Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia
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.
Description
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marrow biopsy, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow and bone is withdrawn through a large needle.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.
Study Design
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Conditions
Leukemia
Intervention
Etanercept
Location
UT MD . Anderson Cancer Center
Houston
Texas
United States
77030
Status
Terminated
Source
M.D. Anderson Cancer Center
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT00509600
- Information obtained from ClinicalTrials.gov on August 29, 2012
Medical and Biotech [MESH] Definitions
Abelson Murine Leukemia Virus
A replication-defective strain of Murine leukemia virus (LEUKEMIA VIRUS, MURINE) capable of transforming lymphoid cells and producing a rapidly progressing lymphoid leukemia after superinfection with FRIEND MURINE LEUKEMIA VIRUS; MOLONEY MURINE LEUKEMIA VIRUS; or RAUSCHER VIRUS.
Friend Murine Leukemia Virus
A strain of Murine leukemia virus (LEUKEMIA VIRUS, MURINE) producing leukemia of the reticulum-cell type with massive infiltration of liver, spleen, and bone marrow. It infects DBA/2 and Swiss mice.
Moloney Murine Leukemia Virus
A strain of Murine leukemia virus (LEUKEMIA VIRUS, MURINE) arising during the propagation of S37 mouse sarcoma, and causing lymphoid leukemia in mice. It also infects rats and newborn hamsters. It is apparently transmitted to embryos in utero and to newborns through mother's milk.
Leukemia, Prolymphocytic
A chronic leukemia characterized by a large number of circulating prolymphocytes. It can arise spontaneously or as a consequence of transformation of CHRONIC LYMPHOCYTIC LEUKEMIA.
Leukemia, Prolymphocytic, T-cell
A lymphoid leukemia characterized by a profound LYMPHOCYTOSIS with or without LYMPHADENOPATHY, hepatosplenomegaly, frequently rapid progression, and short survival. It was formerly called T-cell chronic lymphocytic leukemia.
Clinical Trials
A Multi-center Study to Evaluate the Safety of Etanercept in the Treatment of Lichen Planus
This is a research study whose purpose is to test the effectiveness of etanercept (Enbrel) in the treatment of moderate to severe lichen planus. No studies have been done on the effective...
Study Evaluating Etanercept in Rheumatoid Arthritis, Ankylosing Spondylitis and Psoriatic Arthritis
This is a Phase 4 open label, non-interventional, multi-center study to evaluate the safety of Enbrel (etanercept) treatment in patients receiving etanercept 25mg sc twice weekly or 50mg o...
The Efficacy and Safety of Adding Methotrexate to Etanercept in Psoriasis
The purpose of this study is to evaluate the efficacy of adding methotrexate to etanercept compared with etanercept monotherapy as measured by the proportion of subjects achieving a 75% im...
The purpose of this study is to evaluate the efficacy of re-treatment with etanercept 50mg subcutaneous injections twice weekly for 12 weeks in subjects who have previously been treated et...
Evaluating the Safety of Etanercept 50 mg Twice Weekly in Subjects With Psoriasis
The purpose of this study is to determine the effect of etanercept 50 mg twice weekly compared to placebo over 12 weeks on the psoriasis area and severity index (PASI) in subjects with pso...
PubMed Articles
We report a 16-year-old female case of intractable adult-onset Still's disease accompanied by macrophage activation syndrome, who went into full remission after switching from infliximab to etanercept...
We determined the effects of etanercept on the serum concentrations of neuropeptides in RA patients. In a total of 11 patients who had been injected with etanercept, the serum levels of substance P, c...
Expression of antimicrobial peptides and proteins in etanercept-treated psoriasis patients.
Recent papers highlight the role of dysregulated expression of antimicrobial peptides and proteins (AMPs) in the pathogenesis of psoriasis. Etanercept, a blocker of the pro-inflammatory cytokine tumou...
Abstract Background Knowledge on sequential treatment with biologics for psoriasis regarding efficacy and safety is sparse. This also applies to the efficacy and safety of adalimumab in patients previ...
