LeukemiaPhase 2NCT00509600
Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia
A Phase 2 study testing Etanercept in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Leukemia
- What is being tested?
- Etanercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2004to Apr 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
UT MD . Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Phase IIA Window Study of Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia
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.
Read the detailed 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.
- Conditions
- Leukemia
- Being tested
- Etanercept
- Keywords
- Juvenile Myelomonocytic Leukemia, JMML, Leukemia, Etanercept, Enbrel
- Medical subject headings
- Leukemia, Leukemia, Myelomonocytic, Juvenile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EtanerceptDrug
Also called Enbrel
0.8 mg/kg Subcutaneously Once A Week for 90 Days
| Study group | Role | What they receive |
|---|---|---|
| Etanercept | Experimental | 0.8 mg/kg subcutaneously weekly for 90 days Receives: Drug: Etanercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient Response
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.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2007 (estimated)
- Study startSep 2004
- Primary completionApr 2009
- Study completionApr 2009
Why it stopped: Slow enrollment; study terminated.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- Robert J. Wells, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00509600
- Sponsor's protocol ID
- 2004-0215
- Status last verified
- Aug 2012
- First submitted
- 27 Jul 2007
- Last updated
- 7 Aug 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIA Window Study of Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia
- ClinicalTrials.gov ID
- NCT00509600
- Acronym / sponsor ID
- 2004-0215
- Conditions
- Leukemia
- Keywords
- Juvenile Myelomonocytic Leukemia, JMML, Leukemia, Etanercept, Enbrel
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow enrollment; study terminated.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Amgen
- Study start
- Sep 2004
- Primary completion
- Apr 2009 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 31 Jul 2007 (estimated)
- Results
- Posted 2 Dec 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Etanercept | Experimental | Drug: Etanercept0.8 mg/kg subcutaneously weekly for 90 days |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EtanerceptAlso: Enbrel0.8 mg/kg Subcutaneously Once A Week for 90 Days | Drug | Etanercept |
Primary outcome
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- UT MD Anderson Cancer Center websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert J. Wells, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Etanercept in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Aug 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Dec 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Patient Response (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2012.