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

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago (Other)

Overall Status

Unknown status

CT.gov ID

NCT00179816

Collaborator

(none)

Enrollment

Location

161.1

Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

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

Study Type:

Interventional

Anticipated 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 :

Apr 1, 1999

Anticipated Primary Completion Date :

Sep 1, 2012

Anticipated Study Completion Date :

Sep 1, 2012

Outcome Measures

Primary Outcome Measures

Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors. [annually]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 21 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.