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

Soft Tissue Sarcoma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00179816
• Other study ID #: BMT 0499 Solid
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: September 10, 2005
• Last updated: October 7, 2010
• Start date: April 1999
• Est. completion date: September 2012

Study information

• Verified date: October 2010
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Contact: Morris Kletzel, M.D.
• Phone: 773.880.4000
• Email: mkletzel[@]northwestern.edu
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 21 Years

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ewing's Sarcoma
• Germ Cell Tumor
• Hepatoblastoma
• Hodgkin Disease
• Hodgkin's Disease
• Sarcoma
• Sarcoma, Ewing
• Soft Tissue Sarcoma

Intervention

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.

Locations

Country	Name	City	State
United States	Children's Memorial Hospital	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors.		annually	Yes
Secondary	Evaluate length of remission and long term disease free survival in chemotherapy responsive high-risk pediatric solid tumor patients treated using this approach.		Annually	Yes
Secondary	Evaluate correlation between cell dose and time to engraftment in high-risk pediatric solid tumor patients treated using this approach.		Time to engraftment	Yes