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

NCT ID: NCT03515707

Last Updated: 2021-02-01

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-10
• Study Completion Date: 2022-07-30

Brief Summary

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.

Detailed 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.

Conditions

Acute Myeloid Leukemia; Minimal Residual Disease Negativity

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (busulfan, etoposide, ASCT)

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.

Group Type: EXPERIMENTAL

Autologous Hematopoietic Stem Cell Transplantation

Intervention Type: PROCEDURE

Undergo ASCT

Busulfan

Intervention Type: DRUG

Given IV or oral

Etoposide

Intervention Type: DRUG

Given IV

Laboratory Biomarker Analysis

Intervention Type: OTHER

Correlative studies

Interventions

Autologous Hematopoietic Stem Cell Transplantation

Undergo ASCT

Intervention Type: PROCEDURE

Busulfan

Given IV or oral

Intervention Type: DRUG

Etoposide

Given IV

Intervention Type: DRUG

Laboratory Biomarker Analysis

Correlative studies

Intervention Type: OTHER

Other Intervention Names

Autologous Hematopoietic Cell Transplantation; Autologous Stem Cell Transplantation; 1, 4-Bis[methanesulfonoxy]butane; BUS; Bussulfam; Busulfanum; Busulfex; Busulphan; CB 2041; CB-2041; Glyzophrol; GT 41; GT-41; Joacamine; Methanesulfonic Acid Tetramethylene Ester; Methanesulfonic acid, tetramethylene ester; Mielucin; Misulban; Misulfan; Mitosan; Myeleukon; Myeloleukon; Myelosan; Mylecytan; Myleran; Sulfabutin; Tetramethylene Bis(methanesulfonate); Tetramethylene bis[methanesulfonate]; WR-19508; Demethyl Epipodophyllotoxin Ethylidine Glucoside; EPEG; Lastet; Toposar; Vepesid; VP 16-213; VP-16; VP-16-213

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 69 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Fred Hutchinson Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Leona A. Holmberg

Role: PRINCIPAL_INVESTIGATOR

Fred Hutch/University of Washington Cancer Consortium

Locations

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, United States

Countries

United States

Other Identifiers

NCI-2017-02069

Identifier Type: REGISTRY

Identifier Source: secondary_id

9784

Identifier Type: OTHER

Identifier Source: secondary_id

P30CA015704

Identifier Type: NIH

Identifier Source: secondary_id

View Link

RG9217025

Identifier Type: OTHER

Identifier Source: secondary_id

9784

Identifier Type: -

Identifier Source: org_study_id