Skip to content

Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay

Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay

Status
Completed
Phases
NA
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT01872819
Enrollment
16
Registered
2013-06-07
Start date
2013-08-02
Completion date
2014-11-17
Last updated
2018-07-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Chronic Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts

Brief summary

This clinical trial uses a laboratory test called a high throughput sensitivity assay in planning treatment for patients with relapsed or refractory acute myeloid leukemia. The aim is to try to identify drugs that may be effective in killing leukemia cells for those patients who will not be cured with conventional chemotherapy. This assay will test multiple drugs simultaneously against a patient's own donated blood sample. The goal is to use this laboratory assay to best match a drug to a patient's disease.

Detailed description

PRIMARY OBJECTIVES: I. To obtain results from a high throughput drug sensitivity assay within 10 days, procure drug within 14 days and initiate treatment within 21 days. SECONDARY OBJECTIVES: I. To achieve a response (cytoreduction or at least partial response) greater that than expected for comparable refractory patient populations with other salvage regimens. OUTLINE: A patient receives a drug intervention based on the results of a high throughput sensitivity assay. This assay best matches a drug to the patient's disease.

Interventions

Undergo high throughput drug sensitivity assay

DRUGchemotherapy

Patients receive 1 of 160 possible interventions

BIOLOGICALbiological therapy

Patients receive 1 of 160 possible interventions

Sponsors

National Cancer Institute (NCI)
CollaboratorNIH
University of Washington
Lead SponsorOTHER

Study design

Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* Diagnosis of acute myeloid leukemia by World Health Organization (WHO) criteria (except acute promyelocytic leukemia), acute leukemias of ambiguous lineage by WHO criteria, or myelodysplastic syndrome refractory anemia with excess blasts (RAEB)-2 by WHO classification or advanced myeloproliferative neoplasm with \>= 10% blasts in the bone marrow or peripheral blood, including chronic myelomonocytic leukemia (CMML)-2 by WHO classification who have failed 2 inductions at initial diagnosis or failed \>= 2 salvage regimens for relapsed acute myeloid leukemia (AML) * Patients who have had a 1st remission for \>= 1 year must have received cytotoxic chemotherapy as a salvage regimen * Eastern Cooperative Oncology Group (ECOG) performance status 0 - 3 * Expectation that we can obtain about 100 million blasts from blood and/or marrow (for example, circulating blast count of 5,000 or greater) * Bilirubin =\< 1.5 x institutional upper limit of normal (IULN) unless elevation is thought to be due to Gilbert's syndrome, hemolysis, or hepatic infiltration by the hematologic malignancy * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) and serum pyruvate glutamate transaminase (SPGT) (alanine aminotransferase \[ALT\]) =\< 2.5 x IULN, unless elevation in thought to be due to hepatic infiltration by the hematologic malignancy * Alkaline phosphatase =\< 2.5 X ULN * Serum creatinine =\< 2.0 mg/dL * Stable or improving on appropriate antimicrobial therapy for infection, without ongoing fever * Informed consent * Willing to use contraception

Exclusion criteria

* No other concomitant treatment for leukemia * No other active cancer that requires systemic chemotherapy or radiation * Significant organ compromise that will increase risk of toxicity or mortality * Pregnancy or lactation

Design outcomes

Primary

MeasureTime frameDescription
Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AMLUp to 21 daysWhether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.

Secondary

MeasureTime frameDescription
Rate of Complete Response, Defined by Criteria of Cheson et al.Baseline up to 2 yearsNumber of patients who achieved a Complete Response (CR) with Minimal Residual Disease (MRD), a Complete Response with incomplete hematologic recovery (CRi), or showed reduced blasts in their bone marrow by flow cytometry (Cytoreduction). Cheson et al. defines a CR as: Bone Marrow blasts \<5%, absence of circulating blasts and blasts with Auer rods, absence of extramedullary disease, absolute neutrophil count \>1.0 x 10\^9/L, and platelet count \>100 x 10\^9/L. Cheson et al. defines a CRi as: all CR criteria except for residual neutropenia (\<1.0 x 10\^9/L) or thrombocytopenia (\<100 x 10\^9/L).

Countries

United States

Participant flow

Participants by arm

ArmCount
Treatment (Chemotherapy, Biological Therapy)
Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay. antitumor drug screening assay: Undergo high throughput drug sensitivity assay chemotherapy: Patients receive 1 of 160 possible interventions biological therapy: Patients receive 1 of 160 possible interventions
16
Total16

Baseline characteristics

CharacteristicTreatment (Chemotherapy, Biological Therapy)
Age, Categorical
<=18 years
0 Participants
Age, Categorical
>=65 years
6 Participants
Age, Categorical
Between 18 and 65 years
10 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
16 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
Race (NIH/OMB)
Asian
2 Participants
Race (NIH/OMB)
Black or African American
0 Participants
Race (NIH/OMB)
More than one race
0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
Race (NIH/OMB)
White
14 Participants
Region of Enrollment
United States
16 participants
Sex: Female, Male
Female
6 Participants
Sex: Female, Male
Male
10 Participants

Adverse events

Event typeEG000
affected / at risk
deaths
Total, all-cause mortality
— / —
other
Total, other adverse events
0 / 0
serious
Total, serious adverse events
11 / 14

Outcome results

Primary

Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML

Whether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.

Time frame: Up to 21 days

Population: 14 patients were treated.

ArmMeasureValue (MEDIAN)
Treated PatientsAchievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML5.1 days
Secondary

Rate of Complete Response, Defined by Criteria of Cheson et al.

Number of patients who achieved a Complete Response (CR) with Minimal Residual Disease (MRD), a Complete Response with incomplete hematologic recovery (CRi), or showed reduced blasts in their bone marrow by flow cytometry (Cytoreduction). Cheson et al. defines a CR as: Bone Marrow blasts \<5%, absence of circulating blasts and blasts with Auer rods, absence of extramedullary disease, absolute neutrophil count \>1.0 x 10\^9/L, and platelet count \>100 x 10\^9/L. Cheson et al. defines a CRi as: all CR criteria except for residual neutropenia (\<1.0 x 10\^9/L) or thrombocytopenia (\<100 x 10\^9/L).

Time frame: Baseline up to 2 years

Population: 14 patients received therapy. Out of 14 patients treated, 9 were evaluable (4 patients died prior to D14-21 marrow and 1 patient refused the D14-21 marrow).

ArmMeasureGroupValue (NUMBER)
Treated PatientsRate of Complete Response, Defined by Criteria of Cheson et al.CR with MRD1 Patients
Treated PatientsRate of Complete Response, Defined by Criteria of Cheson et al.CRi2 Patients
Treated PatientsRate of Complete Response, Defined by Criteria of Cheson et al.Cytoreduction6 Patients

Source: ClinicalTrials.gov · Data processed: Mar 10, 2026