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

Phase 1

Completed

• Conditions: Primary Ciliary Dyskinesia
• Interventions: Drug: RCT1100

• Registration Number: NCT05737485
• Lead Sponsor: ReCode Therapeutics

Brief 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.

Study Timeline

• Study First Submitted: 2023-01-20
• Study Start: 2023-02-18
• Study First Submitted QC: 2023-02-20
• Study First Posted: 2023-02-21
• Primary Completion: 2024-07-25
• Study Completion: 2025-01-13
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-24
• Last Update Posted: 2025-04-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

• Healthy, adult, male or female of, 18-65 years of age, inclusive, at screening.
• Participant has pathogenic mutations in the DNAI1 gene
• The participant has a forced expiratory volume in one second (FEV1) of at least 50% predicted.

Major

Exclusion Criteria

• History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.

• History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin.

• Medically significant hemoptysis

• Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening.

• Active tuberculosis infection.

• Laboratory abnormalities in clinical laboratory tests at screening:

  1. Serum creatinine level
  2. Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
  3. Hematological or coagulation values outside the normal reference range

• Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN

• History of alcohol abuse or drug addiction with the last year of screening.

• Active smoker (vaping included).

Other protocol defined inclusion/exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RCT1100	RCT1100	Drug: RCT1100 single dose

Primary Outcome Measures

Name	Time	Method
The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).	From Baseline Through Day 180	Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as an adverse event of special interest (AESI): "Fever", which will include body temperature and any associated symptoms (chills, myalgia).

Trial Locations

Locations (4)

New Zealand Clinical Research; 🇳🇿 Auckland, New Zealand

PPD - Las Vegas Research Unit; 🇺🇸 Las Vegas, Nevada, United States

Macquarie University; 🇦🇺 Sydney, New South Wales, Australia

Royal Brompton Hospital; 🇬🇧 London, United Kingdom