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This study evaluated the safety and tolerability of a treatment called RCT1100 in healthy adults and in people with primary ciliary dyskinesia (PCD). It enrolled people aged 18-75 who had disease-causing mutations in the DNAI1 gene and a certain level of lung function. It was conducted in Phase 1 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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/exclusion criteria may apply.