Actively Recruiting
Tovorafenib for the Treatment of Newly Diagnosed or Recurrent Craniopharyngioma in Children and Young Adults
Led by Sabine Mueller, MD, PhD · Updated on 2026-04-23
57
Participants Needed
20
Research Sites
52 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
Researchers are evaluating the tolerability and effectiveness of tovorafenib, a pan-RAF-kinase inhibitor, as a single treatment for children and young adults with newly diagnosed or recurrent craniopharyngioma. This phase 2 study aims to determine progression-free survival and maintenance of quality of life over 12 months, comparing results with historical data. The study also explores visual and neuroendocrine function over several years and investigates changes in tumor biology and patient satisfaction during therapy. Participants are divided into two groups: those with newly diagnosed craniopharyngioma and those with recurrent disease. All participants undergo biopsy or resection as standard care. Before surgery, patients receive one dose of tovorafenib. Those with measurable disease after surgery continue weekly maintenance doses. Those with complete tumor removal enter a follow-up phase. Non-surgical recurrent cases also receive weekly tovorafenib. Treatment is oral and given at a recommended phase 2 dose. Throughout the study, participants complete physical function and quality of life assessments, as well as visual and neuroendocrine evaluations at set intervals up to five years. Tissue and fluid samples collected before and after treatment help researchers study tumor changes at molecular levels. Patient-reported outcomes assess satisfaction and therapy-related side effects in context of social and racial factors. Safety is monitored continuously, and participation may last several years depending on follow-up needs.
CONDITIONS
Brief Title
Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Newly diagnosed craniopharyngioma based on imaging and central review
- Recurrent craniopharyngioma with histologic confirmation or imaging consistent with diagnosis
- Age between 1 and 39 years
- Surgical candidates planned for biopsy or resection or with available archival tumor tissue
- Measurable disease for participants continuing maintenance therapy
- Karnofsky score ≥ 50 (age > 16) or Lansky score ≥ 50 (age ≤ 16)
- Stable or decreasing corticosteroid use, with dose limits
- Adequate bone marrow, renal, liver, neurologic, and pulmonary function
- Willingness to use effective contraception if applicable
- Ability to understand and sign informed consent and complete quality of life assessments
You will not qualify if you...
- Previous tumor-directed therapy for newly diagnosed participants
- Chemotherapy or radiotherapy within 3 weeks prior to study entry (6 weeks for some drugs)
- Recent use of biologic or monoclonal antibody treatments within specified washout periods
- Rapidly progressive symptoms requiring urgent surgery or radiation
- Uncontrolled neuroendocrine dysfunction
- Significant active cardiovascular disease or recent heart events
- Allergic reactions to similar compounds as tovorafenib
- Severe nausea, vomiting, or malabsorption preventing adequate absorption
- Uncontrolled infections or other illnesses
- Current use of other investigational agents
- Pregnancy or breastfeeding in women of childbearing potential
- Use of strong CYP2C8 inhibitors or inducers not allowed by study
- Inability to return for follow-up visits or assessments required by the study
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 - 7 days ± 2 days
Participants receive one dose of tovorafenib within 7 days prior to their planned biopsy or resection.
1 visit (in-person) for dosing prior to biopsy or resection
Duration - Ongoing weekly dosing until disease progression or discontinuation
Participants undergo biopsy or resection and those with measurable disease continue on maintenance tovorafenib once weekly. Participants with gross total resection enter follow-up only.
Weekly visits for treatment and monitoring
Duration - Up to 5 years
Participants with gross total resection or those completing treatment are monitored for progression free survival, quality of life, and long-term visual and neuroendocrine function.
Periodic visits for up to 5 years for assessments
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
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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