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Sponsored by: |
Columbia University |
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Information provided by: | Columbia University |
ClinicalTrials.gov Identifier: | NCT00531310 |
CML, a malignant disorder of stem cells, is characterized by increases in both immature and mature myeloid, erythroid, and lymphoid cells, as well as platelets in the peripheral blood. The cytogenetic hallmark of CML is the Philadelphia(Ph)chromosome found in the malignant cells of 95% of patients. CML comprises 7-20% of all leukemias with an overall incidence in the general population estimated at 1 to 2 per 100,000. The peak incidence occurs in the fifth decade, however, all age groups, including children, are affected. The only reported environmental risk factor is exposure to excessive ionizing radiation that is documented in only a very small percentage of patients. Clinically, CML is characterized by an initial chronic phase in which patients may report mild constitutional symptoms; however, 40-50% are asymptomatic and are diagnosed based upon abnormal blood counts discovered during a routine examination. The chronic phase typically lasts three to five years, and is followed by an accelerated phase distinguished by progressive systemic symptoms, an increasing resistance to conventional chemotherapy, and a rise in the peripheral blood and bone marrow blast count. This evolves rapidly into a blastic crisis characterized by immature cells resembling the blasts characteristic of acute leukemia. The presence of 30% or more blastic cells in the blood or marrow is diagnostic of this final blastic phase which is typically fatal within 3 to 6 months.
The primary treatment options for CML have traditionally been monotherapy with either busulfan or hydroxyurea. Both agents are able to control the clinical symptoms associated with CML, as well as induce hematological remissions in 80% of chronic phase patients. However, complete cytogenetic remissions with either agent are rare, and neither is able to prevent eventual progression to the terminal blastic phase; therefore, these therapies can only be considered palliative.
The primary purpose of this clinical research trial is to study the feasibility of a reduced intensity allogenic transplant for CML. This study will also determine the side effects as well as the response rate.
Condition | Intervention | Phase |
---|---|---|
Chronic Myeloid Leukemia |
Drug: Reduced Intensity Conditioning |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Pilot Study of Reduced Intensity Allogeneic Stem Cell Transplantation in Patients With Chronic Myeloid Leukemia (CML) and Adoptive Cellular Immunotherapy Only in Patients With Persistent Disease and Matched Family Donors |
Estimated Enrollment: | 40 |
Study Start Date: | January 2003 |
Estimated Study Completion Date: | March 2008 |
Estimated Primary Completion Date: | December 2007 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Arm A: Experimental
Matched Family Donor
|
Drug: Reduced Intensity Conditioning
Busulfan and Fludarabine
|
Arm B: Experimental
Unrelated Donor Transplant/ Cord Blood Transplant
|
Drug: Reduced Intensity Conditioning
Busulfan and Fludarabine
|
Further study details will be provided by Columbia University, Division of Pediatric Blood and Marrow Transplantation.
STUDY DESIGN:
Patients less than 30 years of age with CML in 1st or 2nd chronic phase or 1st accelerated phase and a matched related donor, an unrelated cord blood donor, or an unrelated adult donor, will receive 6 days of IV Fludarabine, 4 doses of IV Busulfex, and 5 doses of Alemtuzumab. Patients with persistant RT-PCR positive BCR-Abl and/or Philadelphia chromosome positivity by cytogentics after Day + 100 and a matched related donor would receive DLIx1. If still BCR-Abl or Philadelphia chromosome positive at Day +180 and a matched related donor, patients would receive a second dose of DLI. All patients will receive STI-571 (Gleevec) after hematological reconstitution. Studies for immune reconstitution, chimersim, and MRD will be performed postt AlloSCT.
Ages Eligible for Study: | up to 30 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Adequate cardiac function defined as:
Adequate pulmonary function defined as:
-DLCO >40% by pulmonary function test For children who are uncooperative, no evidence of dyspnea at rest,no exercise intolerance, and a pulse oximetry >94% in room air.
Disease Status
Exclusion Criteria:
Patient in 3rd or greater accelerated phase
United States, New York | |
Morgan Stanley Children's Hospital of NYP | |
New York, New York, United States, 10032 |
Principal Investigator: | Mtchell S Cairo, MD | Columbia University Medical Center |
Responsible Party: | Columbia University ( Mitchell Cairo, MD ) |
Study ID Numbers: | CHNY-01-505, AAAA8191 |
Study First Received: | September 15, 2007 |
Last Updated: | June 3, 2008 |
ClinicalTrials.gov Identifier: | NCT00531310 |
Health Authority: | United States: Institutional Review Board |
CML allogeneic SCT matched family donor |
unrelated donor cord blood donor persistent disease |
Leukemia Chronic myelogenous leukemia Hematologic Diseases Busulfan Myeloproliferative Disorders |
Leukemia, Myelogenous, Chronic, BCR-ABL Positive Fludarabine Fludarabine monophosphate Leukemia, Myeloid Bone Marrow Diseases |
Neoplasms Neoplasms by Histologic Type |