A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
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
About This Trial
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Who May Be Eligible (Plain English)
Original Eligibility Criteria
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Treatments Being Tested
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Placebo
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
RCT2100
RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
Ivacaftor
ivacaftor administered orally for 6 weeks
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