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

NCT05737485 | Completed Phase 1 FDA Drug

• 1 other identifier: RCT1100-101
• Study Type: interventional
• Target: 9
• Locations: 4 countries
• Sites: 4

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.

Conditions

Primary Ciliary Dyskinesia

Keywords

Primary Ciliary Dyskinesia; PCD; Kartagener Syndrome

Outcome Measures

Primary Outcomes (1)

• The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).

  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).

  From Baseline Through Day 180

Study Arms (1)

RCT1100

EXPERIMENTAL

Drug: RCT1100 single dose

Drug: RCT1100

Interventions

RCT1100 DRUG

RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

RCT1100

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Healthy, adult, male or female of, 18-75 years of age, inclusive, at screening.
• Participant has disease causing mutations in the DNAI1 gene
• The participant has a forced expiratory volume in one second (FEV1) of at least 50% predicted.

You may not qualify if:

• 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:
• Serum creatinine level
• Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
• 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).

Sponsors & Collaborators

• ReCode Therapeutics: lead

Study Sites (4)

PPD - Las Vegas Research Unit

Las Vegas, Nevada, 89113, United States

Location

Macquarie University

Sydney, New South Wales, 2109, Australia

Location

New Zealand Clinical Research

Auckland, New Zealand

Location

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

MeSH Terms

Conditions

Ciliary Motility Disorders; Kartagener Syndrome

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Bronchiectasis; Bronchial Diseases; Respiratory System Abnormalities; Dextrocardia; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Situs Inversus

Study Officials

• John Matthews, MBBS, MCRP, PhD

  ReCode Therapeutics, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2023
• First Posted: February 21, 2023
• Study Start: February 18, 2023
• Primary Completion: July 25, 2024
• Study Completion: January 13, 2025
• Last Updated: April 29, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); AU (1); NZ (1); GB (1)