Phase I Study of Intrathecal Mafosfamide
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.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|
|Mayo Clinic||Jacksonville, Florida 32224|
|M.D. Anderson Cancer Center||Houston, Texas 77030|
|Texas Children's Hospital||Houston, Texas|
|Children'sHospital Los Angeles||Los Angeles, California 90027|
|Children's Hospital National Medical Center||Washington, District of Columbia 20010|
|Children's Healthcare of Atlanta||Atlanta, Georgia 30342|
|Pediatric Branch, National Cancer Institute||Bethesda, Maryland 20892|
|Neurological Research Center||Bennington, Vermont 05210|
|Children's Hospital and Medical Center||Seattle, Washington 98105|