NCT06237335

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 192 (estimated)
• Sponsor: ReCode Therapeutics · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Accepted

Summary

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

Detailed 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).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
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

Timeline

• Start date: 2024-02-01
• Primary completion: 2026-08-30
• Completion: 2026-12-31
• First posted: 2024-02-01
• Last updated: 2026-01-16

Locations

23 sites across 5 countries: United States, France, Netherlands, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study