Primary Ciliary Dyskinesia (PCD)Phase 1NCT06633757
Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance
A Phase 1 study testing RCT1100 in people with Primary Ciliary Dyskinesia (PCD), run by ReCode Therapeutics at 3 sites in 3 countries.
Trial summary
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Ciliary Dyskinesia (PCD)
- What is being tested?
- RCT1100
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 22 Oct 2024to 10 Feb 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 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.
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- 12-lead ECG with QT interval \>450 msec (or \>480 msec for BBB)
- Laboratory abnormalities in clinical laboratory tests at screening:
- Serum creatinine level
- Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
- 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.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Denmark1 site
Copenhagen University Hospital - Rigshospitalet
Copenhagen, 2100
Germany1 site
Münster University Hospital, Albert-Schweitzer-Campus 1
Münster, North Rhine-Westphalia, 48149
About this study
Official title: A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
Read the detailed description
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.
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Being tested
- RCT1100
- Keywords
- Primary ciliary Dyskinesia, PCD, Kartagener Syndrome
- Medical subject headings
- Ciliary Motility Disorders, Kartagener Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RCT1100Drug
mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
| Study group | Role | What they receive |
|---|---|---|
| PCD Participants | Experimental | RCT1100 mRNA therapy supplied to eligible participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene Receives: Drug: RCT1100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline through Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2024
- Study start22 Oct 2024
- Primary completion30 Jun 2025
- Study completion10 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary Dyskinesia (PCD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReCode Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Matthews, MBBS, MCRP, PhD, Study chair, ReCode Therapeutics, Inc.Heymut Omran, MD, Principal investigator, University Hospital MuensterKim G Nielsen, Dr Med Sci, Principal investigator, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06633757
- Sponsor's protocol ID
- RCT1100-103
- Status last verified
- Apr 2026
- First submitted
- 7 Oct 2024
- Last updated
- 30 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity
- ClinicalTrials.gov ID
- NCT06633757
- Acronym / sponsor ID
- RCT1100-103
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Keywords
- Primary ciliary Dyskinesia, PCD, Kartagener Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- ReCode TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 22 Oct 2024 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 10 Feb 2026 (actual)
- First posted
- 9 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PCD Participants | Experimental | Drug: RCT1100RCT1100 mRNA therapy supplied to eligible participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RCT1100mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer | Drug | PCD Participants |
Primary outcome
To Determine the impact of multiple doses of RCT1100 on MCC
Time frame: Baseline through Week 12
The Change in Mucociliary and Cough Clearance through 1 hour after radiotracer administration by either the albumin-based PRMC or sulfur-colloid technique
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Matthews, MBBS, MCRP, PhD study chair
ReCode Therapeutics, Inc.
Heymut Omran, MD principal investigator
University Hospital Muenster
Kim G Nielsen, Dr Med Sci principal investigator
Copenhagen University Hospital, Rigshospitalet
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RCT1100 in people with Primary Ciliary Dyskinesia (PCD), run by ReCode Therapeutics at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Denmark, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To Determine the impact of multiple doses of RCT1100 on MCC (time frame: Baseline through Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2026.