The purpose of this randomized study is to determine the safety and efficacy of quaratusugene ozeplasmid (Reqorsa) added to osimertinib in NSCLC patients with activating EGFR mutations who have progressed while on treatment with osimertinib. Quaratusugene ozeplasmid consists of non-viral lipid nanoparticles that encapsulate a DNA plasmid with the TUSC2 tumor suppressor gene and is the first systemic gene therapy for cancer. The study is comprised of a Phase 1 dose escalation portion and two Phase 2 portions evaluating safety and efficacy. Enrollment in the Phase 1 dose escalation portion is complete and the recommended Phase 2 dose (RP2D) was determined. Phase 2a has initiated and enrolled patients are treated with quaratusugene ozeplasmid at the RP2D in combination with osimertinib. In Phase 2b, patients will be randomized to receive either quaratusugene ozeplasmid plus osimertinib or platinum-based chemotherapy.
Acclaim-1 is an open-label, multi-center, Phase 1/2 study evaluating quaratusugene ozeplasmid (Reqorsa) plus osimertinib (investigational arm) versus platinum-based chemotherapy (control arm) in patients with advanced metastatic or recurrent NSCLC. Toxicities will be assessed by the Investigator using United States National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Serious Adverse Events and Dose Limiting Toxicities (DLT) will be reviewed by a Safety Review Committee. Phase 1 - Dose Escalation: The RP2D of quaratusugene ozeplasmid when given in combination with osimertinib has been identified. Phase 2a: This expansion cohort will be enrolled to better characterize safety, tolerability, and preliminary anti-tumor activity of the combination therapy. Phase 2b: Quaratusugene ozeplasmid in combination with osimertinib will be further evaluated using the RP2D identified in Phase 1. Patients may receive local therapy, such as radiation therapy, to progressing lesions prior to enrollment. Patients will be randomized to receive either the investigational arm or the control arm in a 1 to 1 ratio and stratified based on prior local radiotherapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
158
Quaratusugene ozeplasmid is an experimental non-viral immunogene therapy utilizing the TUSC2 gene, designed to target cancer cells by interrupting cell signaling pathways that allow cancer cells to grow, reestablishing pathways that promote cancer cell death and modulating the immune response against cancer cells.
Osimertinib is a 3rd generation EGFR tyrosine kinase inhibitor (TKI) oral tablet administered daily, as indicated for treatment of patients with metastatic NSCLC whose tumors have EGFR genetic deletions or mutations.
Cisplatin and carboplatin are intravenously administered platinum agents that are combined with other cytotoxic chemotherapy agents such as pemetrexed.
Valkyrie Clinical Trials
Los Angeles, California, United States
RECRUITINGRocky Mountain Cancer Centers
Lone Tree, Colorado, United States
RECRUITINGCarle Cancer Institute
Urbana, Illinois, United States
RECRUITINGMarkey Cancer Center
Lexington, Kentucky, United States
RECRUITINGMaryland Oncology Hematology
Rockville, Maryland, United States
RECRUITINGThe Valley Hospital - Luckow Pavilion
Paramus, New Jersey, United States
RECRUITINGGabrail Cancer Center Research
Canton, Ohio, United States
RECRUITINGMillennium Oncology
Houston, Texas, United States
TERMINATEDVirginia Cancer Specialists
Fairfax, Virginia, United States
RECRUITINGVirginia Oncology Associates
Norfolk, Virginia, United States
RECRUITINGRecommended Phase 2 Dose (RP2D) - Phase 1
RP2D, which will be the maximum tolerated dose (MTD) or, if the MTD is not defined by the safety data, RP2D will be determined based on an integrated assessment of all available clinical safety and preliminary efficacy data.
Time frame: First 21-day treatment cycle for each dose level cohort
Overall Response Rate (ORR) - Phase 2a
ORR (complete response \[CR\]+ partial response \[PR\]) according to RECIST using best overall response.
Time frame: Approximately 3 months
Progression-free Survival (PFS) - Phase 2b
PFS from randomization to disease progression or death. Response according to RECIST.
Time frame: Approximately 11 months
Progression-free Survival (PFS) - Phase 1
PFS from first dose to disease progression or death. Response according to RECIST.
Time frame: Approximately 9 months
Overall Response Rate (ORR) - Phase 1
ORR (CR+ PR) according to RECIST using best overall response.
Time frame: Approximately 3 months
Duration of Response (DOR) - Phase 1
DOR (CR + PR) from response to disease progression. Response according to RECIST.
Time frame: Approximately 9 months
Pharmacokinetics (PK) of Quaratusugene Ozeplasmid - Phase 1
Concentration of quaratusugene ozeplasmid in whole blood samples.
Time frame: First 21-day treatment cycle
Progression-free Survival (PFS) - Phase 2a
PFS from first dose to disease progression or death. Response according to RECIST.
Time frame: Approximately 11 months
Time to Progression (TTP) - Phase 2a
TTP from first dose to disease progression. Response according to RECIST.
Time frame: Approximately 11 months
Overall Survival (OS) - Phase 2a
OS from first dose until death or discontinuation due to withdrawal of consent.
Time frame: Approximately 21 months
Pharmacokinetics (PK) of Quaratusugene Ozeplasmid - Phase 2a
Concentration of quaratusugene ozeplasmid in whole blood samples.
Time frame: Approximately 22 days
Overall Response Rate (ORR) - Phase 2b
ORR (CR+ PR) according to RECIST using best overall response.
Time frame: Approximately 3 months
Time to Progression (TTP) - Phase 2b
TTP from first dose to disease progression. Response according to RECIST.
Time frame: Approximately 11 months
Duration of Response (DOR) - Phase 2b
DOR (CR + PR) according to RECIST from response to disease progression.
Time frame: Approximately 11 months
Overall Survival (OS) - Phase 2b
OS from randomization to death or discontinuation due to withdrawal of consent.
Time frame: Approximately 21 months
Incidence of Adverse Events - Phase 2b
Treatment-related adverse events (AEs) graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events.
Time frame: Approximately 11 months
Pharmacokinetics (PK) of Quaratusugene Ozeplasmid - Phase 2b
Concentration of quaratusugene ozeplasmid in whole blood samples.
Time frame: Approximately 22 days
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