Immunotoxin Therapy in Treating Children With Progressive or Recurrent Glioblastoma Multiforme or Anaplastic Astrocytoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00052624
Recruitment Status : Unknown
Verified April 2004 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : January 27, 2003
Last Update Posted : May 15, 2018
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Immunotoxins can locate tumor cells and kill them without harming normal cells. Immunotoxin therapy may be an effective treatment for glioblastoma multiforme and anaplastic astrocytoma.

PURPOSE: Phase I trial to study the effectiveness of immunotoxin therapy in treating children who have progressive or recurrent glioblastoma multiforme or anaplastic astrocytoma

Condition or disease Intervention/treatment Phase
Brain and Central Nervous System Tumors Biological: transferrin-CRM107 Phase 1

Detailed Description:


  • Determine the maximum tolerated dose of intratumoral transferrin-CRM107 in pediatric patients with progressive or recurrent glioblastoma multiforme or anaplastic astrocytoma.
  • Determine the safety of this drug in these patients.
  • Determine the efficacy of this drug in these patients.
  • Compare the efficacy of this drug in patients with different histological types of tumor, degrees of transferrin receptor expression, and serum antidiphtheria antibody titer levels.

OUTLINE: This is a dose-escalation, open-label, multicenter study. Patients are assigned to 1 of 2 treatment groups by age (5-9 vs 10-18).

All patients undergo stereotactic radiosurgery for tumor biopsy and placement of 2 intratumoral silastic infusion catheters pre-loaded with transferrin-CRM107 (Tf-CRM107).

  • Group 1 (ages 5-9): Patients receive intratumoral Tf-CRM107 over 3-7 days via catheter. Treatment repeats after 6-10 weeks in the absence of unacceptable toxicity. Three cohorts of 3-6 patients receive escalating doses of Tf-CRM107 until the maximum tolerated dose (MTD) is determined.
  • Group 2 (ages 10-18): Patients receive intratumoral Tf-CRM107 as in group 1. Two cohorts of 3-6 patients receive escalating doses of Tf-CRM107 until the MTD is determined.

The MTD in both groups is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed monthly for 6 months and then every 3 months for 6 months.

PROJECTED ACCRUAL: A total of 3-30 patients will be accrued for this study.

Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Multicenter Trial Of Intratumoral/Interstitial Therapy With HN66000, NC66000 (TransMID) In Patients Between 5 and 18 Years Of Age With Progressive Or Recurrent Glioblastoma Multiforme Or Anaplastic Astrocytoma
Study Start Date : July 2002

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed glioblastoma multiforme or anaplastic astrocytoma with the following tumor characteristics:

    • Unifocal
    • Unilateral and supratentorial
    • Diameter no greater than 3.5 cm by contrast-enhanced MRI
  • No more than 1 satellite tumor
  • Recurrent or progressive disease

    • Progressive disease defined as at least 25% increase in tumor volume by serial MRI or CT scans and/or at least 15% increase in the largest cross-sectional area of tumor as defined by the area of contrast agent enhancement
  • Must have received prior conventional treatment comprising both of the following:

    • Surgery (biopsy or debulking)
    • Radiation therapy
  • No evidence of mass effect on CT scan or MRI with more than a 5 mm midline shift and/or nausea, vomiting, reduced level of consciousness, or clinically significant papilledema



  • 5 to 18

Performance status

  • Karnofsky 60-100% OR
  • Lansky Play 50-100%

Life expectancy

  • At least 3 months


  • Platelet count at least 100,000/mm^3
  • Absolute neutrophil count at least 1,000/mm^3


  • Bilirubin no greater than 2.0 mg/dL
  • AST and ALT no greater than 2.5 times the upper limit of normal (ULN)
  • PT or aPTT no greater than 1.5 times ULN


  • Not specified


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception during and for at least 2 months after study
  • No acute viral, bacterial, or fungal infection requiring therapy

    • Topical treatment for oral candidiasis allowed
  • No other concurrent medical condition that would preclude anesthesia


Biologic therapy

  • No prior transferrin-CRM107


  • More than 1 month since prior chemotherapy (6 weeks for nitrosoureas or mitomycin)
  • More than 3 months since prior biodegradable polymer wafers
  • No concurrent chemotherapy

Endocrine therapy

  • Must be on stable dose of steroids for 7 days prior to infusion


  • See Disease Characteristics
  • More than 3 months since prior radiotherapy
  • More than 3 months since prior stereotactic radiosurgery
  • More than 6 weeks since prior craniospinal irradiation
  • No prior brachytherapy
  • No concurrent radiotherapy


  • See Disease Characteristics
  • More than 1 month since prior surgery including tumor surgery or debulking
  • No other concurrent surgery


  • More than 30 days since prior investigational agents
  • No other concurrent investigational therapy
  • No other concurrent anti-cancer drugs

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00052624

United States, California
Children's Hospital of Orange County
Orange, California, United States, 92868-3874
United States, South Carolina
Hollings Cancer Center at Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Xenova Biomedix
Study Chair: Patrick Rossi, MD Xenova Biomedix Identifier: NCT00052624     History of Changes
Other Study ID Numbers: MUSC-10550
CDR0000258574 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: May 15, 2018
Last Verified: April 2004

Keywords provided by National Cancer Institute (NCI):
recurrent childhood cerebral astrocytoma

Additional relevant MeSH terms:
Nervous System Neoplasms
Central Nervous System Neoplasms
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms by Site
Nervous System Diseases