Chemotherapy and Donor Stem Transplant for the Treatment of Patients With High Grade Brain Cancer

  • End date
    May 9, 2025
  • participants needed
  • sponsor
    M.D. Anderson Cancer Center
Updated on 11 August 2022
Accepts healthy volunteers


This phase I trial investigates the side effects and effectiveness of chemotherapy followed by a donor (allogeneic) stem cell transplant when given to patients with high grade brain cancer. Chemotherapy drugs, such as fludarabine, thiotepa, etoposide, melphalan, and rabbit anti-thymocyte globulin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells.



I. To assess tolerability of allogenic hematopoietic cell transplantation (HCT) among patients with chemo-responsive high-grade central nervous system (CNS) malignancies as defined by transplant-related mortality (TRM) at day 30 as well as rate of grade III organ toxicity or higher (Bearman Regimen-Related Toxicities Scale) attributable to conditioning occurring within 30 days.


I. Median time to platelet and neutrophil engraftment. II. Incidence of acute graft-versus-host disease (aGVHD) by day 100. III. Incidence of chronic GVHD at day 100 and one year. IV. Rate of grade II organ toxicity through day 100. V. Rate of graft failure (primary and secondary) through day 100. VI. Rate of infectious complications through day 100. VII. Progression free survival at day 180. VIII. Cumulative incidence of relapse, overall survival, and progression-free survival at 100 days and 1 year.


Patients receive thiotepa intravenously (IV) over 2-4 hours and etoposide IV over 60 minutes on days -8 to -6, melphalan IV over 20 minutes on days -5 and -4, and fludarabine phosphate IV over 1 hour on days -5 to -3. Patients receiving umbilical cord transplant only also receive lapine T-lymphocyte immune globulin IV over 4-12 hours on days -4 and -3. Patients then undergo HCT on day 0. Patients also receive tacrolimus IV or cyclosporine IV beginning on day -2 to and mycophenolate mofetil orally (PO) every 8 hours or IV from days 0-40 and tapered to day 90.

After completion of study treatment, patients are followed up at 100, 180, 270 and 360 days.

Condition Anaplastic Ependymoma, Atypical Teratoid/Rhabdoid Tumor, Central Nervous System Germ Cell Tumor, Choroid Plexus Carcinoma, Intracranial Myeloid Sarcoma, Malignant Brain Neoplasm, Malignant Glioma, Medulloblastoma, Primitive Neuroectodermal Tumor, Recurrent Anaplastic Ependymoma, Recurrent Atypical Teratoid/Rhabdoid Tumor, Recurrent Malignant Brain Neoplasm, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Primitive Neuroectodermal Tumor
Treatment fludarabine phosphate, mycophenolate mofetil, etoposide, melphalan, Tacrolimus, Hematopoietic Cell Transplantation, thiotepa, Lapine T-Lymphocyte Immune Globulin
Clinical Study IdentifierNCT04521946
SponsorM.D. Anderson Cancer Center
Last Modified on11 August 2022


Yes No Not Sure

Inclusion Criteria

Pathological criteria for any high grade primary or recurrent malignant brain tumor - medulloblastoma (patients who are ineligible for tandem autologous transplants or who are at least 3 months post autologous HCT), primitive neuroectodermal tumor (PNET), atypical teratoid rhabdoid tumor (ATRT), malignant glioma, CNS germ cell tumor, intracranial sarcomas, choroid plexus carcinoma, anaplastic ependymoma. High grade tumors defined as those that are grade III or higher based on World Health Organization (WHO) classification grading system or for medulloblastoma: group 3 and 4 molecular subtypes
Patients have to be in at least, a chemo-responsive disease status
Available suitable HCT donor
Creatinine clearance or glomerular filtration rate (GFR) >= 50 ml/min/1.73m^2, and not requiring dialysis
Diffusion capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) >= 50% predicted. If unable to perform pulmonary function tests, then O2 saturation >= 92% in room air
Bilirubin =< 3x upper limit of normal (ULN) (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome)
Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 5x for age
DONOR: HCT will be done using stem cell sources in the following order of preference (and fulfilling minimal cell dose requirements per institutional standards)
Matched related donor bone marrow (10 of 10 human leukocyte antigen [HLA] alleles [HLA-A, B, C, DR, and DQ]). Matched related donor peripheral blood stem cell (PBSC) is allowed only if collection of bone marrow (BM) is not available or refused by guardian/donor
Matched allogeneic umbilical cord blood: related
High-resolution matching at A,B, DRB1 (minimum 4/6)
Killer-cell immunoglobulin-like receptor (KIR) major histocompatibility complex (MHC) class 1 preferential mismatch (minimum 4/6)
Matched allogeneic umbilical cord blood: unrelated
High-resolution matching at A,B, DRB1(minimum 4/6)
KIR MHC class 1 preferential mismatch (minimum 4/6)

Exclusion Criteria

Lack of histocompatible suitable graft source
End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning regimen
Renal failure requiring dialysis
Congenital heart disease resulting in congestive heart failure
Ventilatory failure: requires invasive mechanical ventilation
Human immunodeficiency virus (HIV) infection
Uncontrolled bacterial, viral, or fungal infections
A female of reproductive potential who is pregnant, planning to become pregnant during the study, or is nursing a child
Any patient who does not fulfill inclusion criteria listed above
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