Clinical trial NCT00179803: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: 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
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2005-09-16
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: high dose chemotherapy with stem cell rescue thiotepa, carboplatin \& etoposide
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: 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
timeFrame: To end of study
Secondary outcomes:
measure: 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.
timeFrame: To end of study
measure: 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.
timeFrame: To end of study
measure: 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.
timeFrame: To end of study
measure: 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.
timeFrame: To end of study
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Stewart Goldman, MD
role: PRINCIPAL_INVESTIGATOR
References: