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

This study is currently recruiting participants.
Verified February 2013 by Seoul National University Hospital
Sponsor:
Information provided by:
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01342237
First received: April 20, 2011
Last updated: February 21, 2013
Last verified: February 2013
History of Changes
  Purpose

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.


Condition Intervention Phase
Brain Tumors
 Drug: HDCT 1(TTC), HDCT2(MEC)
 Phase 2

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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Seoul National University Hospital:

Primary Outcome Measures:
  • To evaluate event free survival rate [ Time Frame: 1 month ] [ Designated as safety issue: No ]
    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 [ Time Frame: 1, 3, 6, 12 month ] [ Designated as safety issue: No ]
  • To evaluate the incidence and severity of toxicity [ Time Frame: 1, 3, 6, 12 month ] [ Designated as safety issue: No ]
  • To evaluate overall survival rate and relapse rate [ Time Frame: 1, 3, 6, 12 month ] [ Designated as safety issue: No ]

Estimated Enrollment: 33
Study Start Date: March 2011
Estimated Study Completion Date: February 2014
Estimated Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: topotecan Drug: HDCT 1(TTC), HDCT2(MEC)
  1. 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)
  2. 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)
Other Names:
  • Topotecan
  • Thiotepa
  • Carboplatin
  • Melphalan
  • Etoposide

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01342237

Contacts
Contact: Hyoung Jin Kang, MD, PhD 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Hyery Kim, MD 82 2 2072 0177 taban@hanmail.net

Locations
Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Daehangno, Jongno-gu, Korea, Republic of
Contact: Hyoung Jin Kang, MD, ph.D     82 2 2072 3304     kanghj@snu.ac.kr    
Contact: Hyery Kim, MD     82 2 2072 0177     taban@hanmail.net    
Sponsors and Collaborators
Seoul National University Hospital
Investigators
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
  More Information

No publications provided

Responsible Party: Hyo Seop Ahn, Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT01342237     History of Changes
Other Study ID Numbers: SNUCH-SCT-1102
Study First Received: April 20, 2011
Last Updated: February 21, 2013
Health Authority: South Korea: Korea Food and Drug Administration (KFDA)
South Korea: Institutional Review Board

Keywords provided by Seoul National University Hospital:
Brain tumors
Stem cell transplantation
topotecan
thiotepa
 carboplatin
melphalan
etoposide

Additional relevant MeSH terms:
Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Etoposide
Etoposide phosphate
Melphalan
Thiotepa
Carboplatin
Topotecan
 Antineoplastic Agents, Phytogenic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors

ClinicalTrials.gov processed this record on May 16, 2013