This is a multi-dose 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 determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
UNC
Chapel Hill, North Carolina, United States
Copenhagen University Hospital - Rigshospitalet
Copenhagen, Denmark
Münster University Hospital, Albert-Schweitzer-Campus 1
Münster, North Rhine-Westphalia, Germany
To Determine the impact of multiple doses of RCT1100 on MCC
The Change in Mucociliary and Cough Clearance through 1 hour after radiotracer administration by either the albumin-based PRMC or sulfur-colloid technique
Time frame: Baseline through Week 12
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