Vemurafenib for Craniopharyngioma

Recruiting · 18+ · All Sexes · Salt Lake City, UT

This study is evaluating whether a combination of two drugs may help treat a type of brain tumor.

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About the trial for Craniopharyngioma

Eligible Conditions
Adamantinoma · Craniopharyngioma · BRAF V600E Mutation Present · Papillary 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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Laboratory Biomarker Analysis
Quality-of-Life Assessment
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved
FDA approved


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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Measurable disease and/or non-measurable disease
Progressive disease required in cohort B, defined as an increase in the bidirectional area by 25% within the past 13 months after surgery or radiation; progressive or recurrent disease is not required in cohort A, but is allowed provided it is a new diagnosis and patient has not received prior treatment.
Cohort A: No prior therapy received other than surgery
Cohort B: Prior radiation therapy required (any type of prior radiation is allowed)
For patients treated with external beam radiation therapy, interstitial brachytherapy or radiosurgery, an interval of >= 3 months must have elapsed from completion of radiation therapy to registration
Recovered to Common Terminology Criteria for Adverse Events (CTCAE) grade 1 or less toxicity attributed to radiation with exception of alopecia, fatigue
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 5 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 5 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

Overall survival
Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
Progression-free survival
Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
Response rate
Defined as the number of responses achieved during treatment with BRAF and MEK inhibitors divided by the total number of evaluable patients and assessed by contrast-enhanced magnetic resonance imaging or computed tomography. Point estimates will be generated for response rates within each cohort with corresponding 95% binomial confidence intervals. Simon's two-stage design with one interim analysis for futility will be applied to evaluate response rate within each cohort.

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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