Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia
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| ClinicalTrials.gov Identifier: NCT00509600 |
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Recruitment Status :
Terminated
(Slow enrollment; study terminated.)
First Posted : July 31, 2007
Results First Posted : December 2, 2010
Last Update Posted : August 7, 2012
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Leukemia | Drug: Etanercept | Phase 2 |
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marrow biopsy, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow and bone is withdrawn through a large needle.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 Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 1 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Phase IIA Window Study of Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia |
| Study Start Date : | September 2004 |
| Actual Primary Completion Date : | April 2009 |
| Actual Study Completion Date : | April 2009 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Etanercept
0.8 mg/kg subcutaneously weekly for 90 days
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Drug: Etanercept
0.8 mg/kg Subcutaneously Once A Week for 90 Days
Other Name: Enbrel |
- Patient Response [ Time Frame: 12 weeks ]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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 6 Months to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
- 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.
- Adequate hepatic function (bilirubin equal or less than 2.0 mg/dl; ALT equal or less than 3x normal)
- Adequate renal function (serum creatinine equal or less than 2 x normal)
- Performance Status: Have a Karnofsky score >50.
- Written, informed consent according to institution guidelines.
Exclusion Criteria:
- Pregnant or lactating.
- 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.
- Febrile neutropenia at study entry.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00509600
| United States, Texas | |
| UT MD . Anderson Cancer Center | |
| Houston, Texas, United States, 77030 | |
| Principal Investigator: | Robert J. Wells, MD | M.D. Anderson Cancer Center |
| Responsible Party: | M.D. Anderson Cancer Center |
| ClinicalTrials.gov Identifier: | NCT00509600 |
| Other Study ID Numbers: |
2004-0215 |
| First Posted: | July 31, 2007 Key Record Dates |
| Results First Posted: | December 2, 2010 |
| Last Update Posted: | August 7, 2012 |
| Last Verified: | August 2012 |
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Juvenile Myelomonocytic Leukemia JMML Leukemia Etanercept Enbrel |
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Leukemia Leukemia, Myelomonocytic, Juvenile Neoplasms by Histologic Type Neoplasms Leukemia, Myeloid Myelodysplastic-Myeloproliferative Diseases Bone Marrow Diseases Hematologic Diseases Etanercept Anti-Inflammatory Agents, Non-Steroidal |
Analgesics, Non-Narcotic Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents Antirheumatic Agents Gastrointestinal Agents Immunosuppressive Agents Immunologic Factors |