Know Cancer

forgot password

Phase 2
Open (Enrolling)
Brain Tumors

Thank you

Trial Information

High risk/recurrent central nervous system (CNS) tumors have a poor prognosis so that tandem
high dose chemotherapy (HDCT) with hematopoietic progenitor stem cell rescues has been
chosen as potentially curative therapy. Many institutions have used carboplatin, thiotepa,
etoposide (CTE) for conditioning regimen of 1st HDCT and cyclophosphamide, melphalan (CM)
for conditioning regimen of 2nd HDCT. Our institution applied this regimen to the 38
pediatric patients with high risk brain tumor since 1996. Although the 3 year overall
survival rate and event free survival rate were improved to 69% and 47.9%, respectively, the
results showed relatively high treatment related mortality (TRM) rate of 21%. Toxicity of
this tandem regimen was also reported as being high up to 32% in other researches as well so
that this regimen is considered not feasible due to toxicity. In this study, the
investigators plan to improve event free survival rate and reduce treatment related
toxicities of pediatric patients with high risk/recurrent CNS tumors by administrating
tandem high dose chemotherapy and autologous stem cell rescue with topotecan, thiotepa,
carboplatin (TTC) for 1st HDCT and melphalan, etoposide, carboplatin (MEC) for 2nd HDCT.

Inclusion Criteria:

1. High risk pediatric brain tumors Newly diagnosed medulloblastoma, CNS PNET, ATRT,
Choroid plexus carcinoma, pineoblastoma with residual tumor over 1.5cm2 after
operation or with leptomeningeal seeding at diagnosis

2. All high grade or malignant brain tumor, age < 3 years

3. Recurrent embryonal brain tumors, recurrent CNS germ cell tumor

4. Age : no limitation

5. Performance status : ECOG 0-2.

6. Patients must be free of significant functional deficits in major organs, but the
following eligibility criteria may be modified in individual cases.

Heart: a shortening fraction ≥ 28%. Liver: total bilirubin < 2 ⅹ upper limit of
normal; ALT < 3 ⅹ upper limit of normal. Kidney: creatinine < 2 ⅹ normal or a
creatinine clearance (GFR) > 60 ml/min/1.73m2.

7. Patients must lack any active viral infections or active fungal infection.

8. Patients (or one of parents if patients age < 20) should sign informed.

Exclusion Criteria:

1. Patients who do not reach partial response prior to high dose chemotherapy.

2. Pregnant or nursing women.

3. Malignant (except brain tumor) or nonmalignant illness that is uncontrolled or whose
control may be jeopardized by complications of study therapy.

4. Psychiatric disorder that would preclude compliance.

5. Patients who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study.

Type of Study:


Study Design:

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

Outcome Measure:

To evaluate event free survival rate

Outcome Description:

To evaluate event free survival rate after high dose chemotherapy and autologous stem cell rescue in pediatric patients with high risk brain tumor

Outcome Time Frame:

1 month

Safety Issue:


Principal Investigator

Hyoung Jin Kang, M.D., Ph.D

Investigator Role:

Principal Investigator

Investigator Affiliation:

Seoul National University Hospital


South Korea: Korea Food and Drug Administration (KFDA)

Study ID:




Start Date:

March 2011

Completion Date:

February 2014

Related Keywords:

  • Brain Tumors
  • Brain tumors
  • Stem cell transplantation
  • topotecan
  • thiotepa
  • carboplatin
  • melphalan
  • etoposide
  • Brain Neoplasms