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

Study Description

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Ewing's Sarcoma; Soft Tissue Sarcoma; Hepatoblastoma; Hodgkin's Disease; Germ Cell Tumor	Drug: High-Dose Chemotherapy with Tandem PBSC Rescue.	Phase 1; Phase 2

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 Design

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	12 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.
Study Start Date :	April 1999
Estimated Primary Completion Date :	September 2012
Estimated Study Completion Date :	September 2012

Arms and Interventions

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Intervention Details:

Drug: High-Dose Chemotherapy with Tandem PBSC Rescue.
Patients on this study will undergo a tandem Peripheral Blood Stem Cell Rescue following high-dose chemotherapy. The first Peripheral Blood Stem Cell Rescue will consist of Etoposide, Carboplatin, Cyclophosphamide, and Mesna. Once the patient recovers, the patient will be evaluated again and will then undergo a second stem cell transplant consisting of the chemotherapy drugs; Melphalan, Cyclophosphamide, and Mesna.
Other Name: Cyclophosphamide call also be referred to as Cytoxan

Outcome Measures

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. 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 ]

Secondary Outcome Measures :

1. 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 ]
2. 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 ]

Eligibility Criteria

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	up to 21 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Morris Kletzel, M.D.	773.880.4000 ext 4564	mkletzel@northwestern.edu
Contact: Meredith Marshall	773-880-3459	MeMarshall@childrensmemorial.org

Locations

United States, Illinois
Children's Memorial Hospital	Recruiting
Chicago, Illinois, United States, 60614

Sponsors and Collaborators

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

More Information

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Morris Kletzel, Children's Memorial Hospital
ClinicalTrials.gov Identifier:	NCT00179816 History of Changes
Other Study ID Numbers:	BMT 0499 Solid
First Posted:	September 16, 2005
Last Update Posted:	October 11, 2010
Last Verified:	October 2010

Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:

wilm's tumor; stem cell transplantation; solid tumor

Additional relevant MeSH terms:

Sarcoma; Neoplasms, Germ Cell and Embryonal; Sarcoma, Ewing; Hodgkin Disease; Hepatoblastoma; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Osteosarcoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Lymphoma; Lymphoproliferative Disorders; Lymphatic Diseases	Immunoproliferative Disorders; Immune System Diseases; Neoplasms, Complex and Mixed; Cyclophosphamide; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Antirheumatic Agents; Antineoplastic Agents, Alkylating; Alkylating Agents; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Myeloablative Agonists