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Stem Cell Transplant for High Risk Central Nervous System (CNS) Tumors

Primary Purpose

Glioblastoma, Astrocytoma, Pineoblastoma

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Stem Cell Transplant
Sponsored by
Ann & Robert H Lurie Children's Hospital of Chicago
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glioblastoma focused on measuring brain tumor, medulloblastoma, germ cell tumor, CNS tumor, glioblastoma, astrocytoma, pineoblastoma, rhabdoid tumor, supratentorial PNET, high grade astrocytoma

Eligibility Criteria

18 Months - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patient's age must be greater than (>) 18 months and less than or equal to (≤) 25 years at the time of diagnosis or recurrence. Neuroradiographic evidence of a recurrent posterior fossa medulloblastoma or recurrent CNS germ cell tumor. The presence of a histologically confirmed high grade astrocytoma, GBM, rhabdoid tumor, supratentorial PNET, or pineoblastoma either at the time of diagnosis or recurrence. Patients must be brought to state of minimum residual disease by surgical reduction and/or chemotherapy and/or radiation therapy or a combination of above prior to myeloablative chemotherapy and tandem stem cell rescue. Documentation of chemotherapy sensitivity is required for enrollment. Chemotherapy-sensitive tumors are defined as those tumors which have had a reduction of 50% after 2-4 cycles of chemotherapy (CTX or platinum). For patients with no evidence of disease post resection, continued complete remission after 2-4 cycles of chemotherapy defines chemosensitivity. Adequate physiologic function, defined as follows: creatinine clearance > 70 ml/minutes/1.73 m2. SGPT < 10 x normal and bilirubin < 10 mg/dl. Adequate complete blood count (CBC): hemoglobin > 10 gm/dl, absolute neutrophil count (ANC) > 1500/ul, and platelets > 100,000/ul. Informed consent. The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies provided by the United States (U.S.) Department of Health and Human Services. Protocol approval. Approval for the use of this institution's Human Rights Committee must be obtained in accordance with the institutional assurance policies of the U. S. Department of Health and Human Services. Patients with high-risk medulloblastoma after initial surgery. To allow non-English speaking patients to participate in this study, bilingual health care services will be provided in the appropriate language. Exclusion Criteria: Patients with brain stem glioma are ineligible.

Sites / Locations

  • Children's Memorial Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

high dose chemotherapy

Arm Description

Outcomes

Primary Outcome Measures

To determine if the use of sequential myeloablative chemotherapy with peripheral blood stem cell rescue will increase the overall survival rate in patients with newly diagnosed high risk CNS tumors

Secondary Outcome Measures

The overall survival and progression free survival in children with recurrent CNS malignancies after obtaining a state of minimum residual disease with submyeloablative chemotherapy, surgery, and/or radiation.
To determine the progression free survival and overall survival using sequential myeloablative chemotherapy as compared to historical controls with single autologous stem cell rescue following myeloablative chemotherapy.
Determine the long term neurocognitive, endocrinologic, cardiopulmonary, and hematologic sequelae of sequential myeloablative chemotherapy and stem cell rescues in patients treated for high risk CNS and recurrent CNS tumors.
Determine the feasibility and utility of the myeloablative preparatory regimen of Carboplatinum, VP-16 and Thiotepa administered in an outpatient setting, and to determine the cost savings obtained via this strategy.

Full Information

First Posted
September 10, 2005
Last Updated
August 3, 2020
Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
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1. Study Identification

Unique Protocol Identification Number
NCT00179803
Brief Title
Stem Cell Transplant for High Risk Central Nervous System (CNS) Tumors
Official Title
Phase II Prospective Study of Sequential Myeloablative Chemotherapy With Stem Cell Rescue for the Treatment of Selected High Risk CNS Tumors and Recurrent CNS Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Completed
Study Start Date
March 1998 (undefined)
Primary Completion Date
January 2008 (Actual)
Study Completion Date
September 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The primary goal of this study is to determine if a stem cell transplant in patients with newly diagnosed high risk CNS tumors (glioblastoma multiforme [GBM], high grade astrocytoma, pineoblastoma, rhabdoid tumor, supratentorial primitive neuroectodermal tumor [PNET]) increases overall survival.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glioblastoma, Astrocytoma, Pineoblastoma, Rhabdoid Tumor, Supratentorial Neoplasms
Keywords
brain tumor, medulloblastoma, germ cell tumor, CNS tumor, glioblastoma, astrocytoma, pineoblastoma, rhabdoid tumor, supratentorial PNET, high grade astrocytoma

