NCT05286788

MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Recruiting now Phase 2 Last updated 8 April 2026

What this trial tests

Phase 2 trial testing Binimetinib Oral Tablet [Mektovi] in Adamantinous Craniopharyngioma in 38 participants. Currently enrolling.

Timeline

10 April 2023

Primary endpoint
10 April 2027

10 April 2027

Quick facts

Lead sponsor	Nationwide Children's Hospital
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	38
Start date	10 April 2023
Primary completion	10 April 2027
Estimated completion	10 April 2027
Sites	10 locations across Canada, United States, Australia

Drugs / interventions tested

• Binimetinib Oral Tablet [Mektovi] — full drug profile →

Conditions studied

• Adamantinous Craniopharyngioma — all drugs for Adamantinous Craniopharyngioma →
• Recurrent Adamantinomatous Craniopharyngioma — all drugs for Recurrent Adamantinomatous Craniopharyngioma →

Sponsor

Nationwide Children's Hospital

Who can join

Adults 1 to 39, any sex, with Adamantinous Craniopharyngioma or Recurrent Adamantinomatous Craniopharyngioma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2. The biological activity of binimetinib that has been evaluated bith in vitro and in vivo in a wide variety of tumor types In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. BRAF-mediated brain tumors in adults and children: A review and the Australian and New Zealand experience.
   Trinder SM, McKay C, Power P, Topp M, et al · · 2023 · cited 12× · PMID 37124503 · DOI 10.3389/fonc.2023.1154246
2. Recurrent adamantinomatous craniopharyngiomas show MAPK pathway activation, clonal evolution and rare TP53-loss-mediated malignant progression.
   Apps JR, Gonzalez-Meljem JM, Guiho R, Pickles JC, et al · · 2024 · cited 8× · PMID 39127699 · DOI 10.1186/s40478-024-01838-4
3. Pituitary Tumorigenesis-Implications for Management.
   Vamvoukaki R, Chrysoulaki M, Betsi G, Xekouki P. · · 2023 · cited 7× · PMID 37109772 · DOI 10.3390/medicina59040812
4. Current clinical trials for craniopharyngiomas: what's on the horizon?
   Joshi N, Mueller S, Kline C. · · 2025 · cited 4× · PMID 40042714 · DOI 10.1007/s11060-024-04899-6
5. Targeted therapy in pediatric central nervous system tumors: a review from the National Pediatric Cancer Foundation.
   Siegel BI, Patil P, Prakash A, Klawinski DM, et al · · 2025 · cited 3× · PMID 40094009 · DOI 10.3389/fonc.2025.1504803
6. BMI trajectories in the first 3 months after childhood craniopharyngioma resection: a plea for early management of BMI changes.
   Hulsmann SC, Hoving EW, Bakker B, Janssens GO, et al · · 2025 · cited 3× · PMID 39952232 · DOI 10.1530/ec-24-0533
7. Molecular subtypes of adamantinomatous craniopharyngiomas.
   An W, Li S, An Y, Lin Z. · · 2025 · cited 3× · PMID 39898434 · DOI 10.1093/neuonc/noaf030
8. A Patient-Derived Organoid Biobank of Adamantinomatous Craniopharyngioma as a Platform for Drug Discovery.
   Zhang H, Wang C, Fan J, Chen Z, et al · · 2026 · cited 1× · PMID 41250940 · DOI 10.1002/advs.202503924