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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.

Descripción general del estudio

Descripción detallada

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.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

22

Fase

  • Fase temprana 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

  • Nombre: Michaella Iacoboni, RN
  • Número de teléfono: 410-955-8837
  • Correo electrónico: msheeh13@jhmi.edu

Ubicaciones de estudio

    • Georgia
      • Atlanta, Georgia, Estados Unidos, 30322
    • Maryland
      • Baltimore, Maryland, Estados Unidos, 21287
        • Johns Hopkins SKCCC
        • Contacto:
          • Karisa C Schreck, MD, PhD
          • Número de teléfono: 410-955-8837
          • Correo electrónico: ksolt1@jhmi.edu

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

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 de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: 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)
Otros nombres:
  • pre-radiation
Experimental: 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).
Otros nombres:
  • with radiation

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Proportion of evaluable patients who complete at least 75% of the oral study drug intervention
Periodo de tiempo: 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
Periodo de tiempo: 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

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Radiographic response rate
Periodo de tiempo: 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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Colaboradores

Investigadores

  • Silla de estudio: Karisa C Schreck, MD, PhD, Johns Hopkins University

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

10 de noviembre de 2026

Finalización primaria (Estimado)

10 de noviembre de 2028

Finalización del estudio (Estimado)

23 de diciembre de 2029

Fechas de registro del estudio

Enviado por primera vez

18 de agosto de 2026

Primero enviado que cumplió con los criterios de control de calidad

20 de agosto de 2026

Publicado por primera vez (Actual)

21 de agosto de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

26 de agosto de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

25 de agosto de 2026

Última verificación

1 de agosto de 2026

Más información

Términos relacionados con este estudio

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

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