Brief Title
Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors
Official Title
High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.
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.
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 Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors.
Secondary Outcome
Evaluate length of remission and long term disease free survival in chemotherapy responsive high-risk pediatric solid tumor patients treated using this approach.
Condition
Ewing's Sarcoma
Intervention
High-Dose Chemotherapy with Tandem PBSC Rescue.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
12
Start Date
April 1999
Completion Date
September 2012
Primary Completion Date
September 2012
Eligibility 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.
Gender
All
Ages
N/A - 21 Years
Accepts Healthy Volunteers
No
Contacts
Morris Kletzel, M.D., 773.880.4000, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00179816
Organization ID
BMT 0499 Solid
Study Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Study Sponsor
Morris Kletzel, M.D., Principal Investigator, Ann & Robert H Lurie Children's Hospital of Chicago
Verification Date
October 2010