Primary Ciliary DyskinesiaPhase 1NCT05737485
Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects
A Phase 1 study testing RCT1100 in people with Primary Ciliary Dyskinesia, run by ReCode Therapeutics at 4 sites in 4 countries.
Trial summary
This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability 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
- 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
- 9 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 18 Feb 2023to 13 Jan 2025
- 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
Major Inclusion Criteria:
- Healthy, adult, male or female of, 18-75 years of age, inclusive, at screening.
- Participant has disease causing mutations in the DNAI1 gene
- The 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.
- 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.
- 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
- History of alcohol abuse or drug addiction with the last year of screening.
- Active smoker (vaping included).
Other protocol defined inclusion/exclusion criteria may apply.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
United States1 site
PPD - Las Vegas Research Unit
Las Vegas, Nevada, 89113
About this study
Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100
This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.
Read the detailed description
The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation in the DNAI1 Gene.
- Conditions
- Primary Ciliary Dyskinesia
- 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
RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
| Study group | Role | What they receive |
|---|---|---|
| RCT1100 | Experimental | Drug: RCT1100 single dose Receives: Drug: RCT1100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as an adverse event of special interest (AESI): "Fever", which will include body temperature and any associated symptoms (chills, myalgia).
Measured: From Baseline Through Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2023
- Study start18 Feb 2023
- Primary completion25 Jul 2024
- Study completion13 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary DyskinesiaStudies 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
- 9 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.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05737485
- Sponsor's protocol ID
- RCT1100-101
- Status last verified
- Apr 2025
- First submitted
- 20 Jan 2023
- Last updated
- 29 Apr 2025
- 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 29 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100
- ClinicalTrials.gov ID
- NCT05737485
- Acronym / sponsor ID
- RCT1100-101
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- Primary Ciliary Dyskinesia, PCD, Kartagener Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- ReCode TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 18 Feb 2023 (actual)
- Primary completion
- 25 Jul 2024 (actual)
- Study completion
- 13 Jan 2025 (actual)
- First posted
- 21 Feb 2023 (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 the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.
Detailed description
The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation 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 |
|---|---|---|
| RCT1100 | Experimental | Drug: RCT1100Drug: RCT1100 single dose |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| RCT1100RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer | Drug | RCT1100 |
Primary outcome
The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Time frame: From Baseline Through Day 180
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as an adverse event of special interest (AESI): "Fever", which will include body temperature and any associated symptoms (chills, myalgia).
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 29 Apr 2025.
- 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.
4 sites in 4 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, run by ReCode Therapeutics at 4 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 29 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Australia, New Zealand, United Kingdom. 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?
The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). (time frame: From Baseline Through Day 180). 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 29 Apr 2025.