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Phase IIA Window Study of Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia


Phase 2
6 Months
18 Years
Not Enrolling
Both
Leukemia

Thank you

Trial Information

Phase IIA Window Study of Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia


Etanercept blocks a hormone called Tumor Necrosis Factor (TNF), which has been shown to play
a role in helping the growth of leukemic cells in JMML.

Before participants can start treatment on this study, they will have what are called
"screening tests". These tests will help the doctor decide if patients are eligible to take
part in the study. You will have a complete medical history and physical exam. About two
tablespoons of blood and urine will be collected for routine tests as well as to test for
the liver and kidney function. You will have a bone marrow biopsy performed to monitor
disease activity. To collect a bone marrow biopsy, an area of the hip or chest bone is
numbed with anesthetic and a small amount of bone marrow and bone is withdrawn through a
large needle. You will have cells from inside of the mouth tested for genetic changes by
swabbing the side of the cheeks.

If you are found to be eligible, etanercept will be given as an injection under the skin
once a week for up to 90 days.

During the study, you will have weekly follow-up tests that will include physical exam and
lab tests. About 2 tablespoons of blood will be collected each time. Urine will also be
collected for testing at least every 3 weeks while on the study.

If the disease gets worse or intolerable side effects occur, you will be taken off study and
alternative treatment options will be discussed.

This is an investigational study. The FDA has approved etanercept for use in adults and
children with rheumatoid arthritis and juvenile rheumatoid arthritis. Its use in this study
is experimental. A total of up to 30 patients will take part in this study. All will be
enrolled at M. D. Anderson.


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.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Patient Response

Outcome Description:

Response is defined as a WBC < 15,000 and platelets > 75,000 at 12 weeks; toxicity is defined as a grade 3 or worse infection or non-hematologic toxicity within the first 4 weeks.

Outcome Time Frame:

12 weeks

Safety Issue:

No

Principal Investigator

Robert J. Wells, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

2004-0215

NCT ID:

NCT00509600

Start Date:

September 2004

Completion Date:

April 2009

Related Keywords:

  • Leukemia
  • Juvenile Myelomonocytic Leukemia
  • JMML
  • Leukemia
  • Etanercept
  • Enbrel
  • Leukemia
  • Leukemia, Myelomonocytic, Acute
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Juvenile

Name

Location
UT MD . Anderson Cancer Center Houston, Texas  77030