Avutometinib for Solid Tumor Cancers in Children and Young Adults
This study is testing a drug called avutometinib for children and young adults (ages 3 to 30) who have advanced or recurrent solid tumor cancers. Researchers want to find the safest dose of avutometinib that causes the fewest or mildest side effects. Participants will receive avutometinib by mouth twice a week, three weeks on and one week off, in cycles lasting 28 days. The study will measure the safety of avutometinib for up to 12 months. This study is currently looking for a small number of participants, specifically 3 people.
- Study design
- This is an interventional study with a planned enrollment of 3 participants. It is not specified if it is randomized or blinded.
- What's involved
- Participants will take avutometinib by mouth once daily twice a week, three weeks on/one week off, for cycles of 28 days.
- Compensation
- Not stated in the trial record.
- Follow-up
- The safety of avutometinib will be measured for up to 12 months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT06104488
A Study of Avutometinib for People With Solid Tumor Cancers
Active, Not Recruiting
PHASE1Ages 3–30InterventionalTreatmentMemorial Sloan Kettering Cancer CenterInvestigator-initiated
~3 participants
Updated 2026-06-15 on ClinicalTrials.gov
What's tested:Avutometinib
At a glance
Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety of avutometinib
Measured over up to 12 months
Conditions
Where it's being run
2 sites across 2 statesGeorgia1
New York1
Study leadership
- Sameer Farouk Sait, MD · PRINCIPAL_INVESTIGATOR · Memorial Sloan Kettering Cancer Center
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age ≥ 3 year and ≤ 30 years at the time of informed consent. \*Patients over 18 years of age will be treated at the adult RP2D. The accrual for patients \>18 and ≤ 30 years will be limited to no more than 5 patients overall and will not participate in the dose escalation.
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.
Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age.
Participants must have one of the following:
Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR - Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR \[20/80, 6/24, or 2.5/10\] or more in one or both eyes).
≥ 20% increase in volume of enhancing tumor
≥ 2mm increase in greatest linear dimension of enhancing tumor Participants with diagnosis of NF2-SWN may enroll without tissue/biopsy confirmation. Genetic variants are classified as benign (B), likely benign (LB), likely pathogenic (LP), pathogenic (P), or variant of uncertain clinical significance (VUS) according to the standards and guidelines developed by the American College of Medical Genetics and Genomics, the Association for Molecular Pathology, and the College of American Pathologists \[62\]. Only patients with pathogenic and likely pathogenic variants in NF2 will be permitted to enroll \[61\].
Tissue-based or liquid biopsy NGS or quantitative polymerase chain reaction (qPCR) or RNA based fusion detection (ARCHER or other similar platform).
Fluorescence in situ hybridization (FISH)
For subjects enrolled by a liquid biopsy test; blood samples will be required and should be sent prior to enrollment and used for retrospective confirmation in the Sponsor's designated central laboratory (MSKCC).
Patients must meet the following disease status criteria:
Solid tumor: Patients must have either measurable disease as measured by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (Version 1.1) or evaluable disease.
Neuroblastoma subjects are permitted to have evaluable disease only (e.g., bone disease only, evaluable by MIBG or PET).
Primary Brain Tumors: Patients with primary brain tumors are eligible and can either have measurable disease (defined as at least equal or greater than twice the slice thickness in two perpendicular diameters on MRI) OR evaluable disease (clear MRI evidence of disease that may not be measurable in two perpendicular diameters) OR diffuse leptomeningeal disease OR positive CSF cytology alone.
NF2 related schwannoma or meningioma: At least one volumetrically measurable and ≥1 cc NF2-SWN related VS or meningioma (histological confirmation not required), designated as target tumor.
Tumor is refractory or recurrent/progressive after standard therapy (at least one prior standard therapy appropriate for tumor type and stage of disease) unless available standard therapies are considered inadequate for the patient.
Patients must have a body surface area (BSA) ≥ 0.8 m2
Patients must be able to swallow intact capsules.
Patients may have received prior treatment with a RAF inhibitor (1st or 2nd generation) or MEK inhibitor but only as monotherapy (regardless of prior response to therapy).
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and meet minimum durations (shown below) from prior therapy.
Adequate hepatic function (within 28 days prior to C1D1), defined as:
Adequate renal function (within 28 days prior to C1D1) defined as a maximum serum creatinine for age and gender defined below. Patients that do not meet the criteria in Table 3 but have a 24 hour Creatinine Clearance or absolute GFR (radioisotope or iothalamate) ≥ 85ml/min are eligible.
Adequate hematologic function (within 7 days prior to C1D1), defined as:
Creatine phosphokinase (CPK) ≤ 2.5 x ULN.
Adequate recovery from toxicities related to prior treatments to at least Grade 1 by CTCAE v 5.0. Exceptions include alopecia and peripheral neuropathy grade ≤ 2.
Males or females of reproductive potential must agree to use an effective method of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control until one month after the last dose for female patients and 3 months after the last dose for male pts. Male patients should follow the same direction for sperm donation.
Exclusion
History of rhabdomyolysis.
Concurrent ocular disorders:
Patients with history of glaucoma, history of retinal vein occlusion (RVO), predisposing factors for RVO, including uncontrolled hypertension, uncontrolled diabetes.
Patients with history of retinal pathology or evidence of visible retinal pathology that is considered a risk factor for RVO, intraocular pressure \> 21 mm Hg as measured by tonometry, or other significant ocular pathology, such as anatomical abnormalities that increase the risk for RVO.
Patients with a history of corneal erosion (instability of corneal epithelium), corneal degeneration, active or recurrent keratitis, and other forms of serious ocular surface inflammatory conditions.
Patients with a history of hypersensitivity to any of the inactive ingredients (hydroxypropylmethylcellulose, mannitol, magnesium stearate) of the investigational product.
Ongoing active diarrhea requiring medication (e.g., loperamide, bile acid sequestrant such as cholestyramine) within 7 days.
Clinically significant cardiac disease or risk factors at screening including any of the following:
Known hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection that is active and/or requires therapy.
Exposure to strong CYP3A4 inhibitors and inducers within 14 days prior to the first dose and during the course of therapy (see appendix A).
Known strong and moderate inducers or inhibitors of CYP3A4/5, including enzyme-inducing anti-convulsant drugs (EIACDs), grapefruit, echinacea, grapefruit hybrids, pummelos, starfruit, and Seville oranges.
Substrates of CYP3A4/5 with a narrow therapeutic index.
Herbal preparations/medications (except for vitamins) including, but not limited to:
St. John's wort, Kava, ephedra (ma huang), gingko biloba, dehydroepiandrosterone
(DHEA), yohimbe, saw palmetto, black cohosh and ginseng.
Pregnant or breastfeeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities.
What this trial measures
- Safety of avutometinibup to 12 months
The primary objective is to assess the safety of avutometinib in the pediatric population. CTCAE Version 5 will be utilized for toxicity evaluation. Adverse Events should be recorded from treatment start through 30 days after the last dose of study drug.