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Tandem High Dose Chemotherapy and Autologous Stem Cell Rescue for High Risk Pediatric Brain Tumors

Primary Purpose

Brain Tumors

Status
Unknown status
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
HDCT 1(TTC), HDCT2(MEC)
Sponsored by
Seoul National University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Brain Tumors focused on measuring Brain tumors, Stem cell transplantation, topotecan, thiotepa, carboplatin, melphalan, etoposide

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Seoul National University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

topotecan

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

To evaluate treatment related mortality
To evaluate the incidence and severity of toxicity
To evaluate overall survival rate and relapse rate

Full Information

First Posted
April 20, 2011
Last Updated
November 17, 2013
Sponsor
Seoul National University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT01342237
Brief Title
Tandem High Dose Chemotherapy and Autologous Stem Cell Rescue for High Risk Pediatric Brain Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
November 2013
Overall Recruitment Status
Unknown status
Study Start Date
March 2011 (undefined)
Primary Completion Date
February 2014 (Anticipated)
Study Completion Date
February 2014 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor
Seoul National University Hospital

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
Detailed Description
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Brain Tumors
Keywords
Brain tumors, Stem cell transplantation, topotecan, thiotepa, carboplatin, melphalan, etoposide

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
33 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
topotecan
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HDCT 1(TTC), HDCT2(MEC)
Other Intervention Name(s)
Topotecan, Thiotepa, Carboplatin, Melphalan, Etoposide
Intervention Description
TTC Topotecan (2 mg/m2 once daily i.v. on days -8, -7, -6, -5, -4) Thiotepa (300 mg/m2 once daily i.v on days -8, -7, -6) Carboplatin (500 mg/m2 once daily i.v on days -5, -4, -3) (max. 700 mg/day) MEC Melphalan (140 mg/m2 once daily i.v on day -7, 70 mg/m2 once daily i.v on day -6) Etoposide (200 mg/m2 once daily i.v on days -8, -6) Carboplatin (350 mg/m2 once daily i.v on days -8, -7, -6, -5)
Primary Outcome Measure Information:
Title
To evaluate event free survival rate
Description
To evaluate event free survival rate after high dose chemotherapy and autologous stem cell rescue in pediatric patients with high risk brain tumor
Time Frame
1 month
Secondary Outcome Measure Information:
Title
To evaluate treatment related mortality
Time Frame
1, 3, 6, 12 month
Title
To evaluate the incidence and severity of toxicity
Time Frame
1, 3, 6, 12 month
Title
To evaluate overall survival rate and relapse rate
Time Frame
1, 3, 6, 12 month

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 All high grade or malignant brain tumor, age < 3 years Recurrent embryonal brain tumors, recurrent CNS germ cell tumor Age : no limitation Performance status : ECOG 0-2. 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. Patients must lack any active viral infections or active fungal infection. Patients (or one of parents if patients age < 20) should sign informed. Exclusion Criteria: Patients who do not reach partial response prior to high dose chemotherapy. Pregnant or nursing women. Malignant (except brain tumor) or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy. Psychiatric disorder that would preclude compliance. Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hyoung Jin Kang, MD, PhD
Phone
82 2 2072 3304
Email
kanghj@snu.ac.kr
First Name & Middle Initial & Last Name or Official Title & Degree
Hyery Kim, MD
Phone
82 2 2072 0177
Email
taban@hanmail.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyoung Jin Kang, M.D., Ph.D
Organizational Affiliation
Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
State/Province
Daehangno, Jongno-gu
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hyoung Jin Kang, MD, ph.D
Phone
82 2 2072 3304
Email
kanghj@snu.ac.kr
First Name & Middle Initial & Last Name & Degree
Hyery Kim, MD
Phone
82 2 2072 0177
Email
taban@hanmail.net

12. IPD Sharing Statement

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Tandem High Dose Chemotherapy and Autologous Stem Cell Rescue for High Risk Pediatric Brain Tumors

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