RCT1100 for Primary Ciliary Dyskinesia

Recruiting at 3 trial locations

Overseen By Cathy Vo Buu

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: ReCode Therapeutics

Must not be taking: Anticoagulants

Disqualifiers: Cancer, Tuberculosis, Hemoptysis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

12 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants should not be on anticoagulation therapy for a pulmonary embolus or have had a pulmonary embolus recently. It's best to discuss your specific medications with the trial team.

Is RCT1100 safe for humans?

The safety data for low-dose atropine, which may be related to RCT1100, shows that it is generally safe in humans, with common mild side effects like dilated pupils and photophobia (sensitivity to light). Severe adverse events are rare, and lower doses are associated with fewer side effects. ^{1 2 3 4 5}

Research Team

Heymut Omran, MD

Principal Investigator

University Hospital Muenster

Kim G Nielsen, Dr Med Sci

Principal Investigator

Rigshospitalet, Denmark

John Matthews, MBBS, MCRP, PhD

Principal Investigator

ReCode Therapeutics, Inc.

Eligibility Criteria

This trial is for adults aged 18-75 with PCD (a lung condition) caused by specific genetic mutations in the DNAI1 gene. They must be able to breathe out a certain amount of air quickly, indicating their lungs work at least 40% as well as expected.

Inclusion Criteria

My lung function, measured by FEV1, is at least 40% of the expected value.

I have a confirmed mutation in the DNAI1 gene.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of inhaled RCT1100 via nebulizer to assess mucociliary clearance

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• RCT1100

Trial Overview The study tests RCT1100, an inhaled medication, looking at its safety and how well it works. It's given multiple times to see if it can help clear mucus from the lungs better than current treatments.

Participant Groups

Group I: PCD Participants Experimental Treatment 1 Intervention

RCT1100 mRNA therapy supplied to eligible participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene

Who Is Running the Clinical Trial?

ReCode Therapeutics

Lead Sponsor

Trials

6

Recruited

390+

Findings from Research

In a phase 3 study involving 758 adults with dry eye disease, OC-01 nasal spray significantly improved tear production compared to a placebo, with 47.3% and 49.2% of patients in the OC-01 treatment groups achieving a 10-mm improvement in Schirmer test scores at week 4.

OC-01 was well tolerated, with most treatment-emergent adverse events being mild and nonocular, although a higher percentage of patients in the OC-01 groups reported side effects compared to the vehicle group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease: The ONSET-2 Phase 3 Randomized Trial. Wirta, D., Vollmer, P., Paauw, J., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Low-dose (0.01%) atropine eye-drops to reduce progression of myopia in children: a multicentre placebo-controlled randomised trial in the UK (CHAMP-UK)-study protocol. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

The Effect of Long-Term Low-Dose Atropine on Refractive Progression in Myopic Australian School Children. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Is 0.01% Atropine an Effective and Safe Treatment for Myopic Children? A Systemic Review and Meta-Analysis. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease: The ONSET-2 Phase 3 Randomized Trial. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Incidence of Adverse Events Induced by Atropine in Myopic Children: A Meta-Analysis. [2023]