This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
PPD - Las Vegas Research Unit
Las Vegas, Nevada, United States
Macquarie University
Sydney, New South Wales, Australia
New Zealand Clinical Research
Auckland, New Zealand
Royal Brompton Hospital
London, United Kingdom
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).
Time frame: From Baseline Through Day 180
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