Actively Recruiting
Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults
Led by Sabine Mueller, MD, PhD · Updated on 2026-04-23
57
Participants Needed
20
Research Sites
285 weeks
Total Duration
On this page
Sponsors
S
Sabine Mueller, MD, PhD
Lead Sponsor
D
Day One Biopharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Newly diagnosed craniopharyngioma confirmed by imaging and central radiology review
- Planned for standard of care biopsy or resection and surgical candidates
- Recurrent craniopharyngioma with histologic confirmation or imaging consistent with craniopharyngioma
- Age between 1 and 39 years
- Karnofsky performance score \u2265 50 for participants older than 16 years, Lansky score \u2265 50 for participants 16 years or younger
- Stable or decreasing dose of dexamethasone no higher than 0.1 mg/kg/day (maximum 4 mg/day) for at least 1 week prior to enrollment
- Adequate bone marrow, renal, liver, neurologic, and pulmonary function
- Willingness to provide archival tumor tissue if recurrent and not surgical candidate
- Ability to complete Pediatric Quality of Life Inventory (PedsQL) Core Module
- Consent and assent provided as appropriate
- Enrollment on PNOC COMP if open at the enrolling institution
You will not qualify if you...
- Prior tumor-directed therapy for newly diagnosed participants
- Chemotherapy or radiotherapy within 3 weeks (or 6 weeks for nitrosoureas or mitomycin C) before study entry for recurrent participants
- Not recovered from adverse events due to prior therapies
- Prior treatment with RAS-pathway inhibitors including tovorafenib
- Rapidly progressive symptoms requiring urgent surgery or radiation
- Uncontrolled neuroendocrine dysfunction symptoms
- Active or recent serious cardiovascular disease
- Allergic reactions to compounds similar to tovorafenib
- Significant nausea, vomiting, malabsorption, or bowel resection affecting drug absorption
- Uncontrolled infections or intercurrent illness
- Receiving other investigational agents
- Pregnant or breastfeeding women
- Current treatment with strong CYP2C8 inhibitors or inducers (except allowed medications)
- Inability to return for follow-up visits or assessments required for toxicity monitoring
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 20 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Rady Children's Hospital/University of California, San Diego
San Diego, California, United States, 92037
Actively Recruiting
3
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
4
University of Florida
Gainesville, Florida, United States, 32611
Actively Recruiting
5
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
6
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
7
John Hopkins University
Baltimore, Maryland, United States, 21218
Actively Recruiting
8
Dana-Farber/Boston Children's Harvard Medical School
Boston, Massachusetts, United States, 02215
Actively Recruiting
9
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
10
Children's Minnesota
Minneapolis, Minnesota, United States, 55404
Actively Recruiting
11
St. Louis Children's Hospital Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
12
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
13
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
14
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19103
Actively Recruiting
15
University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
16
John Hunter Children's Hospital
New Lambton Heights, New South Wales, Australia, 2310
Actively Recruiting
17
Royal Hobart Hospital
Hobart, Tasmania, Australia, 7000
Actively Recruiting
18
Children's Cancer Centre, Monash Children's Hospital
Clayton, VIC 3168, Australia
Actively Recruiting
19
Royal Children's Hospital, Childrens' Cancer Centre
Parkville, Victoria, Australia, 3052
Actively Recruiting
20
Perth Children's Hospital
Nedlands, Western Australia, Australia, 6009
Actively Recruiting
Research Team
P
PNOC Operations Office
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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