A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 With Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants With Cystic Fibrosis

ReCode Therapeutics

Summary

This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.

Description

This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1), the safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF (Part 2), and the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF (Part 3).

Eligibility

Age range: 18–60 years
Sex: All
Healthy volunteers: Yes

Detailed criteria

Part 1 Major Inclusion Criteria: * Healthy, adult, male or female, 18-55 years of age, inclusive, at screening. * Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive * The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted * The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination findings at screening. * Understands the study procedures in the info…

Interventions

Drug
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Other
Placebo
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
Drug
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
Drug
RCT2100
RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
Drug
Ivacaftor
ivacaftor administered orally for 6 weeks
Drug
RCT2100
RCT2100 supplied at varying dose strengths. Co- administered via oral inhalation using nebulizer for 4 weeks with ivacaftor after initial 2 weeks of ivacaftor dosing run in period

Locations (23)

The University of Alabama at Birmingham
Birmingham, Alabama
University of Arizona
Tucson, Arizona
Stanford University
Palo Alto, California
UCSD
San Diego, California
National Jewish Health
Denver, Colorado
Emory University
Atlanta, Georgia