In short
Currently, there is one ongoing clinical trial investigating treatment options for craniopharyngioma, specifically focusing on the papillary subtype. This trial is examining a combination of targeted therapies administered before and after surgery to help shrink tumors and prevent recurrence in patients with a specific genetic mutation.
Clinical trial locations
A Study of Dabrafenib and Trametinib Treatment Before and After Surgery in Patients with Papillary Craniopharyngioma
This clinical trial is investigating a combination treatment approach for patients with papillary craniopharyngioma, a rare type of brain tumor that develops near the pituitary gland. The study focuses on using two targeted medications together: dabrafenib and trametinib, which are designed to work against tumors that have a specific genetic change called the BRAF V600E mutation.
Main inclusion criteria:
- Confirmed diagnosis of papillary craniopharyngioma through tissue examination
- Presence of BRAF V600E mutation in the tumor, verified by both special staining techniques and genetic testing
- Either newly diagnosed tumor or tumor that has returned after previous surgery where additional surgery would cause serious complications
- Age 18 years or older
- Ability to perform daily activities with a performance status score of 0-2 on the ECOG scale
- Normal functioning of major organs including liver, kidneys, and bone marrow
Main exclusion criteria:
- Age below 6 years or above 18 years
- Previous treatment with BRAF inhibitors
- Known allergy to dabrafenib or trametinib
- Active infections requiring treatment
- Significant heart problems or irregular heart rhythm
- Severe liver or kidney dysfunction
- Inability to undergo MRI scans
- Pregnancy or breastfeeding
- History of other cancers in the past 5 years (except successfully treated non-melanoma skin cancer)
Trial focus and goals: The primary aim of this study is to determine how effectively dabrafenib and trametinib can shrink tumors before and after surgery. Both medications are taken orally, with dabrafenib administered as capsules and trametinib as tablets. Treatment can continue for up to 12 months, and patients will undergo regular MRI scans to track changes in tumor size. Throughout the study, doctors will monitor several important health factors, including vision, cognitive function, hormone levels, and overall quality of life. The treatment may be given before surgery to help reduce tumor size or after surgery to prevent the tumor from returning. The follow-up period extends up to 2 years to assess long-term treatment effects.
Investigational drugs: Dabrafenib is a BRAF inhibitor that works by blocking a specific protein responsible for tumor growth in cells with the BRAF V600E mutation. Trametinib is a MEK inhibitor that blocks different proteins involved in cancer cell growth and survival. When used together, these medications provide a dual-blocking mechanism that can be more effective than either drug alone. This combination therapy targets the specific genetic abnormalities present in papillary craniopharyngioma tumors, aiming to shrink the tumor and prevent recurrence after surgical removal.
Summary
Currently, there is one active clinical trial for craniopharyngioma, specifically targeting the papillary subtype. This trial is being conducted in Sweden and represents a precision medicine approach, focusing on patients whose tumors have a specific genetic mutation (BRAF V600E). The study combines two targeted therapies, dabrafenib and trametinib, which work together to block cancer cell growth pathways. This trial is particularly significant for patients who have newly diagnosed tumors or those whose tumors have returned after surgery when additional surgery would be too risky. The research extends beyond simply measuring tumor shrinkage, also evaluating important quality-of-life factors such as vision, cognitive function, and hormone levels, with long-term follow-up of up to 2 years.
