Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma
ConditionCraniopharyngioma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorEva Marie Erfurth, MD, PhD
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Histologically verified papillary craniopharyngioma.
BRAF mutated V600E (valine 600 glutamine), verified immunohistochemically and by molecular genetic analysis
Newly diagnosed tumor, or recurrence after previous surgery, where surgery is not considered to be able to be performed radically without the risk of serious or permanent sequelae.
Age over 18 years
Disqualifiers
Ongoing treatment in another drug study or other experimental treatment.
Previous treatment with BRAF or MEK inhibitors.
Hypersensitivity to study drugs.
Ongoing treatment with non-authorized drugs, (strong inducers of CYP2C8 or CYP3A4). If the patient is on unauthorized drugs, they must be discontinued at least 14 days before inclusion.
Trial design
Design model
Single group
Treatments tested in this trial
Oral dabrafenib and trametinib
DrugNeoadjuvant or postoperative treatment of patients with verified BRAF mutated papillary craniopharyngioma
Treatment groups
25 Participants
are divided into 1 treatment groupGroup A: Dabrafenib and trametinibExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Tumor response
To evaluate tumor response measured as maximally reduced tumor volume on MRI during treatment with dabrafenib and trametinib. Maximally reduced volume is defined as the time point where no further reduction of tumor volume can be observed
Time frame
1 month to 5 years (sliding timepoints)Secondary outcomes
1
Response ratio
Response ratio according to RECIST
Time frame
1 year after initiation of study treatment2
Response duration
Duration of response for patients treated without subsequent surgery
Time frame
From time of study drug discontinuation to time of observed increased tumor volume assessed up to 1 year3
Operability after neoadjuvant trial treatment
Number of patients which become operable after neoadjuvant treatment
Time frame
1 year after initiation of study treatment4
Progression-free survival 1 year
Defined as unchanged or diminished tumor volume
Time frame
1 yearOther outcomes
1
Levels of circulating mutated BRAF
BRAF assessed by plasma analysis and compared to baseline levels
Time frame
1 week after initiation of trial treatment2
Levels of circulating mutated BRAF
BRAF assessed by plasma analysis and compared to baseline levels
Time frame
2 weeks after initiation of trial treatment3
Levels of circulating mutated BRAF
BRAF assessed by plasma analysis and compared to baseline levels
Time frame
6 months after initiation trial treatment4
Levels of circulating mutated BRAF
BRAF assessed by plasma analysis and compared to baseline levels
Time frame
12 months after initiation trial treatmentSponsors and contacts
Click on the lead sponsor to view all of their trials.
Eva Marie Erfurth, MD, PhD
Lead sponsor
Skane University Hospital
Sponsor institution
Novartis
Collaborator
Source ClinicalTrials.gov