Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors

October 7, 2010 updated by: Ann & Robert H Lurie Children's Hospital of Chicago

High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.

This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

Significant advances have been made in recent years in the treatment of solid tumors of childhood. However, much of the improvement in survival has been made in low stage and localized disease. Of significance is the fact that the improvements have come in up-front remission rates without translation into significantly high event-free survival(EFS) or overall survival (OS). This is despite the fact that these tumors as a whole are largely chemotherapy responsive.

Recent advances in the understanding of the biology of hematopoeitic stem cells have driven the design of treatment regimens that allow for dose intensification without unacceptable hematologic toxicity. Protocol development has focused on active agents that have a broad range between hematologic and non-hematologic toxicities. This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. This study utilizes PBSC to limit the risk of tumor cell contamination while retaining prompt hematologic recovery from these highly intensified treatments.

Study Type

Interventional

Enrollment (Anticipated)

12

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

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

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Recruiting
        • Children's Memorial 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 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Malignant Diseases:

    • Ewing's sarcoma/PNET:

      • CR1 - Metastatic disease at diagnosis, tumor volume > 100 ml, pelvic bone primary
      • CR2 - Locally recurrent disease

    • Soft tissue sarcoma

      • CR1 - Metastatic disease at diagnosis or locally advanced disease where local control is suboptimal (i.e., inability to provide radiation therapy due to extent of disease).
      • CR2 - Locally recurrent disease (VGPR2 acceptable)

    • Hepatoblastoma:

      • VGPR1 - Patients with metastatic disease at diagnosis who have a persistently elevated alpha FP, or unresectable primary as a way of converting to resectable.
      • CR2/VGPR2

    • Hodgkin's Disease:

      • VGPR1 - Progression on primary therapy/Refractory disease
      • CR2/VGPR2

    • Germ Cell Tumor:

      • CR2/VGPR2 - recurrent disease

    • Wilms Tumor:

      • CR2/VGPR2 - recurrent disease
  • IRB approved signed written informed consent by patient and/or their legally authorized guardian.
  • Patients 21 years of age or younger at initial diagnosis, with older patients considered individually for primary pediatric disease diagnosis.
  • Adequate central venous access (double lumen CVL or 2 single lumen PCVC).
  • Adequate PBSC harvests with a minimum of 2.0 x 108 MNC/kg available for each PBSC rescue.

  • Organ Function:

    • Platelets > 50,000/ml
    • SGOT < 10 x upper limits of normal
    • Creatinine < 1.5 x normal baseline
    • Normal cardiac function in accordance with institutional policies
    • Normal pulmonary function in accordance with institutional policies.

  • Physiologic status:

    • No active infections
    • Adequate performance status as measured by Karnofsky (> 70%) or Lansky scale (> 60%) as appropriate for age.

  • Bone Marrow Status

    • No evidence of morphologic involvement with tumor at the time of transplant

Off Study Criteria:

  • Severe toxicity. Contact the Study Coordinator immediately and complete Adverse Reaction Form.
  • Disease progression or relapse prior to PBSC #1 or between PBSC rescue # 1 and #2.
  • Inability to collect adequate numbers of PBSC for successful transplantation.
  • Patient or parent/guardian refusal to remain on study.

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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors.
Time Frame: annually
annually

Secondary Outcome Measures

Outcome Measure
Time Frame
Evaluate length of remission and long term disease free survival in chemotherapy responsive high-risk pediatric solid tumor patients treated using this approach.
Time Frame: Annually
Annually
Evaluate correlation between cell dose and time to engraftment in high-risk pediatric solid tumor patients treated using this approach.
Time Frame: Time to engraftment
Time to engraftment

Collaborators and Investigators

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

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Investigators

  • Principal Investigator: Morris Kletzel, M.D., Ann & Robert H Lurie Children's Hospital of Chicago

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

April 1, 1999

Primary Completion (Anticipated)

September 1, 2012

Study Completion (Anticipated)

September 1, 2012

Study Registration Dates

First Submitted

September 10, 2005

First Submitted That Met QC Criteria

September 10, 2005

First Posted (Estimate)

September 16, 2005

Study Record Updates

Last Update Posted (Estimate)

October 11, 2010

Last Update Submitted That Met QC Criteria

October 7, 2010

Last Verified

October 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMT 0499 Solid

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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