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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2011 by Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier:
NCT00179803
First received: September 10, 2005
Last updated: March 10, 2011
Last verified: March 2011
  Purpose

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 Intervention Phase
Glioblastoma
Astrocytoma
Pineoblastoma
Rhabdoid Tumor
Supratentorial Neoplasms
Procedure: Stem Cell Transplant
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:

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 [ Time Frame: To end of study ] [ Designated as safety issue: Yes ]

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. [ Time Frame: To end of study ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: March 1998
Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    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.

  Eligibility

Ages Eligible for Study:   18 Months to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00179803

Locations
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Stewart Goldman, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

No publications provided

Responsible Party: Stewart Goldman, MD, Children's Memorial Hospital
ClinicalTrials.gov Identifier: NCT00179803     History of Changes
Other Study ID Numbers: BMT 0398
Study First Received: September 10, 2005
Last Updated: March 10, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Ann & Robert H Lurie Children's Hospital of Chicago:
brain tumor
medulloblastoma
germ cell tumor
CNS tumor
glioblastoma
astrocytoma
pineoblastoma
rhabdoid tumor
supratentorial PNET
high grade astrocytoma

Additional relevant MeSH terms:
Astrocytoma
Glioblastoma
Neoplasms
Rhabdoid Tumor
Supratentorial Neoplasms
Brain Diseases
Brain Neoplasms
Central Nervous System Diseases
Central Nervous System Neoplasms
Glioma
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Complex and Mixed
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Nervous System Diseases
Nervous System Neoplasms
Neuroectodermal Tumors

ClinicalTrials.gov processed this record on November 24, 2014