythroid) using revised international working group 2018 criteria (Platzbecker 2019b), in patients with myelodysplastic syndrome (MDS) with previously untreated lower risk disease and symptomatic anemia, when treated with luspatercept based combination or mono-therapy, according to the presence or absence of specific spliceosome mutations.
SECONDARY OBJECTIVES:
I. Transfusion-free survival. II. Duration of the erythroid response. III. Depth of the erythroid response. IV. Bi- or tri-lineage responses. V. Safety and tolerability. VI. Overall and leukemia-free survival. VII. Minimal residual disease (MRD) with next generation sequencing and flow cytometry VIII. Comparison of Hematologic Improvement - Erythroid and safety between arms 2 and 3 (if both arms are positive).
IX. Comparison of Hematologic Improvement - Erythroid and safety between ring sideroblast + and - populations.
OUTLINE: This is a dose-escalation study of luspatercept in combination, dependent upon arms assignments, with fixed dose epoetin alfa or emavusertib. Patients are randomized to 1 of 3 arms.
ARM 1: Patients receive luspatercept subcutaneously (SC) on day 1 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
ARM 2: Patients receive luspatercept SC on day 1 and epoetin alfa SC once weekly (QW) for each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
ARM 3: Patients receive luspatercept SC on day 1 and emavusertib orally (PO) twice daily (BID) on days 1-21 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and blood sample collection throughout the study.
After completion of study treatment, patients are followed up at 4 weeks and every 3 months until loss of response and/or the development of transfusion dependence then every 6 months thereafter or every 6 months for patients not in HI-E and are transfusion dependent or who have progressed/relapsed.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| NCI-2026-01399 | REGISTRY | CTRP (Clinical Trial Reporting Program) | View | |
| MM1MDS-CTG02 | OTHER | Canadian Cancer Trials Group | View | |
| MM1MDS-CTG02 | OTHER | CTEP | View | |
| U10CA180863 | NIH | None | https://reporter.nih.gov/quic… | View |