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RecruitingPhase 2NCT05465174

Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults

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Trial Parameters

ConditionCraniopharyngioma, Child
SponsorSabine Mueller, MD, PhD
Study TypeINTERVENTIONAL
PhasePhase 2
Enrollment57
SexALL
Min Age1 Year
Max Age39 Years
Start Date2022-09-12
Completion2027-03-01
Interventions
Tovorafenib

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Brief Summary

The current study assesses the tolerability and efficacy of monotherapy with pan-RAF-kinase (Tovorafenib) inhibition for the treatment of children and young adults with craniopharyngioma.

Eligibility Criteria

Inclusion Criteria: Newly Diagnosed Participants: * Newly diagnosed craniopharyngioma, as based on imaging characteristics and central radiology review. Participants will initially be screened within confines of a screening consent and only those participants with findings consistent with craniopharyngioma and without findings suggesting an indeterminate lesion or lesion of an alternative diagnosis (including abnormal tumor markers found in blood or cerebral spinal fluid (CSF), if completed as part of standard of care (SOC) work-up or if lesion concerning for alternate diagnosis) will move ahead with enrollment on the treatment protocol. Additionally, for participants that have undergone initial biopsy to confirm diagnosis, are within 6 weeks of radiographic diagnosis, and are planned to undergo follow up second surgery for additional tumor resection as per standard of care recommendations, these participants will also be considered eligible. * Participants must be surgical candidates

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