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

Primary Ciliary Dyskinesia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05737485
• Other study ID #: RCT1100-101
• Status: Recruiting
• Phase: Phase 1
• Start date: February 18, 2023
• Est. completion date: December 21, 2024

Study information

• Verified date: December 2023
• Source: ReCode Therapeutics
• Contact: Priya Ryali, MBA
• Phone: 650-629-7900
• Email: clinicaltrials[@]recodetx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 9
• Est. completion date: December 21, 2024
• Est. primary completion date: September 21, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Major 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.

Related Conditions & MeSH terms

• Ciliary Motility Disorders
• Dyskinesias
• Primary Ciliary Dyskinesia

Intervention

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

Locations

Country	Name	City	State
Australia	Macquarie University	Sydney	New South Wales
New Zealand	New Zealand Clinical Research	Auckland
United Kingdom	Royal Brompton Hospital	London
United States	PPD - Las Vegas Research Unit	Las Vegas	Nevada
United States	University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
ReCode Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  New Zealand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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