Stem Cell Transplant to Treat Patients With Favorable or Intermediate Risk Minimal Residual Disease Negative Acute Myeloid Leukemia

Fred Hutchinson Cancer Center (FHCC)

Status and phase: Withdrawn; Phase 2

Conditions: Minimal Residual Disease Negativity; Acute Myeloid Leukemia

Treatments: Other: Laboratory Biomarker Analysis; Drug: Etoposide; Procedure: Autologous Hematopoietic Stem Cell Transplantation; Drug: Busulfan

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT03515707

NCI-2017-02069 (Registry Identifier)

RG9217025 (Other Identifier)

P30CA015704 (U.S. NIH Grant/Contract)

9784

Details and patient eligibility

About

This phase II trial studies how well autologous stem cell transplant works in treating patients with favorable or intermediate risk, minimal residual disease (MRD)-negative, acute myeloid leukemia. Giving chemotherapy before a peripheral blood stem cell transplant helps kill any cancer cells that are in the body. After treatment, stem cells are collected from the patient's blood and stored. Higher dose chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Full description

PRIMARY OBJECTIVES:

I. Assess the estimated probability of relapse at 2 years after autologous peripheral blood stem cell (PBSC) transplant.

SECONDARY OBJECTIVES:

I. Estimate the probability of transplant-related mortality (TRM) at 100 days following autologous stem cell transplant (ASCT).

II. Estimate probabilities of overall and disease-free survival. III. Assess if biological and molecular correlative studies can predict better outcome.

OUTLINE:

Patients receive targeted busulfan intravenously (IV) or oral (PO) every 6 hours on days -7 to -4 and etoposide IV on day -3. Patients then undergo autologous stem cell transplant on day 0.

After completion of study treatment, patients are followed up yearly for 2 years.

Sex: All

Ages: 18 to 69 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• AML favorable or intermediate ELN risk
• Achieved true 1st complete response (CR) (absolute neutrophil count [ANC] and platelet count > 1,000/ul and 100,000/ul respectively) after first cycle of induction therapy, with no minimal residual disease (MRD)
• No measurable residual disease (MRD) as assessed by flow cytometry after initial induction therapy
• Performance score Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2
• Creatinine < 2.0 mg/dl and calculated by Cockcroft-Gault (CG) formula or 24 hour measured creatinine clearance (CRCL) > 50
• Not pregnant
• Received 1-2 courses of post remission "consolidation" therapy prior to mobilization PBSC
• No MRD by flow, cytogenetics, fluorescence in situ hybridization (FISH) and molecular testing prior to collection of autologous PBSC collection
• Plan is to collect at least 3 x 10^6 CD34+ PBSC/kg cryopreserved; preference is 4-5 X 10^6 CD34 cells/kg

Exclusion criteria

• Life expectancy is severely limited by diseases other than AML
• Total bilirubin > 2.0 mg/dl or serum glutamic-oxaloacetic transaminase (SGOT)/serum glutamate pyruvate transaminase (SGPT) > 2.5 x upper limit of normal (ULN)
• History of Gilbert's disease
• Uncontrolled arrhythmias, left ventricular ejection fraction (LVEF) < 50% or corrected diffusion capacity of the lung for carbon monoxide (DLCO) < 50%
• Significant active infection that precludes transplant
• Hepatitis B or C viremia at time of ASCT
• History of central nervous system (CNS) involvement with AML

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Treatment (busulfan, etoposide, ASCT)

Experimental group

Description:

Patients receive busulfan IV or oral every 6 hours on days -7 to -4 and etoposide IV on day -3. Patients then undergo autologous stem cell transplant on day 0.

Treatment:

Drug: Busulfan

Procedure: Autologous Hematopoietic Stem Cell Transplantation

Drug: Etoposide

Other: Laboratory Biomarker Analysis