Ignite Creation Date:
2024-05-06 @ 6:02 PM
Last Modification Date:
2024-10-26 @ 2:40 PM
Study NCT ID:
NCT05525273
Status:
RECRUITING
Last Update Posted:
2024-02-14
First Post:
2022-04-20
Brief Title:
Treatment of BRAF B-Rapidly Accelerated Fibrosarcoma Mutated Papillary Craniopharyngioma
Sponsor:
Eva Marie Erfurth MD PhD
Organization:
Skane University Hospital
Study Overview
Official Title:
Neoadjuvant and Postoperative Treatment With Dabrafenib and Trametinib in BRAF Mutated Papillary Craniopharyngioma
Status:
RECRUITING
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Swecranio
Brief Summary:
Subjects with papillary craniopharyngioma harboring a BRAF mutation will be treated with a BRAF MEK inhibitor dabrafenib trametinib after informed consent Study participants will be administered oral dabrafenib and trametinib until maximal tumor volume reduction assessed by MRI Progression free survival cognition ophthalmologic status hypothalamic status and quality of life will be assessed 1 year after initiation of study treatment
Detailed Description:
Background Papillary craniopharyngioma harbours a BRAF mutation in 90 of cases Treatment with BRAF MEK mitogen activated protein kinase inhibitors dabrafenib trametinib may prevent patients from undergoing surgery with a high risk of serious side effects or provide an additional treatment option when further surgery is not advised
Study intervention Subjects with newly diagnosed craniopharyngioma where radical surgery is not considered adequate or patients with recurrence of craniopharyngioma where further surgery is not considered possible without serious sequelae will be asked for informed consent Study participants are treated continuously with dabrafenib and trametinib orally until maximal tumor shrinkage Evaluation is done by MRI to measure tumor volume as well as assessment of performance status quality of life cognition ophthalmologic status performance status and hypothalamic status
Study type The study is a Phase II single armed open label and multicenter study Study drugs are Dabrafenib Tafinlar and trametinib Mekinist Primary outcome To evaluate tumor response in the form of reduced tumor volume on MRI in patients with papillary craniopharyngioma during treatment with dabrafenib and trametinib
Secondary outcomes
To evaluate dabrafenib and trametinib treatment for the following aspects
response according to RECIST Duration of response for patients treated without subsequent surgery
how many patients become operable after neoadjuvant treatment
progression-free survival after 1 and 2 years
quality of life during and after treatment The effect of treatment on vision cognition and hypothalamic effects Exploratory outcomes Levels of circulating BRAF Trial population 25 patients Trial duration Participants are treated with the study treatment for at least one year if the treatment is well tolerated to maximum tumor reduction or longer according to the investigatorss assessment Treatment is discontinued in case of progression unacceptable toxicity or at the request of the patient
Study intervention Subjects with newly diagnosed craniopharyngioma where radical surgery is not considered adequate or patients with recurrence of craniopharyngioma where further surgery is not considered possible without serious sequelae will be asked for informed consent Study participants are treated continuously with dabrafenib and trametinib orally until maximal tumor shrinkage Evaluation is done by MRI to measure tumor volume as well as assessment of performance status quality of life cognition ophthalmologic status performance status and hypothalamic status
Study type The study is a Phase II single armed open label and multicenter study Study drugs are Dabrafenib Tafinlar and trametinib Mekinist Primary outcome To evaluate tumor response in the form of reduced tumor volume on MRI in patients with papillary craniopharyngioma during treatment with dabrafenib and trametinib
Secondary outcomes
To evaluate dabrafenib and trametinib treatment for the following aspects
response according to RECIST Duration of response for patients treated without subsequent surgery
how many patients become operable after neoadjuvant treatment
progression-free survival after 1 and 2 years
quality of life during and after treatment The effect of treatment on vision cognition and hypothalamic effects Exploratory outcomes Levels of circulating BRAF Trial population 25 patients Trial duration Participants are treated with the study treatment for at least one year if the treatment is well tolerated to maximum tumor reduction or longer according to the investigatorss assessment Treatment is discontinued in case of progression unacceptable toxicity or at the request of the patient
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None