Combination Chemotherapy Followed By Donor Bone Marrow or Umbilical Cord Blood Transplant in Treating Children With Newly Diagnosed Juvenile Myelomonocytic Leukemia
NCT ID: NCT00025038
Last Updated: 2013-04-11
Study Results
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Basic Information
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COMPLETED
PHASE2
100 participants
INTERVENTIONAL
2001-06-30
Brief Summary
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Detailed Description
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I. Determine the response rate of children with newly diagnosed juvenile myelomonocytic leukemia treated with R115777, isotretinoin, cytarabine, and fludarabine followed by allogeneic bone marrow or umbilical cord blood transplantation.
II. Determine the safety and toxicity of this regimen in these patients. III. Determine the tolerability of this regimen in these patients. IV. Determine the rate of 2-year event-free survival of patients treated with this regimen.
V. Determine whether prognostic subsets of these patients can be identified based on expression of clinical, genetic (NFI, monosomy 7, RAS gene), or hematopoietic characteristics.
OUTLINE: This is a multicenter study.
Patients may choose to receive upfront window induction therapy with oral R115777 twice daily on days 1-21. Treatment repeats every 28 days for 2 courses in the absence of disease progression or unacceptable toxicity.
Patients with progressive disease or stable disease with unacceptable hematopoietic recovery after 1 course proceed to induction chemotherapy. (R11577 portion of the study closed to accrual as of 08/2005)
All patients receive induction chemotherapy comprising oral isotretinoin once daily beginning on day 1 and fludarabine IV over 30 minutes and cytarabine IV over 4 hours on days 1-5. Treatment with fludarabine and cytarabine repeats every 28 days for 2 courses. Treatment with isotretinoin continues until allogeneic bone marrow or umbilical cord blood transplantation. Patients with progressive disease after 1 course proceed to transplantation.
After completion of isotretinoin, patients receive a preparative regimen comprising total body irradiation twice daily on days -7 to -4, cyclophosphamide IV over 2 hours on days -3 and -2, and anti-thymocyte globulin IV over 4-6 hours every 12 hours on days -3 to -1. Patients undergo allogeneic bone marrow or umbilical cord blood transplantation on day 0. Patients receive oral isotretinoin daily beginning on approximately day 60 and continuing for 1 year.
Patients are followed every 6 months for 5 years and then annually thereafter.
PROJECTED ACCRUAL: A maximum of 100 patients (18-46 receiving R115777 with induction chemotherapy \[R11577 portion of the study closed to accrual as of 08/2005\] and 27-54 receiving induction chemotherapy only) will be accrued for this study within 3.2 years.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Treatment (tipifarnib, bone marrow/umbilical cord transplant)
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tipifarnib
Given orally
isotretinoin
Given orally
fludarabine phosphate
Given IV
cytarabine
Given IV
radiation therapy
Undergo total body irradiation
cyclophosphamide
Given IV
anti-thymocyte globulin
Given IV
allogeneic bone marrow transplantation
Undergo allogeneic bone marrow transplant
double-unit umbilical cord blood transplantation
umbilical cord blood transplantation
Undergo allogeneic cord blood transplant
laboratory biomarker analysis
Correlative studies
Interventions
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tipifarnib
Given orally
isotretinoin
Given orally
fludarabine phosphate
Given IV
cytarabine
Given IV
radiation therapy
Undergo total body irradiation
cyclophosphamide
Given IV
anti-thymocyte globulin
Given IV
allogeneic bone marrow transplantation
Undergo allogeneic bone marrow transplant
double-unit umbilical cord blood transplantation
umbilical cord blood transplantation
Undergo allogeneic cord blood transplant
laboratory biomarker analysis
Correlative studies
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Presenting with all of the following:
* Absence of t(9;22) or bcr/abl by PCR
* Absolute monocyte count greater than 1,000/mm\^3
* Less than 20% bone marrow blasts
* Presenting with at least 2 of the following:
* Elevated F hemoglobin
* Myeloid precursors in peripheral blood
* WBC greater than 10,000/mm\^3
* Sargramostim (GM-CSF) hypersensitivity
* See Disease Characteristics
* Bilirubin no greater than 2.0 mg/dL
* ALT no greater than 3 times normal
* Creatinine no greater than 2 times normal
* No concurrent sargramostim (GM-CSF)
* No concurrent proton pump inhibitors
18 Years
ALL
No
Sponsors
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National Cancer Institute (NCI)
NIH
Responsible Party
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Principal Investigators
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Robert Castleberry
Role: PRINCIPAL_INVESTIGATOR
Children's Oncology Group
Locations
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Children's Oncology Group
Arcadia, California, United States
Countries
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References
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Stieglitz E, Gu CJ, Richardson M, Kita R, Santaguida MT, Ali KA, Strachan DC, Dhar A, Yam G, Anderson W, Anderson E, Hubner J, Tasian SK, Loh ML, Lacher MD. Tretinoin Enhances the Effects of Chemotherapy in Juvenile Myelomonocytic Leukemia Using an Ex Vivo Drug Sensitivity Assay. JCO Precis Oncol. 2023 Sep;7:e2300302. doi: 10.1200/PO.23.00302.
Other Identifiers
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AAML0122
Identifier Type: -
Identifier Source: secondary_id
CDR0000068788
Identifier Type: REGISTRY
Identifier Source: secondary_id
NCI-2012-01861
Identifier Type: -
Identifier Source: org_study_id
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