Houston, Texas 77030

  • Brain and Central Nervous System Tumors


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.

Study summary:

OBJECTIVES: - 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.


DISEASE CHARACTERISTICS: - 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 PATIENT CHARACTERISTICS: Age: - Over 3 Performance status: - ECOG 0-2 Life expectancy: - At least 8 weeks Hematopoietic: - Not specified Hepatic: - No clinically significant liver function abnormalities Renal: - No clinically significant renal function abnormalities Other: - 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) PRIOR CONCURRENT THERAPY: Biologic therapy: - Recovered from prior immunotherapy Chemotherapy: - 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 Radiotherapy: - 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 Surgery: - Not specified Other: - 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

Study is Available At:

Original ID:




Secondary ID:


Study Acronym:

Brief Title:

Mafosfamide in Treating Patients With Progressive or Refractory Meningeal Tumors

Official Title:

Phase I Study of Intrathecal Mafosfamide

Overall Status:


Study Phase:

Phase 1



Minimum Age:

3 Years

Maximum Age:


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

National Cancer Institute (NCI)

Oversight Authority:

United States: Federal Government

Reasons Why Stopped:

Study Type:


Study Design:

Primary Purpose: Treatment

Number of Arms:


Number of Groups:


Total Enrollment:


Enrollment Type:


Overall Contact Information

Official Name:Susan M. Blaney, MD
Study Chair
Texas Children's Cancer Center

Study Dates

Start Date:January 2002
Verification Date:November 2003
Last Changed Date:April 29, 2015
First Received Date:March 8, 2002

Study Outcomes

There are no available Study Outcomes

Study Interventions

Intervention Type:Drug

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:NIH
Agency Type:Lead Sponsor
Agency Name:National Cancer Institute (NCI)

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: April 03, 2020

Modifications to this listing: Only selected fields are shown, please use the link below to view all information about this clinical trial.

This study is not currently recruiting Study Participants. The form below is not enabled.