Status:
COMPLETED
A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD
Lead Sponsor:
ReCode Therapeutics
Conditions:
Primary Ciliary Dyskinesia
Eligibility:
All Genders
18-70 years
Phase:
PHASE1
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 a...
Eligibility Criteria
Inclusion
- Major
- 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
- 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
- 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/exclusion criteria may apply.
Key Trial Info
Start Date :
September 20 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 27 2025
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT06600425
Start Date
September 20 2024
End Date
August 27 2025
Last Update
October 8 2025
Active Locations (2)
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1
Royal Brompton Hospital
London, United Kingdom, SW3 6NP
2
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom, SO16 6YD