NCT05737485

Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: ReCode Therapeutics · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.

Detailed description

The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation in the DNAI1 Gene.

Conditions

• Primary Ciliary Dyskinesia

Interventions

Type	Name	Description
DRUG	RCT1100	RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Timeline

• Start date: 2023-02-18
• Primary completion: 2024-07-25
• Completion: 2025-01-13
• First posted: 2023-02-21
• Last updated: 2025-04-29

Locations

4 sites across 4 countries: United States, Australia, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study