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This study evaluates the safety, tolerability, and effects of the drug RCT1100 in adults with primary ciliary dyskinesia (PCD). Patients aged 18-70 with disease-causing mutations in the DNAI1 gene were able to take part. 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, 18-70 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/exclusion criteria may apply.