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CompletedNCT00003211Updated Nov 7, 2012

Chemotherapy, Radiation Therapy, and Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Medulloblastoma or Supratentorial Primitive Neuroectodermal Tumor

A Phase 2 interventional study of filgrastim and amifostine trihydrate in Brain and Central Nervous System Tumors, sponsored by St. Jude Children's Research Hospital. Completed at 4 sites in 2 countries. Open to participants aged 3 Years to 20 Years. Per ClinicalTrials.gov, last updated 2012-11-07.

Sponsored by St. Jude Children's Research Hospital · Phase 2, Interventional, and Supportive care

Phase
Phase 2
Study type
Interventional
Enrollment
94
Allocation
Non-randomized
Ages
3 Years to 20 Years
Sex
All
01

Study summary

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy and radiation therapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy or radiation therapy and kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of chemotherapy with topotecan, cyclophosphamide, cisplatin, and vincristine plus radiation therapy and peripheral stem cell transplantation in treating children with newly diagnosed medulloblastoma or supratentorial primitive neuroectodermal tumor.

Read the detailed description

OBJECTIVES:

  • Estimate the response rate to topotecan in children with newly diagnosed medulloblastoma or supratentorial primitive neuroectodermal tumors who have measurable residual disease after surgery. (Topotecan window closed to accrual 9/10/2001)
  • Determine the feasibility of four courses of high-dose chemotherapy (vincristine, cisplatin, and cyclophosphamide) with peripheral blood stem cell support after craniospinal irradiation (CSI) in these patients.
  • Estimate the 5-year overall survival and progression-free survival in patients treated with risk-adapted CSI and high-dose chemotherapy.
  • Compare changes in intellectual functioning in patients treated with reduced-dose vs standard-dose CSI.
  • Estimate the incidence of ototoxicity associated with risk-adapted CSI and posterior fossa boost(s) given by 3-D conformal radiotherapy technique combined with amifostine and cisplatin.

OUTLINE: This is a multicenter study. Patients are assigned to 1 of 2 treatment groups based on risk status.

  • Group 1 (average-risk): Patients receive filgrastim (G-CSF) subcutaneously (SC) or IV daily until peripheral blood stem cells (PBSC) are harvested. PBSC are harvested when blood counts recover. Patients then receive craniospinal irradiation (CSI) 5 days a week for 6 weeks. Beginning 6 weeks after completion of CSI, patients receive high-dose chemotherapy comprising vincristine IV followed by cisplatin IV over 6 hours on day -4 and cyclophosphamide IV over 1 hour on days -3 and -2. Patients receive amifostine IV over 1 minute a maximum of 5 minutes prior to cisplatin infusion and then 3 hours into cisplatin infusion. PBSC are reinfused on day 0. Patients receive G-CSF SC beginning on day 1 and continuing for a minimum of 7 days or until blood counts recover. Vincristine IV is administered on day 6. G-CSF is stopped 48 hours prior to beginning subsequent courses of chemotherapy. High-dose chemotherapy repeats every 4 weeks for 4 courses.
  • Group 2 (high-risk): Patients receive topotecan IV over 4 hours on days 1-5 and G-CSF SC or IV beginning 24 hours after completion of the first course of topotecan and continuing until PBSC are harvested. Treatment repeats every 3 weeks for 2 courses. If an adequate number of PBSC are not harvested, the patient undergoes a second harvest of PBSC after the second course of topotecan. Patients then receive CSI, high-dose chemotherapy, amifostine, and PBSC support as in group 1. (Topotecan window closed to accrual 9/10/2001) Patients undergo neuropsychological testing prior to radiotherapy and chemotherapy and then at 1, 2, and 5 years.

Patients are followed at 1, 2, 4, 6, 9, 12, 15, 18, and 24 months and then every 6 months for 3 years.

PROJECTED ACCRUAL: A total of 12-36 patients will be accrued for this study within 5 years.

02

Conditions studied

  • Brain and Central Nervous System Tumors

Keywords

  • untreated childhood supratentorial primitive neuroectodermal tumor
  • untreated childhood medulloblastoma
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In context

Medulloblastoma

239 studies on the registry are indexed under Medulloblastoma; 52 are open to participants now.

This study's enrollment of 94 is above the median of 35 across 199 interventional studies indexed under Medulloblastoma.

Browse Medulloblastoma studies →

Lead sponsor

St. Jude Children's Research Hospital is the lead sponsor of 434 studies on the registry; 99 are open to participants now.

