Refractory CancerPhase 1NCT06104488
A Study of Avutometinib for People With Solid Tumor Cancers
A Phase 1 study testing Avutometinib in people with Refractory Cancer and CNS Tumors, run by Memorial Sloan Kettering Cancer Center at 2 sites in United States.
Trial summary
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects. Read more about this study
- Who is it for?
- Ages 3 to 30 years
- Conditions the study is for
- Refractory Cancer, CNS Tumors, CNS Tumor, Adult
- What is being tested?
- Avutometinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesUnited States
- Study dates
- 20 Oct 2023to 3 Nov 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must meet all of the following inclusion criteria to be eligible to enroll in this study:
- 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:
- Histologically confirmed diagnosis of a pediatric tumor including CNS tumors with activating MAP kinase pathway alterations including but not limited to BRAF/ARAF/CRAF fusions or mutations, KRAS/NRAS/HRAS alterations, PTPN11 or SOS1/2 mutations and/or loss of function alterations in NF1. This will be performed at the enrolling institution and central review is not required OR
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- Participants with a clinical or molecularly confirmed (germline alteration positive) diagnosis of NF1 with symptomatic inoperable plexiform neurofibromas and recurrent/progressive low-grade gliomas are eligible and may enroll without tissue/biopsy confirmation.
- OR
- Patients with recurrent optic pathway gliomas are eligible if they have clinical progression (defined as new or worsening neurologic symptoms including visual dysfunction, as defined below):
- 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).
- OR d) Participants with a confirmed diagnosis of neurofibromatosis 2 schwannomatosis (NF2-SWN) based on revised consensus 2021 criteria [61] with a target NF2-related tumor (vestibular schwannoma, non-vestibular schwannoma, meningioma) with documented radiographic progression defined as either:
- ≥ 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].
- Molecular testing requirements: Genetic alterations (SNVs or fusions) may be identified through local testing in a Clinical Laboratory Improvement Amendments (CLIA) laboratory in the US or equivalently accredited diagnostic lab outside the United States (US) (CLIA certified) by using molecular assays on any tumor samples (either at initial diagnosis or recurrence). Only the following test modalities are permitted:
- Tissue-based or liquid biopsy NGS or quantitative polymerase chain reaction (qPCR) or RNA based fusion detection (ARCHER or other similar platform).
- The reports should be collected for any participants who have completed Next Generation Sequencing (NGS) at any point prior to or during study participation
- 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.
- Exception: NF1 patients with symptomatic and inoperable plexiform neurofibromas that are radiographically progressive or causing significant cosmetic disfigurement or causing significant morbidity are eligible regardless of prior MEK inhibitor exposure. NF2 patients with VS, non VS and meningioma are eligible prior to recurrence since no standard upfront treatment options exist.
- 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.
- Anti-cancer agents not known to be myelosuppressive: ≥ 7 days
- Anti-cancer and cytotoxic agents known to be myelosuppressive: ≥ 14 days
- Immunotherapies (including antibodies, interleukins, interferons, etc.): ≥ 21 days
- Adoptive cellular therapies (including modified T cells, vaccines, etc.): ≥ 42 days
- Autologous stem cell infusion (boost, no conditioning): ≥ 21 days
- Autologous stem cell transplantation (with conditioning): ≥ 42 days
- Allogeneic bone marrow transplantation: ≥ 84 days
- Focal external beam radiation (e.g., limited sites of disease): ≥ 14 days
- Substantial external beam radiation (e.g. whole lung or abdomen): ≥ 42 days
- Radiopharmaceutical therapy (e.g., radiolabeled antibody or MIBG): ≥ 42 days
- Adequate hepatic function (within 28 days prior to C1D1), defined as:
- total bilirubin ≤ 1.5 × upper limit of normal (ULN) for the institution; patients with Gilbert syndrome may enroll if total bilirubin < 3.0 mg/dL (51 μmole/L);
- alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN (or < 5x ULN in patients with liver metastases)
- Albumin ≥ 3.0 g/dL (451 μmole/L).
- 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.
- Age 1 month to < 6 months: Maximum Serum Creatinine (mg/dL), Male: 0.4; Female: 0.4 Age 6 months to < 1 year: Maximum Serum Creatinine (mg/dL), Male: 0.5; Female: 0.5 Age 1 to < 2 years: Maximum Serum Creatinine (mg/dL), Male: 0.6; Female: 0.6 Age 2 to < 6 years: Maximum Serum Creatinine (mg/dL), Male: 0.8; Female: 0.8 Age 6 to < 10 years: Maximum Serum Creatinine (mg/dL), Male: 1; Female: 1 Age 10 to < 13 years: Maximum Serum Creatinine (mg/dL), Male: 1.2; Female: 1.2 Age 13 to < 16 years: Maximum Serum Creatinine (mg/dL), Male: 1.5; Female: 1.4
- ≥ 16 years: Maximum Serum Creatinine (mg/dL), Male: 1.7; Female: 1.4 Table 3. The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.
- Adequate hematologic function (within 7 days prior to C1D1), defined as:
- platelets ≥100,000/mm3; and
- absolute neutrophil count (ANC) ≥ 1000/mm3. Note: Patients may not receive platelet transfusions nor hematopoietic growth factor support, including granulocyte-colony stimulating factor (e.g. filgrastim) and platelet stimulators (e.g. romiplostim) for at least 7 days prior to demonstrating adequate hematologic function.Patients with known malignant bone marrow infiltration are exempt from the above count requirements but should be discussed with sponsor.
- 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.
Who cannot join
- Patients meeting any of the following exclusion criteria are not eligible to enroll in this study:
- 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.
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- 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:
- Any history of congestive heart failure
- Left ventricular ejection fraction (LVEF) < 50% or below the institutional standard lower limit, whichever is higher, as determined by multiple gated acquisition (MUGA) scan or Trans-thoracic echocardiography (TTE)
- QTc > 470 msec regardless of sex (using Bazett formula) on screening ECG (using triplicate ECGs), history of Torsades de Pointes, or a history of congenital long QT syndrome.
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Healthcare of Atlanta (Data Collection Only)
Atlanta, Georgia, 30322
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, 10065
About this study
Official title: \Multi-Center Phase I Study of Avutometinib (VS-6766), a RAF/MEK Clamp in Combination With Defactinib, a FAK Inhibitor, in Pediatric Patients With Refractory or Recurrent Solid Tumors Harboring Activating MAPK Pathway or NF2 Alterations
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects.
- Conditions
- Refractory Cancer, CNS Tumors, CNS Tumor, Adult, CNS Tumor, Childhood, MAP Kinase Family Gene Mutation, NF1
- Being tested
- Avutometinib
- Keywords
- Refractory Cancer, Central nervous system tumor, CNS tumor, Plexiform Neurofibroma, Low-grade Glioma, Optic Pathway Gliomas, Neuroblastoma, Primary Brain Tumor, Solid Tumor, Solid Carcinoma, Avutometinib, Memorial Sloan Kettering Cancer Center, 23-129
- Medical subject headings
- Neoplasms, Central Nervous System Neoplasms, Neurofibroma, Plexiform, Neuroblastoma, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvutometinibDrug
Participants will receive oral avutometinib once daily twice a week, three weeks on/ 1week off, for a period of 28 days per cycle
| Study group | Role | What they receive |
|---|---|---|
| Dose Level -1 | Experimental | If 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. Receives: Drug: Avutometinib |
| Dose Level 1 | Experimental | If 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. Receives: Drug: Avutometinib |
| Dose Level 2 | Experimental | If 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. Receives: Drug: Avutometinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of avutometinib
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.
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2023
- Study start20 Oct 2023
- Primary completion3 Nov 2026
- Study completion3 Nov 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory CancerStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sameer Farouk Sait, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06104488
- Sponsor's protocol ID
- 23-129
- Status last verified
- Nov 2026
- First submitted
- 23 Oct 2023
- Last updated
- 4 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- \Multi-Center Phase I Study of Avutometinib (VS-6766), a RAF/MEK Clamp in Combination With Defactinib, a FAK Inhibitor, in Pediatric Patients With Refractory or Recurrent Solid Tumors Harboring Activating MAPK Pathway or NF2 Alterations
- ClinicalTrials.gov ID
- NCT06104488
- Acronym / sponsor ID
- 23-129
- Conditions
- Refractory Cancer, CNS Tumors, CNS Tumor, Adult, CNS Tumor, Childhood, MAP Kinase Family Gene Mutation, NF1
- Keywords
- Refractory Cancer, Central nervous system tumor, CNS tumor, Plexiform Neurofibroma, Low-grade Glioma, Optic Pathway Gliomas, Neuroblastoma, Primary Brain Tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- 20 Oct 2023 (actual)
- Primary completion
- 3 Nov 2026 (actual)
- Study completion
- 3 Nov 2026 (actual)
- First posted
- 27 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Level -1 | Experimental | Drug: AvutometinibIf 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. |
| Dose Level 1 | Experimental | Drug: AvutometinibIf 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. |
| Dose Level 2 | Experimental | Drug: AvutometinibIf 0 of 3-6 or no more than 1 of 6 evaluable participants experience a DLT, then the dose will be escalated to Dose Level 2 and the same approach will be repeated. If 2 or more DLTs are observed on Dose Level 1 in 2-6 subjects, then further enrollment to Dose Level 1 will stop, the dose will be de-escalated to Dose Level -1 and the same approach will be repeated. The MTD will be the highest dose with <2 DLTs in 6 patients. |
Who may qualify
- Ages
- 3 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (52 listed)
- Patients must meet all of the following inclusion criteria to be eligible to enroll in this study:
- 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:
Key exclusion criteria (20 listed)
- Patients meeting any of the following exclusion criteria are not eligible to enroll in this study:
- History of rhabdomyolysis.
- Concurrent ocular disorders:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvutometinibParticipants will receive oral avutometinib once daily twice a week, three weeks on/ 1week off, for a period of 28 days per cycle | Drug | Dose Level -1, Dose Level 1, Dose Level 2 |
Primary outcome
Safety of avutometinib
Time frame: up 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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sameer Farouk Sait, MD principal investigator
Memorial Sloan Kettering Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Avutometinib in people with Refractory Cancer and CNS Tumors, run by Memorial Sloan Kettering Cancer Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 52 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of avutometinib (time frame: up to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.