Actively Recruiting
Neoadjuvant and Postoperative Treatment With Dabrafenib and Trametinib in BRAF Mutated Papillary Craniopharyngioma
Led by Eva Marie Erfurth, MD, PhD · Updated on 2024-02-14
25
Participants Needed
1
Research Sites
30 weeks
Total Duration
On this page
Sponsors
E
Eva Marie Erfurth, MD, PhD
Lead Sponsor
N
Novartis
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating treatment for patients with papillary craniopharyngioma that has a BRAF mutation, a condition affecting the brain. This Phase II, open-label study explores using a combination of two oral drugs, dabrafenib and trametinib, to shrink tumors and possibly avoid or delay surgery, which can have serious side effects. About 25 patients will participate to assess tumor response and other health outcomes over time. Participants receive oral dabrafenib at 75 mg twice daily and trametinib at 2 mg once daily continuously until MRI scans show maximal tumor reduction or until treatment is stopped due to progression, side effects, or patient choice. The study includes patients newly diagnosed or with recurrent tumors not suitable for radical surgery. Treatment duration is at least one year, with ongoing evaluation by investigators. During the study, participants undergo regular MRI scans to measure tumor size and assessments of cognition, vision, hypothalamic function, and quality of life. Researchers will monitor progression-free survival and tumor response up to five years. Safety and tolerability are tracked, and quality of life assessments occur during and after treatment. The study aims to provide detailed information on how the drugs affect tumor control and patient well-being.
CONDITIONS
Brief Title
Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed papillary craniopharyngioma
- Verified BRAF V600E mutation by immunohistochemistry and molecular genetics
- Newly diagnosed tumor or recurrence where surgery is not safe or possible without serious side effects
- Age over 18 years
- ECOG performance status 0 to 2
- Adequate organ function including neutrophils >1.5 x 10^9, platelets >100 x 10^9, creatinine <1.5 x ULN or creatinine clearance <45 ml/min, bilirubin <1.5 x ULN, ASAT/ALAT <2.5 x ULN
- Ability to understand and give informed consent
- Previous cancer allowed if no current treatment needed
- Agreement to use effective contraception to avoid pregnancy
You will not qualify if you...
- Current participation in another drug or experimental treatment study
- Prior treatment with BRAF or MEK inhibitors
- Allergy or hypersensitivity to study drugs
- Ongoing use of unauthorized drugs that strongly affect CYP2C8 or CYP3A4 enzymes unless stopped 14 days before
- Significant cardiovascular disease making MEK inhibitor treatment unsafe
- Active bleeding or intracranial hemorrhage within 4 weeks before enrollment
- Recent thromboembolic disease within 6 months or unstable anticoagulant therapy within 4 weeks
- Pregnant or breastfeeding women
- Previous central serous retinopathy or retinal vein occlusion
- Recent uveitis or iritis within 4 weeks
- Surgery within the last 3 weeks
- Radiation therapy within 3 months for postoperative patients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 1 year or longer according to investigator's assessment
Participants receive oral dabrafenib and trametinib continuously until maximal tumor shrinkage, unacceptable toxicity, progression, or patient request to stop.
Visits occur periodically for treatment and assessments including MRI scans and evaluations of vision, cognition, quality of life, and hypothalamic status
Trial Site Locations
Total: 1 location
1
Department of Endocrinology
Lund, Sweden, 22185
Actively Recruiting
Research Team
E
Eva Marie Erfurth, MD. PhD.
S
Sara Kinhult, MD. PhD.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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