Clinical trial NCT01321385: Biomarkers in Predicting Response to Treatment in Bone Marrow Samples From Young Patients With Acute Myeloid Leukemia
Official title: Validation of a Classifier for Induction Response Prediction Using Single Cell Network Profiling (SCNP) Assays for Childhood AML
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Leukemia
Keywords: childhood acute myeloid leukemia in remission
recurrent childhood acute myeloid leukemia
untreated childhood acute myeloid leukemia and other myeloid malignancies
childhood acute erythroleukemia (M6)
childhood acute megakaryocytic leukemia (M7)
childhood acute monoblastic leukemia (M5a)
childhood acute monocytic leukemia (M5b)
childhood acute myeloblastic leukemia with maturation (M2)
childhood acute myeloblastic leukemia without maturation (M1)
childhood acute myelomonocytic leukemia (M4)
childhood acute minimally differentiated myeloid leukemia (M0)
Lead sponsor: Children's Oncology Group
Sponsor class: NETWORK
Collaborators: name: National Cancer Institute (NCI)
First posted: 2011-03-23
Enrollment: 97
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Diagnosis of acute myeloid leukemia.

Brief summary: RATIONALE: Studying samples of bone marrow from patients with cancer in the laboratory may help doctors predict how well patients will respond to treatment.

PURPOSE: This research trial is studying biomarkers in predicting response to treatment in bone marrow samples from young patients with acute myeloid leukemia.

Detailed description: OBJECTIVES:

Primary

* To validate the accuracy of a pre-specified acute myeloid leukemia (AML) induction response classifier or the My Profile™ AML Induction Therapy Assay (run in a Good Laboratory Practice \[GLP\] laboratory using Good Manufacturing Practice \[GMP\] reagents) in predicting response to cytarabine-based induction chemotherapy in pediatric patients with non-M3 AML.

Secondary

* To validate the continuous score from the pre-specified induction response classifier as a predictor of response to induction chemotherapy, after controlling for the simultaneous effects of clinical variables at base-line and/or pre-induction therapy (e.g., age, WBC, and percentage \[%\] of blasts), and tests commonly available after the start of induction therapy (e.g., cytogenetics and molecular markers).
* To validate the accuracy of the pre-specified induction response classifier as a binary predictor of induction response using a pre-specified cutpoint to assign patients to no response (NR) or complete response (CR) groups.
* To validate the accuracy of the pre-specified induction response classifier as a binary predictor of induction response using a pre-specified cutpoint to assign patients to NR or CR groups, after controlling for the simultaneous effects of clinical variables at base-line (pre-induction therapy) (e.g., age, WBC, % blasts) and clinical variables, including tests commonly available after start of induction therapy (e.g., age, WBC, cytogenetics).

OUTLINE: Cryopreserved bone marrow mononuclear cell specimens are analyzed by cell networking profiling assays. Results are then compared with patient outcomes and demographics from COG-AAML03P1 and COG-AAML0531 studies.

Molecular markers analyzed include Flt3ITD, NPM1, CEBPA, and MRA.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Diagnosis of acute myeloid leukemia (AML)

  * Non-M3 AML
* Cryopreserved bone marrow mononuclear cell specimens from patients enrolled on COG-AAML03P1 and COG-AAML0531 collected prior to cytarabine-based induction therapy

  * Patient samples from COG-AAML0531 must come from the control arm (i.e., no gemtuzumab ozogamicin induction therapy)

PATIENT CHARACTERISTICS:

* No Down syndrome

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics

Primary outcomes:
measure: Accuracy of induction response prediction
Secondary outcomes:
measure: Duration of complete response
measure: Event-free survival and overall survival
measure: Status (dead vs living)
measure: Date of last follow up
Other outcomes:
Locations:
Study officials:
affiliation: Stanford University
name: Norman J. Lacayo, MD
role: PRINCIPAL_INVESTIGATOR
References: