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

NCT ID: NCT00179816

Last Updated: 2010-10-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

1999-04-30

Study Completion Date

2012-09-30

Brief Summary

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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

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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.

Conditions

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

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Cyclophosphamide call also be referred to as Cytoxan

Eligibility Criteria

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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.
Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role lead

Responsible Party

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Children's Memorial Hospital

Principal Investigators

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Morris Kletzel, M.D.

Role: PRINCIPAL_INVESTIGATOR

Ann & Robert H Lurie Children's Hospital of Chicago

Locations

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Children's Memorial Hospital

Chicago, Illinois, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Morris Kletzel, M.D.

Role: CONTACT

773.880.4000 ext. 4564

Meredith Marshall

Role: CONTACT

773-880-3459

Other Identifiers

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BMT 0499 Solid

Identifier Type: -

Identifier Source: org_study_id