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NYU 05-40 PBMTC ONC-032P:High Dose Temozolomide,Thiotepa and Carboplatin With Autologous Stem Cell Rescue (ASCR) Followed by Continuation Therapy With 13-cis-retinoic Acid in Patients With Recurrent/Refractory Malignant Brain Tumors


Phase 2
6 Months
21 Years
Not Enrolling
Both
Brain Tumors

Thank you

Trial Information

NYU 05-40 PBMTC ONC-032P:High Dose Temozolomide,Thiotepa and Carboplatin With Autologous Stem Cell Rescue (ASCR) Followed by Continuation Therapy With 13-cis-retinoic Acid in Patients With Recurrent/Refractory Malignant Brain Tumors


Researchers have used high doses of combination chemotherapy followed by a stem cell rescue
to treat recurrent brain tumors with moderate success. High dose chemotherapy with stem cell
rescue has resulted in long term survival of about 25% in patients with several different
types of recurrent brain tumors. Stem cells are cells in the bone marrow that produce blood
cells. The stem cells are collected from the blood of the patient before the high dose
chemotherapy. Patients are given high doses of chemotherapy to kill every brain tumor cell,
but in the process the cells of the bone marrow are also killed. The previously collected
stem cells are then infused into the patient to rescue the bone marrow and allow for healthy
blood cells to re-populate and grow in the bone marrow. Initial studies used the drug
etoposide along with carboplatin and thiotepa for the high dose chemotherapy. Patients had
severe side effects, especially severe mouth-sores, thought mainly due to the etoposide, and
some patients died from these side effects.

Recent studies have shown that a new drug, temozolomide, is active against some types of
brain tumors. When it was given as a single drug to children with solid tumors, the side
effects were considered to be tolerable. Temozolomide is given by mouth. In this study,
researchers want to give high dose chemotherapy that includes the drugs temozolomide in
place of etoposide, along with thiotepa and carboplatin. Patients will then be given their
own stem cells back to rescue the bone marrow from the chemotherapy. A preliminary trial
using this new drug combination was performed and has shown that patients tolerate this drug
combination, even at the very high doses that will be used in this protocol.

Another drug that is being used in pediatric cancer treatment is called 13-cis-retinoic
acid. This drug is closely related to vitamin A. It is taken by mouth. Cancer cells are
immature cells that have not "grown up" into adult cells that do work in the body.
13-cis-retinoic acid is thought to act on some types of cancer cells to make them mature
into cells that function in the body. It has also been shown in the laboratory to cause some
brain tumor cells to undergo apoptosis. It has been used in other types of pediatric cancers
and research is just beginning to use it for treatment of recurrent brain tumors. In this
study researchers want to give you 13-cis-retinoic acid for 6 months after you recover from
the high dose chemotherapy with stem cell rescue.


Inclusion Criteria:



1. Patients with recurrent or refractory medulloblastoma/PNET, CNS germ cell tumors,
ependymomas, AT/RT, high grade glioma and other malignant brain tumors. Brainstem
gliomas are eligible if residual disease is < 1.5cc and if the patient is off
decadron.

2. Patients must have recurrent or refractory disease following at least one prior
course of therapy and must have minimal residual disease defined as < 1.5 cm2 of
enhancement. Patients with + CSF cytology, linear or fine nodular leptomeningeal
disease are eligible.

3. Adequate hematologic, renal, liver, and cardiac function as demonstrated by
laboratory values performed within 21 days, inclusive, prior to administration of
temozolomide.

4. Patients must have an adequate number of autologous stem cells available defined as a
minimum of 2 x 106 CD 34+ cells/kg and preferably at least 5 x 106 CD 34+ cells/kg.

Exclusion Criteria:

1. Previous myeloablative therapy

2. Frequent vomiting or medical condition that could interfere with oral medication
intake (e.g., partial bowel obstruction)

3. Previous or concurrent malignancies at other sites with the exception of surgically
cured carcinoma in-situ of the cervix and basal or squamous cell carcinoma of the
skin. Patients with prior malignancies which have not required anti-tumor treatment
within the preceding 24 months are eligible.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

• To assess the event-free survival and overall survival of patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors

Outcome Time Frame:

Day +42 and Day +77

Safety Issue:

Yes

Principal Investigator

Sharon L Gardner, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

New York University School of Medicine

Authority:

United States: Institutional Review Board

Study ID:

NYU 05-40 H12853

NCT ID:

NCT00528437

Start Date:

October 2005

Completion Date:

July 2014

Related Keywords:

  • Brain Tumors
  • Recurrent or refractory medulloblastoma/PNET
  • CNS germ cell tumors
  • Ependymomas
  • AT/RT
  • High grade glioma
  • Other malignant brain tumors
  • Brain Neoplasms

Name

Location
Roswell Park Cancer Institute Buffalo, New York  14263
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Phoenix Children's Hospital Phoenix, Arizona  85016-7710
Nationwide Children's Hospital Columbus, Ohio  43205-2696
Children's Medical Center of Dallas Dallas, Texas  75235
Emory University Atlanta, Georgia  30322
Children's Memorial Hospital Chicago, Illinois  60614
Hawaii Pacific Health Lihue, Hawaii  96766
Riley Hospital for Children Indianapolis, Indiana  46202
Children's Hospital and Clinics of Minnesota Minneapolis, Minnesota  55404
Steven and Alexandra Cohen Children's Medical Center of New York- North Shore LIJ New Hyde Park, New York  11040
NYU Hassenfeld Center New York, New York  10016
Medical Univ. of South Carolina Charleston, South Carolina  29425
Vanderbilt Univ. Nashville, Tennessee  37240
MD Anderson Cancer Center (MDACC) Houston, Texas  77030
Virginia Commonwealth Univ. Richmond, Virginia  23284