RCT1100 for Primary Ciliary Dyskinesia

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and early effectiveness of a new treatment called RCT1100 for individuals with Primary Ciliary Dyskinesia (PCD), a rare lung condition. RCT1100, a type of mRNA therapy, is being studied to assess its tolerability and potential symptom relief. Suitable candidates for this trial have PCD caused by specific genetic mutations in the DNAI1 gene and experience breathing issues. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in humans.

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 there any evidence suggesting that RCT1100 is likely to be safe for humans?

Research shows that RCT1100 is currently being tested for safety in humans. This marks the first use of RCT1100 in people. As the study is in its early phase, it primarily focuses on assessing the treatment's safety and tolerability. No previous information exists about side effects or tolerability, as this is the initial human trial for RCT1100. Researchers are exercising caution and closely monitoring for any issues. The treatment has received orphan drug status from the FDA, indicating its potential as a promising option for treating rare diseases like primary ciliary dyskinesia. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Primary Ciliary Dyskinesia (PCD), which often focus on managing symptoms with antibiotics and therapies to clear airways, RCT1100 offers a novel approach. This treatment is an mRNA therapy specifically designed to target mutations in the DNAI1 gene, which are a common cause of PCD. By addressing the genetic root of the disease, RCT1100 has the potential to improve lung function and overall health more directly and effectively than current options. Researchers are excited because this approach could lead to more personalized and potentially long-lasting benefits for those affected by PCD.

What evidence suggests that RCT1100 might be an effective treatment for Primary Ciliary Dyskinesia?

Research shows that RCT1100 is a new type of treatment called mRNA therapy, developed to help people with primary ciliary dyskinesia (PCD). PCD results from changes in the DNAI1 gene. This therapy replaces faulty genetic instructions with correct ones. In this trial, participants with PCD will receive RCT1100 to evaluate its safety, tolerability, and potential effectiveness. As this treatment is new, most current information focuses on its behavior in the body and its safety for further testing. Early lab research results are promising, but more information is needed to determine its effectiveness in people. ^{1 4 6 7 8}

Who Is on the Research Team?

Kim G Nielsen, Dr Med Sci

Principal Investigator

Rigshospitalet, Denmark

Heymut Omran, MD

Principal Investigator

University Hospital Muenster

John Matthews, MBBS, MCRP, PhD

Principal Investigator

ReCode Therapeutics, Inc.

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05737485

Study Evaluating the Safety and Tolerability of RCT1100 in ... This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.

2. recodetx.com recodetx.com/recode-therapeutics-receives-u-s-fda-orphan-drug-designation-for-rct1100-for-the-treatment-of-primary-ciliary-dyskinesia/

ReCode Therapeutics Receives U.S. FDA Orphan Drug ... ReCode's lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and ...

3. pcd-clinical-studies.com pcd-clinical-studies.com/

A Phase 1b Open-Label Study to Assess RCT1100 in DNAI-1 ... The RCT1100-102 clinical research study is exploring a potential new treatment for individuals with PCD due to disease-causing mutations in the DNAI1 gene.

4. synapse.patsnap.com synapse.patsnap.com/article/first-patient-receives-mrna-therapy-for-primary-ciliary-dyskinesia-in-phase-1-study

First Patient Receives mRNA Therapy for Primary Ciliary ... The focus is on RCT1100, an innovative mRNA-based therapy, which is being assessed for its safety and efficacy in treating primary ciliary ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06600425

NCT06600425 | A Study to Assess the Safety, Tolerability ... This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

6. pharmacytimes.com pharmacytimes.com/view/fda-grants-orphan-drug-designation-to-rct1100-for-primary-ciliary-dyskinesia

FDA Grants Orphan Drug Designation to RCT1100 For ... The FDA has granted RCT1100, a novel investigational inhaled mRNA therapy, orphan drug designation for the treatment of primary ciliary dyskinesia (PCD).

7. clinicaltrialsarena.com clinicaltrialsarena.com/news/recode-primary-ciliary-dyskinesia-trial/

ReCode doses first subject in Phase I primary ciliary ... ReCode Therapeutics has dosed the first subject in a Phase I trial of RCT1100 for primary ciliary dyskinesia caused by DNAI1 gene mutations.

8. recodetx.com recodetx.com/recode-therapeutics-doses-first-patient-in-phase-1-clinical-study-of-novel-mrna-based-therapy-for-the-treatment-of-primary-ciliary-dyskinesia/

ReCode Therapeutics Doses First Patient in Phase 1 ... RCT1100 is the first clinical evaluation of an inhaled mRNA-based therapy designed to restore ciliary function in people with PCD.