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Experimental mRNA inhalation therapy targets rare lung disease

NCT ID NCT06600425

Disease control Sponsor: ReCode Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

7 people

The number who actually took part.

Started

Sep 2024

Finished

Aug 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Major Inclusion Criteria: * Healthy, adult, male or female of, 18-70 years of age, inclusive, at screening. * Participant has clinical diagnosis of PCD and disease-causing mutations in the DNAI1 gene * Participant has a forced expiratory volume in one second (FEV1) of at least 50% predicted. Major Exclusion Criteria: * History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease. * History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin. * Predisposition to bleeding or clinically meaningful hemorrhagic event in the 12 months prior * Medically significant hemoptysis. * Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening. * Active tuberculosis infection. * 12-lead ECG with QT interval \>450 msec (or \>480 msec for BBB) * Laboratory abnormalities in clinical laboratory tests at screening: 1. Serum creatinine level 2. Total bilirubin, aspartate aminotransferase or alanine aminotransferase values 3. Hematological or coagulation values outside the normal reference range * Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN. * COVID-19 infection within 4 weeks of Screening or receipt of COVID-19 vaccine within 2 weeks prior to first dose of RCT1100. * Receipt of vaccine with live virus, attenuated live virus, or live viral components within 2 weeks prior to first dose of RCT1100 or to receive these vaccines during treatment or within 8 weeks of completion of study treatment. Other protocol defined inclusion/exclusion criteria may apply.

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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.

Primary ciliary dyskinesia

Contacts and locations

Locations

  • Royal Brompton Hospital

    London, SW3 6NP, United Kingdom

  • University Hospital Southampton NHS Foundation Trust

    Southampton, SO16 6YD, United Kingdom

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