A Phase 1b, Single-Center, Open-Label Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene
Overview
- Phase
- Phase 1
- Intervention
- RCT1100
- Conditions
- Primary Ciliary Dyskinesia
- Sponsor
- ReCode Therapeutics
- Enrollment
- 7
- Locations
- 2
- Primary Endpoint
- The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).
- Status
- Completed
- Last Updated
- 6 months ago
Overview
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.
Investigators
Eligibility Criteria
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.
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:
- •Serum creatinine level
- •Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
Arms & Interventions
PCD Participants
RCT1100 mRNA therapy supplied to eligible participants with with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene
Intervention: RCT1100
Outcomes
Primary Outcomes
The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).
Time Frame: 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).