Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects

Study Overview
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Brief Summary
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This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.

Detailed Description
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The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation in the DNAI1 Gene.

Official Title
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A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100

Conditions
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Primary Ciliary Dyskinesia

Intervention / Treatment
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* Drug: RCT1100


Participation Criteria
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Eligibility Criteria
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Major Inclusion Criteria: Healthy, adult, male or female of, 18-65 years of age, inclusive, at screening. Participant has pathogenic 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: 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 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RCT1100<br>Drug: RCT1100 single dose | Drug: RCT1100<br>* RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). | 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). | From Baseline Through Day 180 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Primary Ciliary Dyskinesia, PCD, Kartagener Syndrome