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

University of Washington

Status: Completed

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

Treatments: Biological: biological therapy; Other: antitumor drug screening assay; Drug: chemotherapy

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT01872819

NCI-2013-01070 (Registry Identifier)

P30CA015704 (U.S. NIH Grant/Contract)

8003 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

Treatment (chemotherapy, biological therapy)

Experimental group

Description:

Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay.

Treatment:

Biological: biological therapy

Drug: chemotherapy

Other: antitumor drug screening assay