Vemurafenib for Craniopharyngioma
This study is evaluating whether a combination of two drugs may help treat a type of brain tumor.
See full descriptionAbout the trial for Craniopharyngioma
Treatment Groups
This trial involves 2 different treatments. Vemurafenib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
About The Treatment
Eligibility
This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Craniopharyngioma or one of the other 3 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Vemurafenib will improve 1 primary outcome and 2 secondary outcomes in patients with Craniopharyngioma. Measurement will happen over the course of Up to 5 years.
Patient Q & A Section
What is craniopharyngioma?
This is the first report that includes surgical intervention with cure rate for patients with craniopharyngioma at our institution. Findings from a recent study group, only a small percentage received the option of radiotherapy, but this group also experienced excellent cure rates. Despite this, no deaths or significant neurological deterioration rates were observed.
Can craniopharyngioma be cured?
Tumor growth is not completely inhibited by standard radiotherapy. However, the incidence and severity of tumor-induced morbidity and mortality drops significantly for patients who receive a total dose of radiation of more than 53 Gy. Patients who have incomplete tumor debulking surgery will still suffer from long-term tumor-related complications, requiring re-treatment and further hospitalization.
What are the signs of craniopharyngioma?
There is no single sign to indicate a craniopharyngioma with certainty; therefore, all symptoms of the disease must be considered. Symptoms must be present for a period of time to be considered a reliable sign of craniopharyngioma.
What are common treatments for craniopharyngioma?
The treatment of craniopharyngioma should follow existing guidelines for the appropriate treatment of each case. The treatment strategies should be customized to individual patient needs using existing reviews and consensus guidelines. The best treatment approach for each specific case has yet to be established.
What causes craniopharyngioma?
Craniopharyngioma is frequently associated with exposure to ionizing radiation, but no one exposure has been proven to be a cause. In some rare cases, this tumor is associated with inherited conditions.
How many people get craniopharyngioma a year in the United States?
In this country, approximately 2,000 Americans have a craniopharyngioma at the time the study began and only approximately 100,000 are lifetime risk, which would warrant a prospective screening program.
Has vemurafenib proven to be more effective than a placebo?
Vemurafenib resulted in significantly better results versus a placebo. A small risk of venous thrombosis was confirmed. Vemurafenib should be available for patients with early stage pituitary disease who are not candidates for radiotherapy.
What does vemurafenib usually treat?
Although it was known that the BRAF (V850E) mutation can be a driver of many malignancies, the discovery of BRAF in tumor cells in the setting of a pediatric craniopharyngioma opened the door to novel treatment strategies and a better understanding of the tumor biology of this rare subtype of neurocutaneous cancers.
Is vemurafenib typically used in combination with any other treatments?
Vemurafenib has been found to be effective in combination with radiotherapy and/or chemoradiation in people with papillary thyroid cancer. A recent study was able to demonstrate that vemurafenib is effective even while given concurrently with chemoradiation. This drug typically is used in combination with radiation for some other conditions, and the results of this study suggest that it may have some potential to improve outcomes for patients with craniopharyngioma. This drug may also be used in combination with other agents. Because of the lack of data concerning its effectiveness, patients should be discussed about its role with their physicians.
What is the primary cause of craniopharyngioma?
Craniodiagnostic workup and assessment of IGF1 and IGFBP3 levels should be considered in patients with craniopharyngioma. We recommend that serum insulin levels be measured in patients with CRF or CPH. Additionally, IGF1 levels should be measured if CRF or CPH is diagnosed. A IGF1 level of>16-23 pg/ml is considered a high normal value. Interestingly, in this study, all patients with craniopharyngioma showed elevated IGF1 levels compared to the general population. This finding suggests that IGF1 does play an important role in craniopharyngioma pathobiology. Furthermore, IGFBP3 levels are often elevated in CRF or CPH.
Does vemurafenib improve quality of life for those with craniopharyngioma?
Data from a recent study indicate that in patients with craniopharyngioma treated with vemurafenib, improvements in HRQoL, pain, and cognitive function are seen. Patients who have a good initial response experience significantly better long-term outcomes, regardless of disease progression.
Does craniopharyngioma run in families?
These data support a genetic susceptibility to the development of a craniopharyngioma and a family history of the tumor. Further studies to delineate the genetic basis for this association may result in further insight into the etiology of these malignancies.