Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors
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ClinicalTrials.gov Identifier: NCT00179816 |
Recruitment Status
: Unknown
Verified October 2010 by Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was: Recruiting
First Posted
: September 16, 2005
Last Update Posted
: October 11, 2010
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Condition or disease | Intervention/treatment | Phase |
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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 |
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 (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 |

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Drug: High-Dose Chemotherapy with Tandem PBSC Rescue.
- 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 ]
- 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 ]
- 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 ]

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Ages Eligible for Study: | up to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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Malignant Diseases:
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Ewing's sarcoma/PNET:
- CR1 - Metastatic disease at diagnosis, tumor volume > 100 ml, pelvic bone primary
- CR2 - Locally recurrent disease
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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)
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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
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Hodgkin's Disease:
- VGPR1 - Progression on primary therapy/Refractory disease
- CR2/VGPR2
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Germ Cell Tumor:
- CR2/VGPR2 - recurrent disease
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Wilms Tumor:
- CR2/VGPR2 - recurrent disease
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- 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.
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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.
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Physiologic status:
- No active infections
- Adequate performance status as measured by Karnofsky (> 70%) or Lansky scale (> 60%) as appropriate for age.
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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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00179816
Contact: Morris Kletzel, M.D. | 773.880.4000 ext 4564 | mkletzel@northwestern.edu | |
Contact: Meredith Marshall | 773-880-3459 | MeMarshall@childrensmemorial.org |
United States, Illinois | |
Children's Memorial Hospital | Recruiting |
Chicago, Illinois, United States, 60614 |
Principal Investigator: | Morris Kletzel, M.D. | Ann & Robert H Lurie Children's Hospital of Chicago |
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 Key Record Dates |
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 |