Clinical Trials Logo

Clinical Trial 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


Clinical Trial 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 investigators´s assessment. Treatment is discontinued in case of progression, unacceptable toxicity or at the request of the patient. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05525273
Study type Interventional
Source Skane University Hospital
Contact Eva Marie Erfurth, MD. PhD.
Phone +4646172363
Email eva_marie.erfurth@med.lu.se
Status Recruiting
Phase Phase 2
Start date September 1, 2023
Completion date April 10, 2028

See also
  Status Clinical Trial Phase
Active, not recruiting NCT00949156 - Tumor Classification and Its Application in Surgical Treatment of Craniopharyngioma Phase 1
Recruiting NCT05465174 - Nivolumab and Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults Phase 2
Completed NCT02849743 - Intranasal Oxytocin in Hypothalamic Obesity Phase 2
Completed NCT01272622 - Prospective Study of Children and Adolescents With Craniopharyngioma N/A
Recruiting NCT04158284 - Multicenter Registry for Patients With Childhood.Onset Craniopharyngioma, Xanthogranuloma, Cysts of Rathke's Pouch, Meningioma, Pituitary Adenoma, Arachnoid Cysts
Completed NCT03194906 - Memantine for Prevention of Cognitive Late Effects in Pediatric Patients Receiving Cranial Radiation Therapy for Localized Brain Tumors Phase 2
Recruiting NCT06217848 - The Effect of GLP-1 Agonist in Patients With Hypothalamic Obesity: Prospective, Pilot Study Early Phase 1
Completed NCT01881854 - Sleep Wake and Melatonin Pattern in Craniopharyngioma N/A
Active, not recruiting NCT00517959 - SCRT Versus Conventional RT in Children and Young Adults With Low Grade and Benign Brain Tumors Phase 3
Not yet recruiting NCT06299891 - Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity Phase 2
Completed NCT02842723 - Management of Pediatric Craniopharyngioma by a Combination of Partial Surgical Resection, and Protontherapy (Craniopharyngioma) Phase 2
Recruiting NCT02792582 - A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection Phase 2
Completed NCT01484873 - Weight Loss Study for Patients With Obesity Due to Craniopharyngioma or Other Brain Tumor Phase 2
Withdrawn NCT03708913 - Neuromodulation for Hypothalamic Obesity N/A
Completed NCT02162732 - Molecular-Guided Therapy for Childhood Cancer N/A
Recruiting NCT00840047 - Methionine PET/CT Studies In Patients With Cancer Phase 2
Recruiting NCT04087902 - Long-Term Longitudinal QoL in Patients Undergoing EEA
Recruiting NCT04648462 - Proton Therapy Research Infrastructure- ProTRAIT- Neuro-oncology
Completed NCT04937335 - Craniopharyngioma With Tumoral Hemorrhage
Completed NCT03330080 - Examination of Sleep and Family Functioning in Pediatric Craniopharyngioma Patients