Craniopharyngioma Clinical Trial
— SwecranioOfficial title:
Neoadjuvant and Postoperative Treatment With Dabrafenib and Trametinib in BRAF Mutated Papillary Craniopharyngioma
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
Status | Recruiting |
Enrollment | 25 |
Est. completion date | April 10, 2028 |
Est. primary completion date | September 10, 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Sweden | Department of Endocrinology | Lund |
Lead Sponsor | Collaborator |
---|---|
Eva Marie Erfurth, MD, PhD | Novartis |
Sweden,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Levels of circulating mutated BRAF | BRAF assessed by plasma analysis and compared to baseline levels | 1 week after initiation of trial treatment | |
Other | Levels of circulating mutated BRAF | BRAF assessed by plasma analysis and compared to baseline levels | 2 weeks after initiation of trial treatment | |
Other | Levels of circulating mutated BRAF | BRAF assessed by plasma analysis and compared to baseline levels | 6 months after initiation trial treatment | |
Other | Levels of circulating mutated BRAF | BRAF assessed by plasma analysis and compared to baseline levels | 12 months after initiation trial treatment | |
Other | Levels of circulating of mutated BRAF | BRAF assessed by plasma analysis and compared to baseline levels | 3 months after end of trial treatment | |
Primary | 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 | 1 month to 5 years (sliding timepoints) | |
Secondary | Response ratio | Response ratio according to RECIST | 1 year after initiation of study treatment | |
Secondary | Response duration | Duration of response for patients treated without subsequent surgery | From time of study drug discontinuation to time of observed increased tumor volume assessed up to 1 year | |
Secondary | Operability after neoadjuvant trial treatment | Number of patients which become operable after neoadjuvant treatment | 1 year after initiation of study treatment | |
Secondary | Progression-free survival 1 year | Defined as unchanged or diminished tumor volume | 1 year | |
Secondary | Progression-free survival 2 years | Defined as unchanged or diminished tumor volume | 2 years | |
Secondary | QOL after treatment | Quality of life assessed by EQ5D5L (EuroQual 5 dimensions 5 levels) at 1 year after start of study treatment and compared to baseline | 1 year | |
Secondary | QOL after treatment | Quality of life assessed by EORTC QLQ30 (European Organisation for Research and Treatment of Cancer-Quality of Life Questionnaire 30) at 1 year after start of study treatment and compared to baseline | 1 year | |
Secondary | Cognitive status after treatment | Cognitive status assessed by CNS (central nervous system) Vital Signs 1 year after initiation of treatment and compared to baseline | 1 year | |
Secondary | Opthalmologic status after treatment | Opthalmologic status assessed as compound measure of visual acuity and visual field defects 1 year after initiation of treatment and compared to baseline | 1 year | |
Secondary | Hypothalamic status after treatment | Hypothalamic status assessed as compound measure of pituitary and hypothalamic status 1 year after initiation of treatment and compared to baseline | 1 year |
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