NCT06600425

A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD

A Phase 1b, Single-Center, Open-Label Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: ReCode Therapeutics · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is the second in-human 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 assess the safety, tolerability, ciliary rescue, pharmacodynamic biomarkers, and preliminary efficacy of RCT1100 following multiple doses of inhaled RCT1100 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing mutations 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: 2024-09-20
• Primary completion: 2025-06-06
• Completion: 2025-08-27
• First posted: 2024-09-19
• Last updated: 2025-10-08

Locations

2 sites across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study