The purpose of this clinical trial is to evaluate the safety and tolerability of avutometinib and defactinib and to determine the preliminary antitumour activity of avutometinib and defactinib administered at the recommended Phase 2 dose (RP2D). In the Phase 1b of this study parallel biomarker defined arms will be opened, initially in the relapsed GMB setting, enrolling 12 patients onto each arm. These patients will be treated with avutometinib and defactinib double therapy. Avutometinib will be administered orally at 3.2mg twice a week (e.g., on Monday / Thursday or Tuesday / Friday) with or without a meal. The total weekly dose of avutometinib is 6.4mg. Defactinib will be administered orally, at 200mg, twice a day within 30 min after a meal. The total daily dose of defactinib is 400mg. Once a treatment in any biomarker arm has met the "GO" decision (≥3 successes/12 patients) for relapsed GBM in Phase 1b, that arm can progress to Phase 2. The primary objective of Phase 2 is to determine the antitumour activity of investigational agents administered at the RP2D in patients with molecularly defined malignant brain tumours.
The clinical trial will be divided into two parts: Phase 1b (proof of concept of hypothesis-driven biomarker-guided therapies) and Phase 2 (preliminary efficacy testing). This is a study within Minderoo 5G: A Next Generation AGile Genomically Guided Glioma Modular Platform for proof-of-concept molecular hypothesis testing in patients with high grade malignant brain tumours. 5G-RUBY is a Bayesian multi-centre, multi-arm, open-label, adaptive, seamless Phase 1/2 trial of doublet combination of avutometinib and defactinib and triplet combination with temozolomide for patients with malignant brain tumours. 5G-RUBY will recruit patients with glioblastoma (GBM) into two molecularly-defined biomarker arms of patients who have tumours that harbour: * Hyperactivating BRAF mutations or fusions predicted to be pathogenic by COSMIC * NF1 loss Each biomarker arm, within Phase 1, will have robust GO/ADAPT decision points, reviewed by the Safety Review Committee (SRC) to allow for both agility and clear direction for next steps. A 2-stage Bayesian adaptive design will be performed to assess preliminary efficacy. In the Phase 1b of this study parallel biomarker defined arms will be opened, initially in the relapsed GMB setting, enrolling 12 patients onto each arm. These patients will be treated with avutometinib with a total weekly dose of 6.4mg, and defactinib with a total daily dose of 400mg. Phase 2 efficacy testing will be undertaken in the front line adjuvant MRD setting. This can be either as double combination or in triple combination with temozolomide (concomitantly, or sequentially) depending on emerging data. Further details will be provided at once the SRC has assessed data from Phase 1 and formally opened Phase 2.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Enrollment
182
Supplied as 0.8mg capsules.
Supplied as 200mg tablets.
Temozolomide will be supplied as 5, 20, 100, 140, 180 or 250 mg hard capsules.
Cambridge University Hospitals
Cambridge, United Kingdom
RECRUITINGThe Royal Marsden Hospital - Drug Development Unit
Sutton, United Kingdom
RECRUITINGThe Royal Marsden Hospital - Neuro-Oncology Unit
Sutton, United Kingdom
RECRUITINGPhase 1b - To evaluate the safety and tolerability of investigational agent in patients with malignant brain tumours
To identify the incidence, nature and severity of adverse events and laboratory abnormalities, with severity determined according to NCI CTCAE v5.0
Time frame: 12 months
Phase 1b - To determine the preliminary antitumour activity of the investigational agent administered at the RP2D in patients with molecularly defined malignant brain tumours
Antitumour activity will be defined on the basis of the following outcomes. If any of the following occur, patients will be considered to have clinically benefitted: For relapsed GBM: Achievement of overall response of CR or PR per Response Assessment in Neuro-Oncology (RANO) within 6 months or Free of disease progression or death at 6 months For front line unmethylated GBM (MRD): • Progression-free survival (PFS)
Time frame: 12 months
Phase 2 - To determine the antitumour activity of investigational agent administered at the RP2D in patients with molecularly defined malignant brain tumours
Antitumour activity will be defined on the basis of the following outcomes: Progression-free survival (PFS), defined as the time from enrolment to the first occurrence of disease progression or death from any cause (whichever occurs first), as determined by the investigator according to RANO Overall survival (OS), defined as the time from enrolment to death from any cause
Time frame: 9 months
Phase 1b - To identify molecular determinants of response and antitumour activity of the investigational agent in patients with molecularly selected brain tumours
Determine biomarkers of response, including but not limited to genomic, transcriptomic or immunological signatures from responders and non-responders.
Time frame: 12 months
Phase 2 - To identify molecular determinants of response and antitumour activity of the investigational agent in patients with molecularly selected brain tumours
Determine biomarkers of response, including but not limited to genomic, transcriptomic or immunological signatures from responders and non-responders from archival tumour samples
Time frame: 9 months
Phase 2 - To assess changes in Quality of Life over time
Global changes over time in health-related quality of life (HRQOL) as assessed by the EORTC QLQ C30 and EORTC QLQ BN20 (Baseline, end of C1, C3D1)
Time frame: 9 months
Phase 2 - To assess safety and tolerability of combination with TMZ when given as standard of care maintenance therapy
To identify the incidence, nature and severity of adverse events and laboratory abnormalities, with severity determined according to NCI CTCAE v5.0
Time frame: 9 months
Phase 2 - To assess changes in Quality of Life over time
PFS6, defined as the percentage of patients who remained free of disease progression 6 months from study enrolment
Time frame: 9 months
Phase 2 - To assess changes in Quality of Life over time
OS12, defined as the percentage of patients who remained free of death at 12 months from study enrolment
Time frame: 9 months
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