Primary Ciliary Dyskinesia
Conditions
Keywords
Primary Ciliary Dyskinesia, PCD, Kartagener Syndrome
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.
Interventions
DRUGRCT1100
RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
Sponsors
ReCode Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Major Inclusion Criteria: * 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. 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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). |
Countries
Australia, New Zealand, United Kingdom, United States
Outcome results
None listed