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
high dose chemotherapy with stem cell rescue thiotepa, carboplatin & etoposide
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
high dose chemotherapy
Arm Type
Experimental
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplant
Intervention Description
Group A: recurrent medulloblastoma, recurrent germ cell tumor Cytoxan treatment Stem cell autologous harvest Group B: GBM, high grade astrocytoma, rhabdoid tumors, pineoblastoma, or supratentorial PNET Carboplatin and Etoposide treatment Autologous stem cell harvest The preparatory regimen used for Stem Cell Rescue #1 will be Carboplatinum, VP-16 and Thiotepa. If the patient has recuperated his ANC to >1,000 within 50 days after Stem Cell Rescue #1, (sustained without G-CSF support) a neuroradiographic evaluation will be performed. If there is lack of progression, the patient will then proceed to Stem Cell Rescue # 2 with Cyclophosphamide and Melphalan, followed by stem cell rescue.
Primary Outcome Measure Information:
Title
To determine if the use of sequential myeloablative chemotherapy with peripheral blood stem cell rescue will increase the overall survival rate in patients with newly diagnosed high risk CNS tumors
Time Frame
To end of study
Secondary Outcome Measure Information:
Title
The overall survival and progression free survival in children with recurrent CNS malignancies after obtaining a state of minimum residual disease with submyeloablative chemotherapy, surgery, and/or radiation.
Time Frame
To end of study
Title
To determine the progression free survival and overall survival using sequential myeloablative chemotherapy as compared to historical controls with single autologous stem cell rescue following myeloablative chemotherapy.
Time Frame
To end of study
Title
Determine the long term neurocognitive, endocrinologic, cardiopulmonary, and hematologic sequelae of sequential myeloablative chemotherapy and stem cell rescues in patients treated for high risk CNS and recurrent CNS tumors.
Time Frame
To end of study
Title
Determine the feasibility and utility of the myeloablative preparatory regimen of Carboplatinum, VP-16 and Thiotepa administered in an outpatient setting, and to determine the cost savings obtained via this strategy.
Time Frame
To end of study

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Months
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patient's age must be greater than (>) 18 months and less than or equal to (≤) 25 years at the time of diagnosis or recurrence. Neuroradiographic evidence of a recurrent posterior fossa medulloblastoma or recurrent CNS germ cell tumor. The presence of a histologically confirmed high grade astrocytoma, GBM, rhabdoid tumor, supratentorial PNET, or pineoblastoma either at the time of diagnosis or recurrence. Patients must be brought to state of minimum residual disease by surgical reduction and/or chemotherapy and/or radiation therapy or a combination of above prior to myeloablative chemotherapy and tandem stem cell rescue. Documentation of chemotherapy sensitivity is required for enrollment. Chemotherapy-sensitive tumors are defined as those tumors which have had a reduction of 50% after 2-4 cycles of chemotherapy (CTX or platinum). For patients with no evidence of disease post resection, continued complete remission after 2-4 cycles of chemotherapy defines chemosensitivity. Adequate physiologic function, defined as follows: creatinine clearance > 70 ml/minutes/1.73 m2. SGPT < 10 x normal and bilirubin < 10 mg/dl. Adequate complete blood count (CBC): hemoglobin > 10 gm/dl, absolute neutrophil count (ANC) > 1500/ul, and platelets > 100,000/ul. Informed consent. The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies provided by the United States (U.S.) Department of Health and Human Services. Protocol approval. Approval for the use of this institution's Human Rights Committee must be obtained in accordance with the institutional assurance policies of the U. S. Department of Health and Human Services. Patients with high-risk medulloblastoma after initial surgery. To allow non-English speaking patients to participate in this study, bilingual health care services will be provided in the appropriate language. Exclusion Criteria: Patients with brain stem glioma are ineligible.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stewart Goldman, MD
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

12. IPD Sharing Statement

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Stem Cell Transplant for High Risk Central Nervous System (CNS) Tumors

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