Phase 1 / Phase 2: testing Cyclophosphamide for Hematologic Neoplasms

What this phase means

Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are donor lymphocyte infusion, Cyclophosphamide, or Busulfan. The registry lists allocation as na. Background: People with blood cancers often receive blood or bone marrow transplants.

They plan to enroll about 430 people at Bethesda. Taking part typically means taking a study drug (or dummy pill), a procedure as part of the study plus check-ins.

Official title (unrewritten)

Phase I/II Study Using Prophylactic Donor Lymphocyte Infusion Early Post-Transplant After Allogeneic Hematopoietic Cell Transplantation Using Post-Transplantation Cyclophosphamide for High-Risk Hematologic Malignancies

Interventions: donor lymphocyte infusion, Cyclophosphamide, Busulfan, Mycophenolate mofetil, Fludarabine, Sirolimus

Primary outcome (registry wording): determine the maximally tolerated dose of DLI that can be safely administered after HLA-matched-related HCT and after HLA-haploidentical HCT

Who it is for (from inclusion criteria)

• Ages 12 Years to 120 Years
• who can join - Recipient:
• Histologically or cytologically confirmed hematologic malignancy classified as high or very high disease risk by the Refined Disease Risk Index for HCT including one of the following:
• Acute myeloid leukemia (AML) with favorable cytogenetics (t(8;21), inv(16), t(15,17)) with induction failure (persistent disease without first achieving remission of any type) or active relapse
• AML with intermediate cytogenetics (not classified as favorable or adverse) with induction failure or active relapse (AML with intermediate cytogenetics in morphologic complete remission \[CR\] with minimal residual disease detectable by any modality also will be eligible)
• AML with adverse cytogenetics (complex karyotype with \>= 4 abnormalities) regardless of remission status
• Low risk myelodysplastic syndrome (MDS) (\<= 5% blasts, including chronic myelomonocytic leukemia) with adverse cytogenetics (abnormal chromosome 7 or \>= 4 abnormalities) with induction failure or active relapse
• High risk MDS (RAEB-1 or RAEB-2) with intermediate-risk cytogenetics (no abnormal chromosome 7 or \< 4 abnormalities) with induction failure or active relapse