Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas
N/A
25 Years
Both
Neuroblastoma, Central Nervous System Tumors, Lymphomas, Wilms Tumor
Trial Information
Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas
Secondary objectives for this protocol include the following:
- To describe CD133+ graft content post-selection and to describe the yield and purity of
CD133+ content of the graft obtained.
- To describe the negative selection efficiency of this strategy by assessing the
processed product for tumor specific markers, when applicable.
- To characterize the proliferation of clonal progeny of CD133+ cells.
- To characterize lymphocyte and hematopoietic reconstitution (including the kinetics of
platelet engraftment) in these patients.
- To estimate one-year disease-free and overall survival in these transplant recipients.
Inclusion Criteria:
Eligibility will be determined separately for Part I and Part II of this study:
Part I ( Part I Eligibility criteria (eligibility for undergoing apheresis procedure)
- Age ≤ 25 years at initial diagnosis.
- Must have one of the following diagnoses:
- High risk neuroblastoma
- Metastatic or recurrent retinoblastoma
- High risk rain tumors
- Recurrent or refractory Hodgkin disease
- Recurrent or advanced stage Wilms tumor
- Recurrent or metastatic sarcomas
- Recurrent or refractory non-Hodgkin lymphoma
- Desmoplastic small round cell tumor.
- Lansky or Karnofsky Performance Score ≥ 70.
- Creatinine ≤ 2.0 mg/dl.
- Direct bilirubin ≤ 2.0 mg/dl.
- SGPT ≤ 2 x upper limit of normal
- HIV testing
- Negative pregnancy test
- Patients with significant prior radiation therapy to the liver will be excluded.
Part II eligibility criteria (criteria for transplantation of CD133 select stem cell
product)
- Successfully completed Part I of protocol treatment plan and has the following
available:
- Stored autologous bone marrow or peripheral blood stem cells (i.e. 2 x 106 unselected
CD34+ cells/ kg PBSC or 1 x 106 CD34+ cells/ kg BM) for back up.
- Stored autologous bone marrow or peripheral blood stem cells (2 x 106 CD133+ cells/
kg PBSC or 2 x 106 CD133+ cells/ kg BM) for infusion.
- Forced vital capacity greater than or equal to 40% normal or pulse oximetry greater
than or equal to 92% on room air.
- Lansky or Karnofsky Performance Score ≥ 70.
- Creatinine ≤ 2.0 mg/dl.
- Direct bilirubin ≤ 2.0 mg/dl.
- SGPT ≤ 2 x upper limit of normal
- Negative pregnancy test
- Patients with significant prior radiation therapy (in opinion of the PI) to the liver
will be excluded.
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
To determine the safety of the treatment plan using Busulfan and Melphalan followed by infusion of CD133+ selected hematopoietic cells in patients with high-risk malignancies.
Outcome Time Frame:
August 2005
Safety Issue:
Yes
Principal Investigator
Gregory Hale, M.D.
Investigator Role:
Principal Investigator
Investigator Affiliation:
St. Jude Children's Research Hospital
Authority:
United States: Food and Drug Administration
Study ID:
ST133
NCT ID:
NCT00152126
Start Date:
August 2003
Completion Date:
February 2009
Related Keywords:
- Neuroblastoma
- Central Nervous System Tumors
- Lymphomas
- Wilms Tumor
- Autologous stem cell transplantation
- CD133 cell selection
- CliniMACS device
- Tumor marker
- Tumor purging
- Lymphoma
- Wilms Tumor
- Nervous System Neoplasms
- Neuroblastoma
- Central Nervous System Neoplasms
Name | Location |
|---|---|
| St. Jude Children's Research Hospital | Memphis, Tennessee 38105-2794 |
