Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma

• Status: Not yet recruiting
• Trial end date: 2028-04-10
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Eva Marie Erfurth, MD, PhD
• Collaborator: Novartis
• Treatments: Dabrafenib; Trametinib

Criteria

Inclusion Criteria:

1. Histologically verified papillary craniopharyngioma.

2. BRAF mutated V600E (valine 600 glutamine), verified immunohistochemically and by molecular genetic analysis

3. 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.

4. Age over 18 years

5. Functional status according to ECOG (Eastern Cooperative Oncology Group performance status) 0-2

6. Adequate organ function:

neutrophils> 1.5 x 109 platelets> 100 x 109 creatinine <1.5 x ULN (upper limit of normal) or creatinine clearance <45 ml / min bilirubin <1.5 x ULN ASAT (aspartate aminotransferase) / ALAT (alanine aminotransferase) <2.5 x ULN

7. Ability to understand and give informed consent.

8. Previous cancer, which does not require current treatment is allowed.

9. The patient agrees to use an adequate method to avoid pregnancy.

Exclusion Criteria:

1. Ongoing treatment in another drug study or other experimental treatment.

2. Previous treatment with BRAF or MEK inhibitors.

3. Hypersensitivity to study drugs.

4. 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.

5. Known cardiovascular disease where treatment with MEK inhibitors is considered inappropriate, eg severe heart failure, prolongation of QT time, uncontrolled arrhythmia, recent (<6 months) cardiac infarction, uncontrolled hypertension.

6. Active bleeding; intracranial hemorrhage last 4 weeks before inclusion.

7. Thromboembolic disease last 6 months and unstable anticoagulant treatment less than 4 weeks before inclusion.

8. Women who are pregnant or breastfeeding.

9. Previous central serous retinopathy or retinal vein occlusion.

10. Previous uveitis or iritis last 4 weeks before inclusion.

11. Surgery within the last 3 weeks.

12. For postoperative patients; radiation therapy within the last 3 months.