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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05737485
Other study ID # RCT1100-101
Secondary ID
Status Recruiting
Phase Phase 1
First received
Last updated
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
Health authority
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.

Study Design


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
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