Clinical Trial to Evaluate the Safety of RQ-01 in SARS-CoV-2 Positive Subjects

Overview

This is a first in human clinical study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of RQ-01. Adult patients who have tested positive for SARS-CoV-2 virus, have mild symptom(s) of COVID-19 disease, and who are at low risk for developing moderate or severe COVID-19 disease are welcome to participate. The main questions this study aims to answer are: 1. What is the safety profile of RQ-01? 2. What is the reduction in SARS-CoV-2 ribonucleic acid (RNA) shedding after administration of RQ-01? 3. How effective is RQ-01 versus placebo in reducing COVID-19 symptoms? After providing informed consent, subjects will be randomized to RQ-01 (high or low dose) or placebo and will be dosed for 3 consecutive days. Throughout the duration of the trial, subjects will participate in the following activities: perform rapid antigen and polymerase chain reaction (PCR) tests for COVID-19, answer questions about their medical history and medications, answer questions about their COVID-19 history and symptoms, conduct a physical exam, have their vitals measured, and have bloodwork done.

This is a first in human study to evaluate the safety, PK, and PD of RQ-01 when administered to adult subjects who have tested positive for SARS-CoV-2 virus, have mild symptom(s) of COVID-19 disease, and who are at low risk for developing moderate or severe COVID-19 disease. Subjects will be exposed to RQ-01 after nasal administration with the Aptar Bidose nasal spray device (BDSI V3). The decrease in SARS-CoV-2 RNA shedding will be measured by reverse transcription-quantitative polymerase chain reaction (RT- qPCR) compared with placebo. There are currently no Food and Drug Administration (FDA) approved therapies for subjects with mild symptoms of COVID who are low risk for progression to moderate-severe disease. Safety of the subjects in this study is supported by the safety margin for local adverse events, lack of systemic exposure, and availability of antiviral therapies for COVID-19 should subjects' disease progress. The study may be conducted using traditional outpatient clinic sites or a hybrid model, using clinic sites and/or virtual sites, with provisions made to conduct aspects of the study via telehealth and/or within subjects' homes. The hybrid model will provide the flexibility required to offer the trial in areas with increased incidence of COVID-19 disease, to maximize access to trial participation, and to increase diversity of trial subjects. Potential study subjects will be identified for screening based on having at least one mild symptom consistent with COVID-19 disease. Potential study subjects may additionally present with a positive SARS-CoV-2 rapid antigen test (RAT), taken either at-home, or administered at a facility (e.g., urgent care testing site). As detailed within the inclusion criteria, different entry requirements apply depending on the source and timing of the positive rapid antigen test. For all subjects, the time from first positive SARS-CoV-2 rapid antigen test result to time of first dose must not exceed 3 days. Also, the first dose of study drug must be administered within 4 days of the estimated initial onset of symptoms of COVID-19 disease. Subjects who provide written informed consent and meet all eligibility criteria will be randomized in a 1:1:1 manner to RQ-01 (high or low dose) or placebo. On the day of randomization, all subjects must have at least one mild symptom of COVID-19 disease and a positive SARS-CoV-2 RAT result. After the first six subjects are enrolled (2 in each group) and have completed all study procedures through Day 7 an early interim safety analysis will be performed by the Data Monitoring Committee. Enrollment of new subjects will continue only after review by the Data Monitoring Committee and the decision to proceed communicated to the investigators. Prior to the first dose of study drug, all subjects must be trained by study staff on study drug self-administration with the Aptar Bidose nasal spray device, per the instructions provided within the Pharmacy Manual and/or subject-facing materials. Subjects will self-administer all doses of study drug while observed in-person by the Investigator or designee, who must be a trained healthcare professional. Subjects will then be followed for an additional 30 days for overall safety including COVID-19 disease symptoms. If at any point during the study a subject meets the treatment discontinuation criteria outlined within the protocol, including a worsening of COVID-19 disease symptoms, the subject will be immediately discontinued from study drug and will continue in follow-up as described within the protocol.