Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia

• Status: Terminated
• Trial end date: 2009-04-01
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objectives:

1.1 Estimate rate of response and define acute toxicity to etanercept used in an up-front phase II window in newly diagnosed or relapsed JMML.

1.2 Determine if response to Tumor Necrosis Factor (TNF) blockade correlates with genetic basis of Juvenile Myelomonocytic Leukemia (JMML) [mutations in NF1, Ras, SHP2] or levels of TNFa.

1.3 Determine if TNF blockade by etanercept results in inhibition of free levels of TNFa and other cytokines by ELISA and bioassay and improves blood counts.

1.4 Estimate the two year event free survival and overall survival in JMML patients following etanercept and allogeneic hematopoietic stem cell transplantation.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: M.D. Anderson Cancer Center
• Collaborator: Amgen
• Treatments: Etanercept

Criteria

Inclusion Criteria:

1. All children greater than 6 months of age and less than 18 years of age with newly-diagnosed previously untreated or previously diagnosed JMML, which has reoccurred after treatment with chemotherapy, stem cell transplantation, and/or cis-retinoic acid.

2. A diagnosis of JMML is confirmed only if the following criteria for JMML are met: a) ALL of the following: Absence of t(9;22) or BCR-ABL by PCR or FISH; Absolute monocyte count >1000 (1 X 109/µL); <20% bone marrow blasts; b) At least 2 of the following: Elevated Hb F hemoglobin; Myeloid precursors in peripheral blood; WBC >10,000 (10 X 109/µL); GM-CSF hypersensitivity in methylcellulose culture of bone marrow progenitors cells.

3. Adequate hepatic function (bilirubin equal or less than 2.0 mg/dl; ALT equal or less than 3x normal)

4. Adequate renal function (serum creatinine equal or less than 2 x normal)

5. Performance Status: Have a Karnofsky score >50.

6. Written, informed consent according to institution guidelines.

Exclusion Criteria:

1. Pregnant or lactating.

2. Receiving any other chemotherapy. Patients must have been off chemotherapy for at least 2 weeks and must have recovered from acute toxicity of all previous therapy prior to enrollment.

3. Febrile neutropenia at study entry.