Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
N/A
21 Years
Both
Tumors, Malignant Melanoma, Hematological Malignancies, Myelogenous Leukemia, Chronic, Leukemia, Lymphoblastic, Acute
Trial Information
Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
The standard treatment in many disorders of the bone marrow is high dose chemotherapy and
whole-body radiation treatment followed by the stem cell transplant. This type of
transplant not only suppresses or kills off the immune system, but is very toxic to the bone
marrow. This study uses a chemotherapy regimen that will suppress the patient's immune
system; however, it is non-myeloablative (not toxic to the bone marrow). It does not use
whole-body radiation treatment. This approach can minimize the short- and long-term effects
of transplantation. Other studies have shown that using chemotherapy followed by bone
marrow transplantation without whole-body radiation can produce similar results as treatment
with whole-body radiation.
Patients will be given chemotherapy with Fludarabine and Busulfan prior to the stem cell
transplant. This treatment not only destroys diseased cells, but it also kills normal bone
marrow cells. Following this experimental treatment, the patient will be given the stem
cells through a central venous catheter (tube inserted in a vein). When the healthy stem
cells are given to the patient, they will replace the destroyed bone marrow cells and
produce new blood cells. The Allogeneic (not one's own) stem cells used in this
experimental transplant will be obtained from a related matched donor or from an unrelated
matched donor located through the National Marrow Donor Program.
Inclusion Criteria:
- Patients with recurrent solid tumors
- Patients with malignant melanoma
- Patients with hematological malignancies.
- Chronic myelogenous leukemia in chronic or accelerated phase, to include chronic
myelomonocytic leukemia (juvenile chronic myelogenous leukemia [JCML] or CMML).
- Acute lymphoblastic leukemia (ALL)
- First remission high-risk ALL (Ph+ with initial high white blood cell
[WBC]; t (4-11) in infants less than 1 year and CALLA negative)
- Second or subsequent remission ALL or isolated extramedullary disease on or
off therapy.
- Acute non-lymphocytic leukemia (ANLL)
- Patients with ANLL in first remission who have a matched sibling donor.
- ANLL in second remission, or patients who only achieve an initial partial
remission < 15% blasts, or early relapse.
- Myelodysplastic syndromes (MDS): refractory anemia (RA), refractory anemia with
excess blasts (RAEB), refractory anemia with excess blasts in transformation
(RAEB-T) and CMML/JCML.
- Selected immunodeficiencies:
- Wiskott-Aldrich syndrome.
- Severe combined immunodeficiency variants that require ablation.
- Hyper-IGM syndrome.
- Other immune deficiencies after approval from the medical director.
- Bone marrow failure syndromes (single or multiple hematopoietic lines)
- Venous access: A double lumen central vascular access device or its equivalent will
be required for all patients entered on the protocol.
- Informed consent: The donor and the patient and/or the patient's legally authorized
guardian must acknowledge in writing that consent to become a study subject has been
obtained in accordance with the institutional policy approved by the United States
(U.S.) Department of Health and Human Services.
- Patient organ function requirements:
- Adequate renal function: serum creatinine < 2 x normal, or creatinine clearance
calculated by Schwartz formula, of glomerular filtration rate (GFR) > 40
ml/min/1.73m2, or an equivalent GFR as determined by the institutional normal
range.
- Adequate liver function: total bilirubin (ALT)
- Adequate cardiac function: shortening fraction of > 24% by echocardiogram, or
ejection fraction of > 30% by radionuclide angiogram.
- Adequate pulmonary function: DLCO, FEV1 / FVC > 30% by pulmonary function test.
For children who are uncooperative for pulmonary function tests and have no
evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on
room air is considered acceptable.
- Performance status: Lansky >/= 60% for children Karnofsky > 60% status for those > 16 years of age.
Exclusion Criteria:
- Patients who are pregnant
- Inability to find a suitable donor for the patient
- Patient is HIV-positive
- Patient has active Hepatitis B
- Disease progression or relapse prior to HPC infusion
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ rich HPCs after a reduced intensity conditioning regimen.
Outcome Time Frame:
To study end
Safety Issue:
Yes
Principal Investigator
Morris Kletzel, M.D.
Investigator Role:
Principal Investigator
Investigator Affiliation:
Ann & Robert H Lurie Children's Hospital of Chicago
Authority:
United States: Institutional Review Board
Study ID:
BMT 0300 Mini
NCT ID:
NCT00179764
Start Date:
March 2000
Completion Date:
March 2012
Related Keywords:
- Tumors
- Malignant Melanoma
- Hematological Malignancies
- Myelogenous Leukemia, Chronic
- Leukemia, Lymphoblastic, Acute
- Patients with recurrent solid tumors
- Patients with malignant melanoma
- Patients with hematological malignancies
- Acute lymphoblastic leukemia (ALL)
- Acute myelogenous leukemia (AML)
- Neoplasms
- Leukemia
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
- Leukemia, Myeloid
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
- Melanoma
- Hematologic Neoplasms
- Chronic Disease
Name | Location |
|---|---|
| Children's Memorial Hospital | Chicago, Illinois 60614 |
