search
Back to results

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

Primary Purpose

Ewing's Sarcoma, Soft Tissue Sarcoma, Hepatoblastoma

Status
Unknown status
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
High-Dose Chemotherapy with Tandem PBSC Rescue.
Sponsored by
Ann & Robert H Lurie Children's Hospital of Chicago
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ewing's Sarcoma focused on measuring wilm's tumor, stem cell transplantation, solid tumor

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept 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.

Sites / Locations

  • Children's Memorial HospitalRecruiting

Outcomes

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.

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.
Evaluate correlation between cell dose and time to engraftment in high-risk pediatric solid tumor patients treated using this approach.

Full Information

First Posted
September 10, 2005
Last Updated
October 7, 2010
Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
search

1. Study Identification

Unique Protocol Identification Number
NCT00179816
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.
Study Type
Interventional

2. Study Status

Record Verification Date
October 2010
Overall Recruitment Status
Unknown status
Study Start Date
April 1999 (undefined)
Primary Completion Date
September 2012 (Anticipated)
Study Completion Date
September 2012 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ewing's Sarcoma, Soft Tissue Sarcoma, Hepatoblastoma, Hodgkin's Disease, Germ Cell Tumor
Keywords
wilm's tumor, stem cell transplantation, solid tumor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
High-Dose Chemotherapy with Tandem PBSC Rescue.
Other Intervention Name(s)
Cyclophosphamide call also be referred to as Cytoxan
Intervention Description
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.
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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
Title
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

10. Eligibility

Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Morris Kletzel, M.D.
Phone
773.880.4000
Ext
4564
Email
mkletzel@northwestern.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Meredith Marshall
Phone
773-880-3459
Email
MeMarshall@childrensmemorial.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Morris Kletzel, M.D.
Organizational Affiliation
Ann & Robert H Lurie Children's Hospital of Chicago
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs