Experimental Gene-Targeting drug shows promise for rare lung disease
NCT ID NCT06600425
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 28 times
Summary
This early-stage study tested a new drug called RCT1100 in 7 adults with primary ciliary dyskinesia (PCD) due to DNAI1 gene mutations. The main goal was to check safety and tolerability, while also looking for signs that the drug might help restore cilia function. Participants had mild to moderate lung function and were monitored for side effects like fever.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Royal Brompton Hospital
London, SW3 6NP, United Kingdom
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University Hospital Southampton NHS Foundation Trust
Southampton, SO16 6YD, United Kingdom
Conditions
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