This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
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 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
7
RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
Royal Brompton Hospital
London, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom
The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) 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 Week 24
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