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

25 sierpnia 2026 zaktualizowane przez: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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.

Przegląd badań

Szczegółowy opis

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.

Typ studiów

Interwencyjne

Zapisy (Szacowany)

22

Faza

  • Wczesna faza 1

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

  • Nazwa: Michaella Iacoboni, RN
  • Numer telefonu: 410-955-8837
  • E-mail: msheeh13@jhmi.edu

Lokalizacje studiów

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dorosły
  • Starszy dorosły

Akceptuje zdrowych ochotników

Nie

Opis

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

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nielosowe
  • Model interwencyjny: Zadanie sekwencyjne
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: 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)
Inne nazwy:
  • pre-radiation
Eksperymentalny: 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).
Inne nazwy:
  • with radiation

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Proportion of evaluable patients who complete at least 75% of the oral study drug intervention
Ramy czasowe: 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
Ramy czasowe: 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

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Radiographic response rate
Ramy czasowe: 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

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Współpracownicy

Śledczy

  • Krzesło do nauki: Karisa C Schreck, MD, PhD, Johns Hopkins University

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Szacowany)

10 listopada 2026

Zakończenie podstawowe (Szacowany)

10 listopada 2028

Ukończenie studiów (Szacowany)

23 grudnia 2029

Daty rejestracji na studia

Pierwszy przesłany

18 sierpnia 2026

Pierwszy przesłany, który spełnia kryteria kontroli jakości

20 sierpnia 2026

Pierwszy wysłany (Rzeczywisty)

21 sierpnia 2026

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

26 sierpnia 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

25 sierpnia 2026

Ostatnia weryfikacja

1 sierpnia 2026

Więcej informacji

Terminy związane z tym badaniem

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Tak

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

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