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MEKTOVI® for Pediatric Adamantinomatous Craniopharyngioma

This study is testing an oral medication called Binimetinib (brand name MEKTOVI®) for children and young adults aged 1 to 39 years with adamantinomatous craniopharyngioma (ACP), a type of brain tumor. ACP often requires surgery and radiation, which can be challenging. Researchers believe Binimetinib, which targets specific pathways (ERK, MEK) involved in tumor growth, might help. The study aims to see if Binimetinib can lead to a sustained positive response in patients, including those who have had previous radiation or surgery. Up to 38 patients will receive Binimetinib twice a day for cycles lasting 28 days, potentially for up to two years. The study status is currently unclear, and one group of patients (those without prior radiation) is not currently enrolling.

Study design
This is a Phase 2 interventional study planning to enroll up to 38 participants. It has two groups based on whether patients have received prior radiation.
What's involved
Participants will take Binimetinib orally twice a day for 4 weeks per cycle. Treatment may continue for up to two years (26 cycles).
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed from the first day of treatment through 30 days after their last study treatment.

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NCT05286788

MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Recruiting
PHASE2Ages 1–39InterventionalTreatment
Nationwide Children's Hospital
~38 participants
Updated 2026-04-08 on ClinicalTrials.gov
What's tested:Binimetinib Oral Tablet [Mektovi]

At a glance

Recruiting sites
10 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with oral binimetinib
Measured over From Day 1 of treatment through 30 days following end of protocol treatment
+1 more outcome measured

Conditions

Adamantinous Craniopharyngioma
Recurrent Adamantinomatous Craniopharyngioma

Explore related trials

Adamantinomatous Craniopharyngioma

NCT05286788

Where you'd take part

This study runs at 10 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • British Columbia Children's Hospital

    Vancouver, British Columbia, Canadastudy coordinator listed

    Recruiting

  • Children's Hospital Colorado

    Aurora, Coloradostudy coordinator listed

    Recruiting

  • Children's National Medical Center

    Washington D.C., District of Columbiastudy coordinator listed

    Recruiting

  • CHU Sainte-Justine

    Montreal, Quebec, Canadastudy coordinator listed

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohiostudy coordinator listed

    Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohiostudy coordinator listed

    Recruiting

  • Nicklaus Children's Hospital

    Miami, Floridastudy coordinator listed

    Recruiting

  • Perth Children's Hospital

    Perth, Western Australia, Australiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Holly Lindsay, MD · STUDY_CHAIR · Children's Hospital Colorado
  • Todd C Hankinson, MD · STUDY_CHAIR · Children's Hospital Colorado
  • Maryam Fouladi, MD · PRINCIPAL_INVESTIGATOR · Nationwide Children's Hospital

Who to contact

Kelsey H Troyer, PhD
[email protected]
16147223284
Email the study team

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Eligibility criteria

Inclusion

Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and/or solid recurrence or progression at least 6 months post completion of radiation therapy
Stratum 2 (NOT CURRENTLY ENROLLING): Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required. 4. Performance Level: Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients 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. 5. Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments
Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair
Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines.
Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody.
Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation \> 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted.
Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment
Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy.
Surgery: At least 6 weeks must have elapsed since major or intermediate surgery. Major surgery includes major craniotomy for tumor resection of cyst fenestration, organ resection, and exploratory laparotomy. Intermediate procedures include ventriculoperitoneal shunt placement, stereotactic brain biopsy, and intraventricular catheter placement. Minor procedures that are not excluded include skin biopsy/incision and drainage, bone marrow aspirate, and central venous catheter placement, ommaya aspirations, lumbar punctures, and nasal endoscopy to remove packing. 6. Organ Function Requirements
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)
Hemoglobin \>8 g/dL (may be transfused)
Creatinine clearance or radioisotope GFR \> 70ml/min/1.73 m2 or
A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender as follows:
Total bilirubin ≤ 1.5 × institutional upper limit of normal
AST (SGOT) ≤ 2.5 × institutional upper limit of normal
ALT (SGPT) ≤ 2.5 × institutional upper limit of normal
Left Ventricular Ejection Fraction greater than the institutional lower limit of normal by echocardiogram
QTc ≤ 480 msec (by Bazett formula)
Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies. 7. Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion

Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation
Patients who are unable to absorb enteral medications
Administration via NG/NJ/G-tube is allowed 3. Concomitant Medications
Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.
Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible. 4. Study Specific:
Patients who have an uncontrolled infection are not eligible.
Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible.
Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system
Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible.
Patients who have received a prior solid organ transplantation are not eligible.
Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.
Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening.
Patients who have had surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing.
Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.

What this trial measures

  • Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with oral binimetinibFrom Day 1 of treatment through 30 days following end of protocol treatment

    To calculate the number of patients who experience sustained objective response rate \[minor response (MR) + partial response (PR) + complete response (CR)\] of patients with recurrent/progressive previously irradiated Adamantinomatous Craniopharyngioma to treatment with oral binimetinib (Stratum 1).

  • Sustained objective response rate of patients with measurable ACP who have undergone surgery but have not been previously treated with radiation to treatment with oral binimetinibFrom Day 1 of treatment through 30 days following end of protocol treatment

    To calculate the number of patients who experience sustained objective response rate (MR + PR + CR) of patients with measurable Adamantinomatous Craniopharyngioma who have undergone surgery but have not been previously treated with radiation to treatment with oral binimetinib (Stratum 2).