Trial Outcomes & Findings for Messenger RNA (mRNA)-Based, Personalized Cancer Vaccine Against Neoantigens Expressed by the Autologous Cancer (NCT NCT03480152)

No participants were enrolled on Dose Level 1 - 0.04mg vaccine, nor Cohort 2a/2b, Arm 2, Phase II Maximum Tolerated Dose Arm/Group. Participants vaccination started on Dose Level 2 - 0.13 mg.

Two step registration process. Step One: Patient signed consent to create vaccine. Step Two: Once vaccine is available, patient screened for eligibility criteria. If eligibility criteria were met, registration and enrollment was completed and the patient was treated. One patient died between Step one and Step two and was not treated.

Messenger RNA (mRNA)-Based, Personalized Cancer Vaccine Against Neoantigens Expressed by the Autologous Cancer

Clinical response was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Complete Response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm. Partial Response (PR) is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progression.

Here is the number of non-serious adverse events probably related to treatment assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).

Participants blood samples were assessed by fluorescence-activated cell sorting (FACS), enzyme-linked immune absorbent (ELISA)-spot and human soluble cluster of differentiation 137 (CD137) (4-1BB) upregulation assays. Differences of 2-3 fold in these assays over the baseline measures are indicative of true biologic difference.

Here is the count of participants with non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence.

A DLT is all Grade 3 or greater toxicities related to the messenger ribonucleic acid vaccine with exception of Grade 3 fever, Grade 3 pruritic/itching, Grade 3 fatigue, Grade 3 metabolic laboratory abnormalities without significant clinical sequela that resolves to Grade 2 or less within 7 days, Grade 3 autoimmune toxicity that resolves to Grade 2 or less in 7 days and events that are clearly related to the patient's disease.

A MTD is the highest dose at which ≤1 of 6 patient's experienced a dose limiting toxicity (i.e., All Grade 3 or greater toxicities related to the messenger ribonucleic acid vaccine with exception of Grade 3 fever, Grade 3 pruritic/itching, Grade 3 fatigue, Grade 3 metabolic laboratory abnormalities without significant clinical sequela that resolves to Grade 2 or less within 7 days, Grade 3 autoimmune toxicity that resolves to Grade 2 or less in 7 days and events that are clearly related to the patient's disease.) or the highest dose level studied if DLT's are not observed at any of the dose levels.