Primary Ciliary Dyskinesia
Conditions
Keywords
Primary Ciliary Dyskinesia, PCD, Kartagener Syndrome
Brief summary
This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
Detailed description
The primary objective of this study is to assess the safety, tolerability, ciliary rescue, pharmacodynamic biomarkers, and preliminary efficacy of RCT1100 following multiple doses of inhaled RCT1100 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing mutations 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 70 Years
Healthy volunteers
No
Inclusion criteria
Major Inclusion Criteria: * Healthy, adult, male or female of, 18-70 years of age, inclusive, at screening. * Participant has clinical diagnosis of PCD and disease-causing mutations in the DNAI1 gene * 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. * Predisposition to bleeding or clinically meaningful hemorrhagic event in the 12 months prior * 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. * 12-lead ECG with QT interval >450 msec (or >480 msec for BBB) * 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. * COVID-19 infection within 4 weeks of Screening or receipt of COVID-19 vaccine within 2 weeks prior to first dose of RCT1100. * Receipt of vaccine with live virus, attenuated live virus, or live viral components within 2 weeks prior to first dose of RCT1100 or to receive these vaccines during treatment or within 8 weeks of completion of study treatment. Other protocol defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs). | From Baseline Through Week 24 | 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
United Kingdom
Outcome results
None listed