Chemotherapy With CD133+ Select Autologous Hematopoietic Stem Cells for Children With Solid Tumors and Lymphomas

This study has been completed.
Sponsor:
Collaborator:
University of Miami
Information provided by:
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00152126
First received: September 7, 2005
Last updated: February 12, 2009
Last verified: February 2009
  Purpose

Studies have provided evidence that residual microscopic malignant cells in autologous bone marrow or blood stem cell grafts can contribute to posttransplant relapse. Researchers are currently exploring different methods in an attempt to purify or "purge" the stem cell product to minimize the risk of tumor contamination.

The CD133+ antigen is a protein contained on or "expressed" on numerous cells in the human body including specific hematopoietic progenitor (blood forming) cells. However, this antigen is not expressed on certain cancer cells including neuroblastoma. A technique using the investigational CliniMACS cell sorting device has been developed in an effort to filter out only those stem cells that express this CD133+ antigen in order to infuse a hematopoietic stem cell product with no tumor contamination potential.

The primary objective of this study is to establish safety of treating patients with a high dose chemotherapy regimen of Busulfan and Melphalan followed by autologous CD133+ hematopoietic stem cell support. Transplants recipients are expected to achieve engraftment as defined by an absolute neutrophil count of greater than or equal to 500/mm3 for three consecutive days by day 42-post infusion. Thus, safety of the treatment plan will be evaluated in terms of failure to engraft by this specific time period.


Condition Intervention
Neuroblastoma
Central Nervous System Tumors
Lymphomas
Wilms Tumor
Procedure: Stem Cell Transplantation
Drug: Busulfan, Melphalan

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • 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. [ Time Frame: August 2005 ] [ Designated as safety issue: Yes ]

Enrollment: 26
Study Start Date: August 2003
Study Completion Date: February 2009
Primary Completion Date: August 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1 Procedure: Stem Cell Transplantation
Autologous stem cell transplantation
Drug: Busulfan, Melphalan
Transplant recipients will receive high dose Busulfan and Melphalan followed by autologous CD133+ antigen specific hematopoietic stem cell infusion. The autologous graft product will be selected using the investigational CliniMACS device.
Other Names:
  • Autologous stem cell transplant
  • CD133+ antigen specific selection
  • Apheresis

Detailed Description:

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.
  Eligibility

Ages Eligible for Study:   up to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00152126

Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
University of Miami
Investigators
Principal Investigator: Gregory Hale, M.D. St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: Gregory Hale, MD / Principal Investigator, St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00152126     History of Changes
Other Study ID Numbers: ST133
Study First Received: September 7, 2005
Last Updated: February 12, 2009
Health Authority: United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:
Autologous stem cell transplantation
CD133 cell selection
CliniMACS device
Tumor marker
Tumor purging

Additional relevant MeSH terms:
Lymphoma
Wilms Tumor
Nervous System Neoplasms
Neuroblastoma
Central Nervous System Neoplasms
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Complex and Mixed
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Neoplastic Syndromes, Hereditary
Kidney Diseases
Urologic Diseases
Genetic Diseases, Inborn
Nervous System Diseases
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Busulfan
Melphalan

ClinicalTrials.gov processed this record on July 29, 2014