Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults

Part of paid clinical trials in Birmingham, Alabama.

Sponsor: Sabine Mueller, MD, PhD
Study ID: NCT05465174
Phase: PHASE2
Status: Recruiting

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Conditions

Craniopharyngioma
Craniopharyngioma, Child
Recurrent Craniopharyngioma

Eligibility Criteria

Sex: ALL
Age: 1 Year - 39 Years
Healthy Volunteers: Not accepted

Interventions

Tovorafenib — DRUG
Given orally

Study Details

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.

Key Dates

Start date: Sep 12, 2022
Status verified: Apr 2026
Primary completion: Mar 1, 2027
Completion: Mar 1, 2028

Study Design

Enrollment: 57 participants (estimated)
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Arms

Experimental: Group 1: Neoadjuvant Tovorafenib
Participants with newly diagnosed craniopharyngioma will receive one (1) dose of Tovorafenib within 7 days +/- 2 days prior to planned biopsy or resection. At completion of biopsy or resection, participants having undergone biopsy only or sub-total (STR) or near-total resection (NTR) will continue on maintenance monotherapy of Tovorafenib given once weekly at the respected recommended phase 2 dose (RP2D). Participants having undergone a gross total resection (GTR) will enter into follow-up only and will be part of the exploratory cohort.
Experimental: Group 2, Arm A: Neoadjuvant Tovorafenib
Participants with recurrent craniopharyngioma will receive one (1) dose of Tovorafenib within 7 days +/- 2 days prior to planned biopsy or resection. At completion of biopsy or resection, participants having undergone biopsy only or STR or NTR will continue on maintenance monotherapy of Tovorafenib given once weekly at the respected RP2D.
Experimental: Group 2, Arm B: Non-biopsy/resection participants
Non-biopsy/resection participants with recurrent disease will receive monotherapy of Tovorafenib given once weekly at the respected RP2D.

Primary Outcome Measure

Progression free survival rate (PFS) [ Time Frame: Up to 12 months ]

Central Contacts

PNOC Operations Office
877-827-3222
[email protected]

Locations (15)

Facility	City	State	ZIP	Site coordinators
University of Alabama at Birmingham	Birmingham	Alabama	35294	Girish Dhall, MD [email protected] 205-638-9285 Laura K Metrock, MD [email protected] 205 638 9285 Girish Dhall, MD (PRINCIPAL_INVESTIGATOR)
Rady Children's Hospital/University of California, San Diego	San Diego	California	92037	Lanipua Yeh-Nayre [email protected] 520-241-1266 Jennifer Elster, MD (PRINCIPAL_INVESTIGATOR)
University of California, San Francisco	San Francisco	California	94143	PNOC Operations Office [email protected] [email protected] 877-827-3222 Sabine Mueller, MD, PhD, MAS (PRINCIPAL_INVESTIGATOR)
University of Florida	Gainesville	Florida	32611	Elias Sayour, MD, PhD [email protected] 352-294-8347 Elias Sayour, MD, PhD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	60611	Alex Newhouse [email protected] 312-227-4873 Ashley Plant-Fox, MD (PRINCIPAL_INVESTIGATOR)
Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana	46202	Scott Coven [email protected] 317-944-8784 Scott Coven, MD (PRINCIPAL_INVESTIGATOR)
John Hopkins University	Baltimore	Maryland	21218	Kenneth Cohen, MD, MBA [email protected] 410-614-5055 Robyn Gartrell, MD [email protected] (410) 614-5055 Kenneth Cohen, MD, MBA (PRINCIPAL_INVESTIGATOR)
Dana-Farber/Boston Children's Harvard Medical School	Boston	Massachusetts	02215	Kelliie Edwards [email protected] Susan Chi, MD (PRINCIPAL_INVESTIGATOR)
University of Michigan	Ann Arbor	Michigan	48109	Andrea Franson, MD [email protected] 734-232-9335 Carl Koschmann, MD [email protected] 734-232-9335 Andrea Franson, MD (PRINCIPAL_INVESTIGATOR)
Children's Minnesota	Minneapolis	Minnesota	55404	Anne Bendel, MD [email protected] 612-626-2778 Maggie Skrypek, MD [email protected] 612-813-5940 Anne Bendel, MD (PRINCIPAL_INVESTIGATOR) Maggie Skrypek, MD (PRINCIPAL_INVESTIGATOR)
St. Louis Children's Hospital Washington University	St Louis	Missouri	63110	Lisa Murray [email protected] 314-286-2790 Mohamed Shebl Abdelbaki, MD (PRINCIPAL_INVESTIGATOR)
NYU Langone Health	New York	New York	10016	Anna Yaffe [email protected] 212-263-9945 Jessica Clymer, MD (PRINCIPAL_INVESTIGATOR)
Duke University Medical Center	Durham	North Carolina	27710	Daniel Landi, MD [email protected] Daniel Landi, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	19103	Cassie Kline, MD, MAS 267-426-5026 Cassie Kline, MD, MAS (PRINCIPAL_INVESTIGATOR)
University of Utah	Salt Lake City	Utah	84112	Nicholas Whipple, MD, MPH [email protected] 801-662-4700 Priya Chan, MD [email protected] (801) 662-4700 Nicholas Whipple, MD, MPH (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By research site

University of Alabama at Birmingham· Birmingham, AL Rady Children's Hospital/University of California, San Diego· San Diego, CA University of California, San Francisco· San Francisco, CA University of Florida· Gainesville, FL Ann & Robert H. Lurie Children's Hospital of Chicago· Chicago, IL Riley Hospital for Children at Indiana University Health· Indianapolis, IN

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