Phase 1 Study of Chemokine and Cytokine Gene Modified Allogeneic Neuroblastoma Cells for Treatment of Relapsed/Refractory Neuroblastoma Using a Retroviral Vector (CYCHEALL)
The investigators have grown neuroblastoma cells in the laboratory and put into them two
specially produced mouse viruses (retroviruses) that carry the lymphotactin and the IL-2
gene. These lymphotactin and IL-2 genes are meant to help the immune system fight the
cancer. The modified cancer cells will be injected under the patient's skin. There will be
four shots. The second and subsequent shots will have ten times as many cells producing
lymphotactin as the first. The patient will normally have these shots as an outpatient.
Depending on response (if the patient's cancer has stayed the same or gotten smaller), the
patient may be able to have four more of these shots.
Tests during and after treatment:
If the patient's blood has not been tested for HIV, the virus that causes AIDS, then the
investigators will need to do this test. If this virus is present, this research treatment
cannot be used.
Before the second shot, and then again about 2 weeks later, the investigators will remove
some of the modified cells from the patient's body and study them. This will be done by a
skin biopsy at the place where the cells were injected. These tests are to see whether the
changes made in the laboratory are killing cancer cells.
To study how the immunity is working in the patient's body blood samples will be taken two
to four days after each injection. This may be repeated 5-7 days after each injection if the
patient's doctor thinks that it is necessary. After the patient stops receiving the
injections, the blood draws will need to be repeated once a month for a year, and then once
a year for fifteen years.
The patient will need to come to the clinic on the days the investigators take blood and
will need to be seen at Texas Children's Cancer Center at weekly intervals for 6 weeks, then
every other week for 6 weeks, and then monthly for a year. Then the patient will be seen in
clinic or contacted by study personnel once a year for 15 years. Additional visits may be
To see if this research treatment is working, CAT scans, MRIs, or bone scans (these are
different types of x-rays)will be done. Investigators will also take a bone marrow biopsy
(bone marrow will be removed by use of a needle so that it can be looked at under the
microscope). These tests will be done prior to treatment and repeated eight weeks later. In
addition, if the patient receives the second set of four injections, these tests will be
repeated at six months. If the neuroblastoma is not responding (if it has gotten larger),
the investigators will offer the patient treatment with chemotherapy and/or radiation
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To assess the safety of up to four subcutaneous (SC) injections of allogeneic neuroblastoma cells which have been genetically modified by measuring adverse events.
The patients will be given four injections as per the protocol schedule with the evaluation at Week 8 (month 2). This will constitute a course and the evaluation of the dose limiting toxicity will be done during this course. Any patient having grade 4 toxicity or having progressive disease during the course will be considered a failure.
Malcom Brenner, MD, PhD
Texas Children's Hospital
United States: Food and Drug Administration
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