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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I Study of Intrathecal Mafosfamide|
|Study Start Date:||June 1990|
|Estimated Study Completion Date:||September 2005|
Mafosfamide is given directly into the cerebrospinal fluid twice a week for six successive weeks. If after six weeks the disease has not shown any evidence of progression (tumor growth) patients will continue to receive intrathecal (I.T.) mafosfamide once a week for one month, followed by twice monthly (every other week) for four months, followed by once a month. Patients may continue to receive mafosfamide as long as unacceptable side effects do not occur and there is no growth of the tumor.
Patients will have a weekly physical exam and blood tests. The cerebrospinal fluid will be tested each time they receive drug for the presence of cancer cells. If the patient has an Ommaya reservoir (a surgically implanted catheter that is used to sample cerebrospinal fluid from the fluid chambers in the head and to install medication into the cerebrospinal fluid) then the doctor may recommend that they receive one dose of mafosfamide through the Ommaya reservoir and the next dose via lumbar puncture (spinal tap) or lumbar reservoir. A lumbar reservoir is a catheter that is surgically implanted into the lower back. This catheter is used to sample cerebrospinal fluid and to install medication into the cerebrospinal fluid.
All patients must be observed for a minimum of eight hours after the first dose of intrathecal mafosfamide. If the first dose is well tolerated (there are no major side effects), further doses of mafosfamide will be administered with close observation for at least two hours after administration during the first six weeks of drug administration. Administration of the mafosfamide and sampling of the cerebrospinal fluid from the Ommaya reservoir, lumbar reservoir, or spinal tap takes about 30 minutes. In addition, after mafosfamide has been given by lumbar puncture, patients must remain lying down on their stomach for one hour.
In patients who have Ommaya reservoirs or lumbar catheters, samples of spinal fluid will be taken from the reservoirs following the first two doses of mafosfamide. For each series of tests, a total of eight spinal fluid samples will be taken with a needle from the Ommaya reservoir over a period of 24 hours after the dose. A spinal tap will be also be performed two hours after the dose on these two days. If you have a lumbar reservoir, eight spinal fluid samples will be taken with a needle from the Ommaya reservoir, over a period of 24 hours after the dose. These samples of cerebrospinal fluid will be used to measure the amount of mafosfamide found in the cerebrospinal fluid at the time they are drawn. This will help us understand how the body handles mafosfamide.
For patients with leukemia, a bone marrow aspiration (taking some bone marrow out of the pelvis bone) is necessary before receiving intrathecal mafosfamide. This is to make sure that there is no cancer in the bone marrow.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00062881
|United States, California|
|Children'sHospital Los Angeles|
|Los Angeles, California, United States, 90027|
|United States, District of Columbia|
|Children's Hospital National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Florida|
|Jacksonville, Florida, United States, 32224|
|United States, Georgia|
|Children's Healthcare of Atlanta|
|Atlanta, Georgia, United States, 30342|
|United States, Maryland|
|Pediatric Branch, National Cancer Institute|
|Bethesda, Maryland, United States, 20892|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|United States, Texas|
|M.D. Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|United States, Vermont|
|Neurological Research Center|
|Bennington, Vermont, United States, 05210|
|United States, Washington|
|Children's Hospital and Medical Center|
|Seattle, Washington, United States, 98105|