NCT06633757

Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance

A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: ReCode Therapeutics · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Detailed description

The primary objective of this study is to determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.

Conditions

• Primary Ciliary Dyskinesia (PCD)

Interventions

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

Timeline

• Start date: 2024-10-22
• Primary completion: 2025-06-30
• Completion: 2025-12-30
• First posted: 2024-10-09
• Last updated: 2025-10-20

Locations

3 sites across 3 countries: United States, Denmark, Germany

Regulatory

• FDA-regulated drug study