Of its 60 completed or terminated interventional studies of FDA-regulated products, 35 (58%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 20 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

DISEASE CHARACTERISTICS:

  • Histologically proven medulloblastoma or supratentorial primitive neuroectodermal tumor

  • Average-risk group:

    • Localized tumor with no overt evidence of invasion beyond the posterior fossa
    • Less than 1.5 cm2 residual tumor/imaging abnormality
    • No CNS or extraneural metastasis (confirmed by bone scan)
    • Brain stem invasion allowed if above criteria met

  • High-risk group:

    • Metastatic disease within the neuraxis (subarachnoid dissemination) OR greater than 1.5 cm\^2 residual disease at the primary site after surgery
  • No bone involvement by bone scan
  • Must begin study within 28 days of definitive surgery

PATIENT CHARACTERISTICS:

Age

  • 3 to 20 at diagnosis

Performance status

  • ECOG 0-3 (except patients with posterior fossa syndrome)

Life expectancy

  • Not specified

Hematopoietic

  • WBC greater than 3,000/mm\^3
  • Absolute neutrophil count greater than 1,500/mm\^3
  • Platelet count greater than 100,000/mm\^3
  • Hemoglobin greater than 10 g/dL

Hepatic

  • Bilirubin less than 1.5 mg/dL
  • SGPT less than 1.5 times normal

Renal

  • Creatinine less than 1.2 mg/dL OR
  • Creatinine clearance greater than 70 mL/min

Other

  • Not pregnant or nursing
  • Negative pregnancy test
  • HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • No prior chemotherapy

Endocrine therapy

  • Prior corticosteroids allowed

Radiotherapy

  • No prior radiotherapy

Surgery

  • See Disease Characteristics
05

Study design

Phase
Phase 2
Primary purpose
Supportive care
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
94 participants (actual)

Study arms

  • Experimental
    Average-risk

    Participants meeting the eligibility requirements for assignment to the average-risk arm. Interventions: filgrastim, amifostine trihydrate, cisplatin, cyclophosphamide, vincristine sulfate, peripheral blood stem cell transplantation, radiation therapy.

    Biological: filgrastim · Drug: amifostine trihydrate · Drug: cisplatin · Drug: cyclophosphamide · Drug: vincristine sulfate · Procedure: peripheral blood stem cell transplantation · Radiation: radiation therapy

  • Experimental
    High-risk

    Participants meeting the eligibility requirements for assignment to the high-risk arm. Interventions: filgrastim, amifostine trihydrate, cisplatin, cyclophosphamide, vincristine sulfate, peripheral blood stem cell transplantation, radiation therapy.

    Biological: filgrastim · Drug: amifostine trihydrate · Drug: cisplatin · Drug: cyclophosphamide · Drug: vincristine sulfate · Procedure: peripheral blood stem cell transplantation · Radiation: radiation therapy

Interventions

  • Biologicalfilgrastim
  • Drugamifostine trihydrate
  • Drugcisplatin
  • Drugcyclophosphamide
  • Drugvincristine sulfate
  • Procedureperipheral blood stem cell transplantation

    Also known as: PBSCT

  • Radiationradiation therapy
06

Study locations

4 sites
  • St. Jude Children's Research Hospital
    Memphis, Tennessee 38105, United States
  • Texas Children's Cancer Center
    Houston, Texas 77030-2399, United States
  • Children's Hospital at Westmead
    Westmead, New South Wales 2145, Australia
  • Royal Children's Hospital
    Parkville, Victoria 3052, Australia
07

References and documents

Publications

  • Laughton SJ, Merchant TE, Sklar CA, Kun LE, Fouladi M, Broniscer A, Morris EB, Sanders RP, Krasin MJ, Shelso J, Xiong Z, Wallace D, Gajjar A. Endocrine outcomes for children with embryonal brain tumors after risk-adapted craniospinal and conformal primary-site irradiation and high-dose chemotherapy with stem-cell rescue on the SJMB-96 trial. J Clin Oncol. 2008 Mar 1;26(7):1112-8. doi: 10.1200/JCO.2008.13.5293. PubMed 18309946 ↗
  • Gajjar A, Chintagumpala M, Ashley D, Kellie S, Kun LE, Merchant TE, Woo S, Wheeler G, Ahern V, Krasin MJ, Fouladi M, Broniscer A, Krance R, Hale GA, Stewart CF, Dauser R, Sanford RA, Fuller C, Lau C, Boyett JM, Wallace D, Gilbertson RJ. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): long-term results from a prospective, multicentre trial. Lancet Oncol. 2006 Oct;7(10):813-20. doi: 10.1016/S1470-2045(06)70867-1. Erratum In: Lancet Oncol. 2006 Oct;7(10):797. PubMed 17012043 ↗
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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 7, 2012, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00003211
Lead sponsor
St. Jude Children's Research Hospital
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jul 9, 2003
Start date
Oct 1996
Primary completion
Jun 2007
Completion
Jun 2007
Last update
Nov 7, 2012

Study contacts

Amar Gajjar, MD
study chair · St. Jude Children's Research Hospital

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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