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New pill shows promise against childhood brain tumor

NCT ID NCT05465174

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is testing a drug called Tovorafenib for children and young adults with craniopharyngioma, a rare, non-cancerous brain tumor that can cause serious health problems. The drug is taken by mouth and works by blocking a protein that helps the tumor grow. The trial aims to see if the drug can stop the tumor from growing and improve patients' quality of life. About 57 participants with newly diagnosed or recurrent tumors will be enrolled.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Tovorafenib (a targeted drug taken orally)
What this could lead to
If successful, this could provide a new, less invasive treatment option for craniopharyngioma, potentially delaying or avoiding surgery and radiation in children and young adults.
What could go wrong
This is a small, early-phase (Phase 2) trial with only 57 participants. The drug may not work for everyone, and side effects are possible. It is not a cure, and long-term disease control is uncertain.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 57 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2022

Expected to finish

Mar 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 39 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 for biopsy or resection and planned for standard of care biopsy or resection. Recurrent Participants: * Recurrent craniopharyngioma, as based on histologic confirmation at time of initial diagnosis (participants with Adamantinomatous craniopharyngioma (ACP) will only be eligible for the recurrent arm). * Recurrent craniopharyngioma without prior histologic confirmation 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 CSF, if completed as part of SOC work-up or if lesion concerning for alternate diagnosis) will move ahead with enrollment on the treatment protocol. * Participants should be surgical candidates for biopsy or resection. If participants are not surgical candidates, but have available archival tumor tissue, they will be enrolled into the exploratory cohort. * Participants must be willing to provide archival tissue, a minimum of 10-20 paraffin embedded unstained slides OR 1 block with tumor content of 40% or greater is required. Participants who do not meet this criteria may be discussed on a case-by-case basis with the Study Chair(s). * Participants can have been previously treated with surgical resection alone, cyst drainage and biopsy alone, radiation therapy, other systemic therapies, or any combination thereof. * Prior Therapy: * Had their last dose of myelosuppressive chemotherapy \>= 21 days prior to study registration (\>=42 days if nitrosourea therapy). * Had their last dose of hematopoietic growth factor \>=14 days (long-acting growth factor) or \>=7 days (short-acting growth factor) prior to study registration, or beyond the time during which adverse events (AEs) are known to occur. * Had their last dose of biologic (anti-neoplastic agent) \>=7 days prior to study registration, or beyond the time during which AEs are known to occur. * Had their last dose of monoclonal antibodies \>=21 days prior to study registration. Radiation: * Had their last fraction of local irradiation to primary tumor \>=12 weeks prior to registration; investigators are reminded to review potentially eligible cases to avoid confusion with pseudo-progression. * At least 14 days after local palliative radiation (small-port). All Participants: * Age 1 to 39 years. * Participants continuing on maintenance therapy after standard of care biopsy/resection must have measurable disease, as defined as lesions that can be accurately measured in two dimensions (longest diameter to be recorded) with a minimum size of no less than double the slice thickness. Previously irradiated lesions are considered non-measurable except in cases of documented progression of the lesion since the completion of radiation therapy. Participants without measurable disease may continue on study and will be followed for study endpoints, but will not be included as part of target accrual. * Performance Score: Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants \<= 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score. * Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. The participant steroid dose should be no more than a steroid-equivalent of dexamethasone 0.1 mg/kg/day (or maximum 4mg/day; whichever is the lower dose) at time of enrollment. Participants that have been stable on physiologic hormone replacement for hypopituitarism are allowed. * Organ Function Requirements: * Adequate Bone Marrow Function defined as: * Peripheral absolute neutrophil count (ANC) \>=1000/mm3. * Platelet count \>= 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment). * Adequate Renal Function defined as- ---A serum creatinine \< 1.5 Upper Limit normal (ULN) based on age and gender. * Adequate Liver Function defined as- * Bilirubin (sum of conjugated + unconjugated) \<= 1.5 x upper limit of normal (ULN) for age (except in participants with documented Gilbert syndrome). * Serum glutamic-pyruvic transaminase (SGPT)((alanine aminotransferase (ALT)) \<= 3 x ULN. * Serum albumin \>=2 g/dL (20g/L). * Adequate Neurologic Function defined as participants with seizure disorder may be enrolled if well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug. * Adequate Pulmonary Function defined as no evidence of dyspnea at rest, no exercise intolerance due to pulmonary insufficiency, and a pulse oximetry of \> 92% while breathing room air. * prothrombin time (PT) /partial thromboplastin time (PTT)/International Normalized Ratio (INR) within institutional normal limits or deemed appropriate for surgical intervention by the treating team for patients undergoing surgery biopsy/resection * The effects of Tovorafenib on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (non-hormonal contraception; barrier method of birth control; abstinence - note, tovorafenib can make hormonal contraceptives ineffective) prior to study entry, for the duration of study participation and 28 days after completion of Tovorafenib administration, whichever is later. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. * A legal parent/guardian or participants must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate. * Ability to complete the PedsQL Core Module. * Patients must enroll on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution. Exclusion Criteria: Newly Diagnosed Participants: \- Participants should not have undergone any previous tumor-directed therapy. Recurrent Participants: * Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from acute adverse events due to agents administered more than 4 weeks earlier. * Participants must be at least 1 week since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 1 week after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs. * Participants should not have previously received any RAS-pathway, but have not received Tovorafenib will be eligible. All Participants: * Rapidly progressive symptoms that require urgent surgery or radiation therapy, which would prevent central review and or preclude participation with tumor-directed medical management alone. * Uncontrolled symptoms of neuroendocrine dysfunction such as diabetes insipidus, hypothyroidism, panhypopituitarism (participants can be on supplemental medications for hormonal repletion; however, should be on controlled doses for at least 2 weeks prior to enrollment). * Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to registration, ongoing cardiomyopathy, or current prolonged QT interval corrected for heart rate by Fridericia's formula (QTcF) interval \> 440 ms based on triplicate ECG average. * History of allergic reactions attributed to compounds of similar chemical or biologic composition to tovorafenib. * Nausea and vomiting \>= Grade 2, malabsorption requiring supplementation, or significant bowel or stomach resection that would preclude adequate absorption. * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection. * Participants who are receiving any other investigational agents. * Women of childbearing potential must not be pregnant or breast-feeding. * Current treatment with a strong cytochrome P4502C8(CYP2C8) inhibitor or inducer other than those allowed per Section 5.6.1. Medications that are substrates of CYP2C8 are allowed but should be used with caution. * Participants with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    20 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Children's Cancer Centre, Monash Children's Hospital

    RECRUITING

    Clayton, VIC 3168, Australia

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19103, United States

  • Children's Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55404, United States

  • Dana-Farber/Boston Children's Harvard Medical School

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • John Hopkins University

    RECRUITING

    Baltimore, Maryland, 21218, United States

  • John Hunter Children's Hospital

    RECRUITING

    New Lambton Heights, New South Wales, 2310, Australia

  • NYU Langone Health

    RECRUITING

    New York, New York, 10016, United States

  • Perth Children's Hospital

    RECRUITING

    Nedlands, Western Australia, 6009, Australia

  • Rady Children's Hospital/University of California, San Diego

    RECRUITING

    San Diego, California, 92037, United States

  • Riley Hospital for Children at Indiana University Health

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Royal Children's Hospital, Childrens' Cancer Centre

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • Royal Hobart Hospital

    RECRUITING

    Hobart, Tasmania, 7000, Australia

  • St. Louis Children's Hospital Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35294, United States

  • University of California, San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Email: •••••@•••••

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32611, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

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