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

February 12, 2009 updated by: St. Jude Children's Research Hospital

Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

26

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 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:
  • Apheresis
  • Autologous stem cell transplant
  • CD133+ antigen specific selection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
August 2005

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

St. Jude Children's Research Hospital

Collaborators

University of Miami

Investigators

  • Principal Investigator: Gregory Hale, M.D., St. Jude Children's Research Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2003

Primary Completion (Actual)

August 1, 2005

Study Completion (Actual)

February 1, 2009

Study Registration Dates

First Submitted

September 7, 2005

First Submitted That Met QC Criteria

September 7, 2005

First Posted (Estimate)

September 9, 2005

Study Record Updates

Last Update Posted (Estimate)

February 13, 2009

Last Update Submitted That Met QC Criteria

February 12, 2009

Last Verified

February 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ST133

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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