NCT00062881

Brief Summary

This research study is an investigational treatment with the experimental drug Mafosfamide. This treatment is only for children with cancer that has spread to the meninges (tissues that cover the spinal column and brain) and has continued to grow despite treatment with standard therapy. Mafosfamide is a drug reported to have antitumor effects in animals and that has been given safely into the cerebrospinal fluid (the fluid within and surrounding the brain) in a small number of children and adults. Since there is limited experience in adults and children in giving mafosfamide in this way, the main purpose of this study is to determine the appropriate safe dose of mafosfamide when given intrathecally, that is directly into the cerebrospinal fluid. The purposes for this study are to (a) determine what dose of mafosfamide can be safely given into the cerebrospinal fluid through an Ommaya reservoir (surgically implanted catheters used to sample cerebrospinal fluid and to instill medication into the cerebrospinal fluid) and lumbar puncture (spinal tap) or lumbar reservoir; (b) look for side effects of drug treatment; (c) to study the pharmacology (how the human body handles the drug) when given directly into the spinal fluid; and (d) see if this drug is beneficial to the patient.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
65

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jun 1990

Longer than P75 for phase_1

Geographic Reach
1 country

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 1990

Completed
13.1 years until next milestone

First Submitted

Initial submission to the registry

June 17, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 18, 2003

Completed
2.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2005

Completed
Last Updated

March 4, 2020

Status Verified

March 1, 2020

First QC Date

June 17, 2003

Last Update Submit

March 2, 2020

Conditions

Keywords

CancerLeukemiaLymphomaSolid Tumor

Interventions

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

  • Receiving other therapy (either intrathecal or systemic) designed specifically to treat their meningeal malignancy are not eligible for this study. However, patients receiving concomitant chemotherapy to control systemic or bulk CNS disease will be eligible, provided the systemic chemotherapy is not a phase I agent, an agent which significantly penetrates the CNS (e.g., high dose methotrexate, (\> 1 gm/m2), thiotepa, high dose cytarabine, (\> 2 gm/m2 per day), 5-fluorouracil, intravenous 6-mercaptopurine or topotecan), or an agent known to have serious unpredictable CNS side effects.
  • Clinical evidence of obstructive hydrocephalus or compartmentalization of the CSF flow as documented by a radioisotope Indium111 or Technitium99-DTPA flow study are not eligible for this protocol. If a CSF flow block or compartmentalization is demonstrated, focal radiotherapy to the site of block to restore flow and a repeat CSF flow study showing clearing of the blockage is required for the patient to be eligible for the study.
  • Patients who have leukemia or lymphoma and a concomitant bone marrow relapse.
  • Women of childbearing age must not be pregnant or lactating.
  • Patients must not have received any other systemic investigational agent within 14 days prior to, or during, study treatment. The 14 day period should be extended if the patient received any investigational agent which is known to have delayed toxicities after 14 days. Patients must not have received any other intrathecal investigational within 7 days prior to, or during, study treatment. The 7 day period should be extended if the patient received any investigational agent which is known to have delayed toxicities after 7 days or a prolonged half-life.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Children'sHospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

Pediatric Branch, National Cancer Institute

Bethesda, Maryland, 20892, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Neurological Research Center

Bennington, Vermont, 05210, United States

Location

Children's Hospital and Medical Center

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Meningeal NeoplasmsNeoplasmsLeukemiaLymphoma

Condition Hierarchy (Ancestors)

Central Nervous System NeoplasmsNervous System NeoplasmsNeoplasms by SiteNervous System DiseasesNeoplasms by Histologic TypeHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor, Pediatrics-Hema & Oncology

Study Record Dates

First Submitted

June 17, 2003

First Posted

June 18, 2003

Study Start

June 1, 1990

Study Completion

September 1, 2005

Last Updated

March 4, 2020

Record last verified: 2020-03

Locations