Phase I Study of Chemokine and Cytokine Gene Modified Autologous Neuroblastoma Cells for Treatment of Relapsed/Refractory Neuroblastoma Using an Adenoviral Vector (CYCHE)
Neuroblastoma cells taken from the patient will be separated in the laboratory and two
specially produced human viruses (adenovirus) that carry the lymphotactin and the IL-2 gene
were put into the cells. 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. We do not know the best amount of special cells to use, so
different patients will get different numbers of cells.
Before the second shot, and then again about 2 weeks later, we will remove some of the
modified cells from the patient's body and study them. We will do this by removing a section
of skin (referred to as a skin biopsy) at the place where the cells were injected. This test
will help us to see whether or not the modified cells are killing cancer cells.
To study how the immunity is working in the patient's system, we will take blood samples
prior to each injection, two to four days after each injection, and may repeat this 5-7 days
after each injection if your doctor thinks that is necessary.
After patients have received the first set of 4 injections a complete evaluation is
performed to see how this research treatment is working. If these evaluations look okay and
if more injections are available, patients will have the option to receive a second set of 4
injections. If patients receive the second set of 4 injections, they will again have blood
samples taken after each injection.
After injections stop, all patients will have blood samples collected once a month for a
year, and then once a year for fifteen years.
To see if this research treatment is working, we will do CAT scans, MRIs, or bone scans
(these are different types of x-rays). We 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 again eight weeks later. In addition, if patients
receive the second set of four injections, these tests will be repeated at six months.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To determine the safety of up to four subcutaneous (SC) injections of autologous neuroblastoma cells which have been genetically modified by adenoviral vectors to secrete lymphotactin (Lptn) and Interleukin-2 (IL-2).
Malcolm Brenner, MD
Baylor College of Medicine
United States: Food and Drug Administration
|Texas Children's Hospital||Houston, Texas|