CONNECT1905: Treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma
What is the Purpose of this Study?
People in this study will visit the research center two times each month to see the study doctor. The study can last up to 2 years. Treatment happens in cycles, and each cycle is 28 days long, which is about 4 weeks. A person can have up to 26 cycles, which equals about 2 years of treatment. The amount of medicine given will depend on the person’s weight, which is checked before each new cycle. The medicine is called tocilizumab. It blocks a protein called interleukin-6, which causes swelling in the body. This medicine is usually used for diseases where the immune system attacks the body, like arthritis, and it can also help with a problem called cytokine release syndrome after certain cancer treatments.
This study is for a rare brain tumor called Adamantinomatous craniopharyngioma, or ACP. It is not cancer, but it mostly affects children.
Who Can Participate in the Study?
Age: Patients must be at least 1 year old and no older than 39 years when they join.
Diagnosis: Patients must have a confirmed diagnosis of adamantinomatous craniopharyngioma (ACP).
This can be proven by looking at tumor tissue under a microscope.
If tissue cannot be safely taken, doctors may test cyst fluid. ACP cyst fluid is usually thick, cholesterol‑rich, and greenish‑brown.
Imaging scans should show a lobulated mass (part solid, part cystic) with calcifications in the sellar/suprasellar region of the brain.
Disease Status: Patients must have a tumor that can be measured on scans.
What is Involved?
This study wants to learn how a medicine called tocilizumab works for people who have a brain tumor called Adamantinomatous craniopharyngioma, or ACP. Doctors will check both the good effects and the bad effects of the medicine.