Clinical trial NCT03515707: Stem Cell Transplant to Treat Patients With Favorable or Intermediate Risk Minimal Residual Disease Negative Acute Myeloid Leukemia
Official title: Autologous Transplant as Treatment for Favorable or Intermediate Risk MRD-Negative AML Patients After Initial Induction Therapy
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Minimal Residual Disease Negativity
Lead sponsor: Fred Hutchinson Cancer Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2018-05-03
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Relapse
timeFrame: Assessed up to 2 years post autologous stem cell transplant (ASCT)
measure: Treatment related mortality
timeFrame: From first dose of study therapy to day +100
Secondary outcomes:
measure: Disease-free survival
timeFrame: Assessed up to 4 years post-ASCT
measure: Overall survival
timeFrame: Assessed up to 4 years post-ASCT
Other outcomes:
Locations:
city: Seattle
country: United States
facility: Fred Hutch/University of Washington Cancer Consortium
state: Washington
Study officials:
affiliation: Fred Hutch/University of Washington Cancer Consortium
name: Leona A. Holmberg
role: PRINCIPAL_INVESTIGATOR
References: