Inhaled mRNA therapy shows promise for rare lung disease

NCT ID NCT06633757

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tested an inhaled mRNA therapy called RCT1100 in 14 adults with primary ciliary dyskinesia (PCD) caused by a specific genetic mutation (DNAI1). The goal was to see if the treatment could improve mucociliary clearance — the lungs' ability to clear mucus and germs. The study focused on safety and preliminary effectiveness to guide future research.

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 inhaled via nebulizer)

What this could lead to

If successful, this could point toward a treatment that improves lung clearance and respiratory function in people with PCD caused by DNAI1 mutations.

What could go wrong

This is a very early Phase 1b trial with only 14 participants, so results may not apply to all PCD patients. The therapy is still experimental and safety or efficacy are not yet proven.

Disclaimer Read more

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.

primary ciliary dyskinesia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Copenhagen University Hospital - Rigshospitalet

    Copenhagen, 2100, Denmark

  • Münster University Hospital, Albert-Schweitzer-Campus 1

    Münster, North Rhine-Westphalia, 48149, Germany

  • UNC

    Chapel Hill, North Carolina, 27514, United States