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Mafosfamide in Treating Patients With Progressive or Refractory Meningeal Tumors

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: NCT00031928
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : April 30, 2015
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

PURPOSE: Phase I trial to determine the effectiveness of mafosfamide in treating patients who have progressive or refractory meningeal tumors.

Condition or disease Intervention/treatment Phase
Brain and Central Nervous System Tumors Drug: mafosfamide Phase 1

Detailed Description:


  • Determine the qualitative and quantitative toxicity of mafosfamide in patients with progressive or refractory meningeal malignancy.
  • Determine the maximum tolerated dose of this drug in these patients.
  • Determine the cerebrospinal fluid pharmacokinetics of this drug in these patients.

OUTLINE: This is a dose-escalation, multicenter study.

Patients receive intrathecal mafosfamide over 20 minutes twice weekly for 6 weeks (induction therapy). Patients then receive intrathecal mafosfamide once weekly for 4 weeks (consolidation therapy), twice a month for 4 months, and then monthly thereafter (maintenance therapy) in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of mafosfamide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

PROJECTED ACCRUAL: A total of 3000 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3000 participants
Primary Purpose: Treatment
Official Title: Phase I Study of Intrathecal Mafosfamide
Study Start Date : January 2002

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of leukemia or lymphoma with meningeal involvement defined as cerebrospinal fluid cell count at least 5/mm^3 AND evidence of blast cells on cytospin preparation or by cytology OR
  • Diagnosis of other solid tumor with meningeal involvement defined as presence of tumor cells on cytospin preparation or cytology OR presence of measurable meningeal disease on CT or MRI scan
  • Meningeal malignancy must be progressive or refractory to conventional therapy

    • Meningeal malignancies secondary to an underlying solid tumor are allowed at initial diagnosis provided there is no conventional therapy
  • No concurrent bone marrow relapse in leukemia or lymphoma patients
  • No clinical evidence of obstructive hydrocephalus or compartmentalization of the cerebrospinal fluid flow as documented by a radioisotope indium In 111 or technetium Te 99-DTPA flow study

    • Patients demonstrating restored flow after focal radiotherapy are allowed



  • Over 3

Performance status:

  • ECOG 0-2

Life expectancy:

  • At least 8 weeks


  • Not specified


  • No clinically significant liver function abnormalities


  • No clinically significant renal function abnormalities


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception during and for 6 months after study
  • No clinically significant metabolic parameter abnormalities (e.g., electrolytes, calcium, and phosphorus)
  • No significant systemic illness (e.g., infection)


Biologic therapy:

  • Recovered from prior immunotherapy


  • At least 1 week since prior intrathecal chemotherapy (2 weeks for cytarabine (liposomal)) and recovered
  • Concurrent systemic chemotherapy to control systemic or bulk CNS disease allowed with the following exceptions:

    • No phase I agent
    • No agent that significantly penetrates the CNS (e.g., high-dose systemic methotrexate (more than 1 g/m^2), high-dose cytarabine (more than 2 g/m^2), IV mercaptopurine, fluorouracil, topotecan, or thiotepa)
    • No agent known to have serious unpredictable CNS side effects

Endocrine therapy:

  • Not specified


  • See Disease Characteristics
  • Recovered from prior radiotherapy
  • At least 8 weeks since prior craniospinal irradiation
  • Local radiotherapy for symptomatic or bulky CNS disease must be given prior to induction therapy
  • No concurrent whole brain or craniospinal irradiation

    • Concurrent partial brain (e.g., base of brain) or limited-field spinal radiotherapy for asymptomatic bulky (radiographically visible) CNS disease allowed
  • Total CNS radiotherapy dose must not exceed accepted safe tissue tolerances


  • Not specified


  • At least 1 week since any prior CNS therapy
  • At least 7 days since prior intrathecal investigational agent
  • At least 14 days since prior systemic investigational agent
  • No other concurrent intrathecal or systemic investigational agent
  • No other concurrent intrathecal or systemic therapy to treat meningeal malignancy
  • No other concurrent intrathecal therapy or agent that significantly penetrates the blood-brain barrier
  • No concurrent agent known to have serious unpredictable CNS side effects

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): NCT00031928

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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027-0700
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Maryland
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, United States, 20892-1182
United States, Michigan
Josephine Ford Cancer Center at Henry Ford Hospital
Detroit, Michigan, United States, 48202
United States, Minnesota
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
United States, Texas
Texas Children's Cancer Center
Houston, Texas, United States, 77030-2399
University of Texas - MD Anderson Cancer Center
Houston, Texas, United States, 77030-4009
United States, Vermont
Neurological Research Center, Inc.
Bennington, Vermont, United States, 05201
United States, Washington
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, United States, 98105
Sponsors and Collaborators
National Cancer Institute (NCI)
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Study Chair: Susan M. Blaney, MD Texas Children's Cancer Center

Layout table for additonal information Identifier: NCT00031928    
Other Study ID Numbers: CDR0000069240
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: April 30, 2015
Last Verified: November 2003
Keywords provided by National Cancer Institute (NCI):
leptomeningeal metastases
Additional relevant MeSH terms:
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Nervous System Neoplasms
Central Nervous System Neoplasms
Neoplasms by Site
Nervous System Diseases
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents
Immunosuppressive Agents
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists