Experimental mRNA inhalation therapy targets rare lung disease
NCT ID NCT06600425
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tests an inhaled mRNA therapy called RCT1100 in 7 adults with primary ciliary dyskinesia caused by DNAI1 gene mutations. The goal is to see if the treatment is safe and can help restore ciliary function in the lungs. It is a small, open-label trial focused on safety and preliminary effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RCT1100 mRNA therapy
What this could lead to
If successful, this could point toward a treatment that improves ciliary function and respiratory symptoms in people with primary ciliary dyskinesia.
What could go wrong
This is a very early, small Phase 1b study with only 7 participants, so results may not apply broadly. The therapy is still experimental and safety is the main focus.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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