Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00179803
Recruitment Status : Completed
First Posted : September 16, 2005
Last Update Posted : August 5, 2020
Information provided by (Responsible Party):
Stewart Goldman, Ann & Robert H Lurie Children's Hospital of Chicago

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.

Condition or disease Intervention/treatment Phase
Glioblastoma Astrocytoma Pineoblastoma Rhabdoid Tumor Supratentorial Neoplasms Procedure: Stem Cell Transplant Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: high dose chemotherapy with stem cell rescue thiotepa, carboplatin & etoposide
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : March 1998
Actual Primary Completion Date : January 2008
Actual Study Completion Date : September 2009

Arm Intervention/treatment
Experimental: high dose chemotherapy Procedure: Stem Cell Transplant

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 Measures :
  1. 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 Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Months to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT00179803

Layout table for location information
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Layout table for investigator information
Principal Investigator: Stewart Goldman, MD Ann & Robert H Lurie Children's Hospital of Chicago
Layout table for additonal information
Responsible Party: Stewart Goldman, Attending Physician, Ann & Robert H Lurie Children's Hospital of Chicago Identifier: NCT00179803    
Other Study ID Numbers: BMT 0398
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: August 5, 2020
Last Verified: August 2020
Keywords provided by Stewart Goldman, Ann & Robert H Lurie Children's Hospital of Chicago:
brain tumor
germ cell tumor
CNS tumor
rhabdoid tumor
supratentorial PNET
high grade astrocytoma
Additional relevant MeSH terms:
Layout table for MeSH terms
Rhabdoid Tumor
Supratentorial Neoplasms
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Complex and Mixed
Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases