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Safety and Feasibility of First-line Avutometinib and Defactinib in Patients With Newly Diagnosed High-grade Gliomas

This clinical trial is designed with two treatment stages: Stage 1: one cycle of study drug treatment prior to initiation of first-line standard radiation in all study participants. Stage 2: two additional cycles of study drug treatment (8 weeks total) concomitantly with standard of care radiation (~6 weeks) for participants with pathology-confirmed MGMT (enzyme O-6-methylguanine-DNA methyltransferase) unmethylated GBM only.

The co-primary endpoints of the study are 1) feasibility of completing Stage 1 of treatment prior to radiation in all study participants and starting radiation within 6 weeks, and 2) safety of study drug across both treatment parts. The investigators will additionally evaluate the radiographic response rate after the first cycle of drug in all patients based upon RANO 2.0 (Response Assessment in Neuro-Oncology) criteria as well as safety of study drug in all patients (secondary endpoints). Exploratory endpoints will include characterization of ERK (extracellular signal-regulated kinase) and FAK (Focal adhesion kinase) dependence in pre-treatment tissue (by immunohistochemistry and/or 'omics) and correlation of expression and co-mutations with response and survival.

A total of up to 22 evaluable patients can be enrolled in this study. Evaluable patients are those who have received at least one dose of study drug treatment. Patients who do not start drug will be replaced, up to a total of 28 patients.

Studieoversikt

Detaljert beskrivelse

Patients with a presumptive diagnosis of high-grade glioma based on frozen pathology and measurable (by RANO2.0 2D criteria) residual enhancing disease on post-operative MRI will be screened and consented within 2 weeks of surgery. All participants will start treatment within 2 weeks of surgery and receive study drug for one cycle at the FDA-approved dose (avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice a day; 3 wks on/1 wk off). Co-primary endpoint for all participants will be safety of the study intervention in this population and feasibility of receiving study drug and starting radiation therapy within 6 weeks of surgery.

After one cycle of drug, all patients whose pathology reveals MGMT unmethylated, IDH (enzyme isocitrate dehydrogenase 1)-wildtype Glioblastoma, WHO (World Health Organization) Grade 4 will remain on study drug treatment: they will receive 2 additional cycles of study drug (8 weeks total) and concurrently with standard-of-care radiation (6 weeks total). These participants will remain on study until their post-radiation MRI 4 weeks after completing radiation, or 30 days after last dose of study drug, whichever is later. Co-primary endpoint for this sub-population will be safety of study drug administration for 3 cycles before and during radiation therapy (as measured by CTCAE v6, TRAEs (Treatment Related Adverse Events).

Those who do not qualify for the radiation phase will be taken off treatment with study drug and receive standard-of-care therapy.

A safety assessment period will be conducted for the first 6 patients with MGMT unmethylated, IDH-wild type Glioblastoma, WHO grade 4. Enrollment will be paused until all 6 patients are through the safety assessment period, which is a total of 16 weeks from the start of Cycle 1 Day 1 (C1D1) until 4 weeks after last dose of study drug.

After treatment, all participants will come off treatment and be followed for progression, treatments, and survival.

Evaluable participants are those who receive at least one dose of Avutometinib and defactinib.

Studietype

Intervensjonell

Registrering (Antatt)

22

Fase

  • Tidlig fase 1

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

Inclusion Criteria:

  • Patient must be able to provide written informed consent.
  • ≥ 18 years of age
  • Likely high-grade glioma based on frozen pathology in patients with no prior diagnosis of a glioma or astrocytoma.
  • Patient appropriate for standard therapy for high-grade glioma that includes 6 weeks of radiation.
  • Patient is not deemed at a high risk of symptomatic progression within 4 weeks by the treating physician and investigator team.
  • Karnofsky Performance Scale (KPS) ≥ 70%.
  • Post-operative MRI completed with 72 hours of surgery that includes FLAIR sequences
  • Measurable disease per RANO 2.0 enhancing criteria. Leptomeningeal disease not allowed.
  • Patient is on a tapering dose of steroids anticipated to be on a total daily dose of dexamethasone < 2 mg for at least 5 days by start of study intervention.
  • Willing to submit archival tumor sample if available
  • Adequate organ and marrow function defined as:
  • Ability to swallow and retain orally administered medications (no liquid suspensions available).
  • Female participants of childbearing potential must have a negative serum pregnancy test prior to study start.
  • Male participants must also be documented to be surgically sterile or agree to use adequate contraception and not to donate sperm
  • Patients must have no concurrent malignancy except curatively treated basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix, breast, or bladder. Patients with other malignancies must be disease-free for > 2 years.
  • Patients with human immunodeficiency virus (HIV) who are on effective antiretroviral therapy are eligible if the viral load was assessed as undetectable within 6 months prior to baseline.

Exclusion Criteria:

  • Any prior glioma or astrocytoma, or any prior cancer diagnosis resulting in irradiation of the skull or scalp
  • Significant bi-hemispheric disease or midline shift observed on post-operative MRI likely to result in rapid clinical deterioration
  • Those who have undergone diagnostic biopsy only for suspected primary brain cancer.
  • Those who are on other investigational agents at the time of screening and including patients on carmustine and/or gamma tiles
  • History of clinically significant ophthalmologic disorders
  • Impairment in gastrointestinal function or disease that may significantly alter the absorption of oral study drug
  • Clinically significant cardiovascular disease
  • History of recent (≤ 90 days) thromboembolic or cerebrovascular event
  • Known history of any positive test for hepatitis B virus or hepatitis C virus indicating acute or chronic infection, and/or detectable virus.
  • Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection requiring antibiotics or psychiatric illness/social situations that would limit compliance with study requirements, are ineligible.
  • Patients may not take any of the listed contraindicated medications for 7 days prior to the start of study drug, with the exception of dexamethasone; see section 5.6 "concomitant therapy":

    1. Warfarin
    2. Strong CYP2C9 or CYP3A4 inhibitors or inducers
    3. P-glycoprotein inhibitors or inducers
  • Patients may not be on H2 blocker or PPI (Proton pump inhibitors) for 2 days prior to start of study drug.
  • Subjects with a history of hypersensitivity to any of the active or inactive ingredients (hydroxypropylmethylcellulose, mannitol, magnesium stearate) of the investigational products.
  • Pregnant or breastfeeding
  • Active or past medical history of interstitial lung disease (ILD)/pneumonitis, including drug-induced or radiation ILD/pneumonitis, pulmonary fibrosis, or adult respiratory distress syndrome (ARDS
  • History of medically significant rhabdomyolysis

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Sekvensiell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Stage 1: Presumed GBM 1 cycle prior to radiation
1 cycle of avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice a day) for 3 weeks on/1 week off during the pre-radiation window (one cycle = 4 weeks total)
avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice a day for 3 weeks on/1 week off (one cycle = 4 weeks total)
Andre navn:
  • pre-radiation
Eksperimentell: Stage 2: MGMT-unmethylated GBM with Radiation
2 additional cycles of 3.2 mg orally twice weekly and defactinib 200 mg orally twice a day for 3 weeks on/1 week off during radiation (one cycle = 4 weeks total)
Patients whose final pathology is MGMT promoter unmethylated GBM will receive an additional 2 cycles of avutometinib/defactinib concurrently with radiation (without temozolomide).
Andre navn:
  • with radiation

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Proportion of evaluable patients who complete at least 75% of the oral study drug intervention
Tidsramme: 4 weeks
Feasibility of a pre-radiation (pre-RT) therapeutic treatment
4 weeks
Safety as assessed by Number of patients who experience grade 3 and Grade 4 adverse events
Tidsramme: From start of treatment (day 0) up to 16 weeks post start of treatment
Using CTCAE v6.0, toxicity will be assessed starting with C1D1 and continue until 4 weeks after the last dose of study drug (evaluated 16 weeks from start of treatment; total study period). Number of patients who experience grade 3 and Grade 4 events based on the 6.0 CTCAE Assessment tool.
From start of treatment (day 0) up to 16 weeks post start of treatment

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Radiographic response rate
Tidsramme: 4 weeks
Participants with Radiographic response following 1 cycle (4weeks) of study drug by bi-dimensional enhancing criteria determined by RANO 2.0
4 weeks

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Samarbeidspartnere

Etterforskere

  • Studiestol: Karisa C Schreck, MD, PhD, Johns Hopkins University

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

10. november 2026

Primær fullføring (Antatt)

10. november 2028

Studiet fullført (Antatt)

23. desember 2029

Datoer for studieregistrering

Først innsendt

18. august 2026

Først innsendt som oppfylte QC-kriteriene

20. august 2026

Først lagt ut (Faktiske)

21. august 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

26. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

25. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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