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Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults

(PNOC029)

Sponsored by Sabine Mueller, MD, PhD

Quick Self-Assessment

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Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Feb 27, 2026

Phase

Clinical Trial

Sponsor

Sabine Mueller, MD, PhD

Enrollment Target

57

Start Date

Sep 2022

๐Ÿ“Š
1 of 57 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

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 candidate

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

57 participants

๐ŸŽ‚

Age Range

1 Year - 39 Years

โšง

Gender

ALL

๐Ÿข

Sponsor

Sabine Mueller, MD, PhD

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Birmingham

Alabama

Location available
View Birmingham location page
RECRUITING

San Diego

California

Location available
View San Diego location page
RECRUITING

San Francisco

California

Location available
View San Francisco location page
RECRUITING

Gainesville

Florida

Location available
View Gainesville location page
RECRUITING

Chicago

Illinois

Location available
View Chicago location page
RECRUITING

Indianapolis

Indiana

Location available
View Indianapolis location page
RECRUITING

Baltimore

Maryland

Location available
View Baltimore location page
RECRUITING

Boston

Massachusetts

Location available
View Boston location page
RECRUITING

Ann Arbor

Michigan

Location available
View Ann Arbor location page

๐Ÿ“ And 6 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

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Looking for Craniopharyngioma Treatment in Birmingham?

Join others in Alabama exploring innovative treatment options through clinical research

Craniopharyngioma Treatment Options in Birmingham, Alabama

If you're searching for Craniopharyngioma treatment in Birmingham, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Birmingham, San Diego, San Francisco and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Craniopharyngioma. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Alabama
Now Enrolling
Up to 57 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Craniopharyngioma?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Craniopharyngioma

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Craniopharyngioma Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05465174. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.