A Pragmatic Clinical Trial to Prevent Relapse for Myeloid Malignancies With Measurable Disease Prior to Allogeneic Transplant

Phase 2Not Yet RecruitingNCT07572929

Medical College of Wisconsin450 enrolled

Overview

This is a prospective, open-label investigation to determine whether measurable residual disease (MRD) guided assignment of maintenance therapy is effective in patients with myeloid malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). Pre-HCT, patients will undergo usual disease assessments which should include immunophenotypic and/or molecular testing. Based on the results of these tests, patients may or may not be recommended to receive maintenance therapy post-transplant, depending on the presence or absence of a residual malignant clone.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

450

Conditions

Myeloid Disorders Myelodysplastic Syndromes Myeloproliferative Neoplasm Acute Myeloid Leukemia

Interventions

Maintenance TherapyDRUG

Maintenance therapy will be suggested by the PIs, however final choice of maintenance is at the discretion of the treating physician.

No interventionOTHER

Patients will receive no maintenance therapy.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age ≥18 years at time of transplant. * Must have a diagnosis of a myeloid malignancy, myelodysplastic syndrome, myeloproliferative neoplasm (including primary or secondary myelofibrosis), or acute myeloid leukemia or biphenotypic acute leukemia. * Pre-transplant disease assessment must include at least one: * Immunophenotypic assessment by flow for aberrant blasts * NGS to identify persistence of malignant clones using a standard clinically available assay * PCR based measurement of prior identified mutations (if applicable) * Ability to understand a written informed consent document, and the willingness to sign it. Exclusion Criteria: None

Outcomes

Primary Outcomes

Number of subjects with relapse-free survival

All patients will have standard histological analysis of bone marrow biopsies done post-transplant after engraftment, per institutional standard, at one year. For acute leukemia (AML or ALL) or myelodysplastic syndrome, relapse is defined as greater than 5% blasts on histologic analysis of bone marrow biopsy. Among patients assigned to Arm B, institution of any therapy with intent to treat hematologic malignancy will also be considered evidence of relapse.

Time frame: One Year