RCT1100 for Primary Ciliary Dyskinesia

No longer recruiting at 4 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, Alcohol abuse, others

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called RCT1100 for individuals with Primary Ciliary Dyskinesia, a condition affecting the lungs and airways. The main goal is to determine if RCT1100 is safe and well-tolerated in humans. The trial seeks volunteers with specific gene mutations related to this condition who can exhale at least half the expected air volume in one second. Those diagnosed with this lung condition and meeting the criteria might be suitable for this trial. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I need to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research shows that RCT1100 is under study to assess its safety and tolerability. This marks the first human trial of RCT1100. The primary aim of these studies is to gather initial safety data. As an early-phase trial, information on RCT1100's tolerability remains limited. However, early-phase studies typically focus on identifying side effects and understanding how the body processes the treatment. More detailed safety information will emerge as the studies progress and more participants join. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike most treatments for Primary Ciliary Dyskinesia (PCD), which typically focus on symptom management through antibiotics and airway clearance therapies, RCT1100 offers a novel approach. RCT1100 is a new drug that researchers are excited about because it directly targets the underlying cause of PCD by potentially correcting the faulty cilia function. This unique mechanism of action could not only improve symptoms but also address the root problem, offering hope for more effective management of the condition.

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

Research shows that RCT1100 is a new treatment under study for primary ciliary dyskinesia (PCD), a condition often caused by changes in the DNAI1 gene. RCT1100 aims to fix these genetic problems. Participants in this trial will receive RCT1100 as part of the experimental treatment arm. Although results from human trials are not yet available, the treatment is expected to help correct the genetic defect. Early studies are examining the safety and tolerability of the treatment, which are important first steps in assessing its potential effectiveness. As more research is conducted, the effects on PCD will become clearer. ^{1 3 6 7 8}

Who Is on the Research Team?

John Matthews, MBBS, MCRP, PhD

Principal Investigator

ReCode Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for healthy adults aged 18-55 who are not able to have children. Participants should be in good health as judged by medical history, lab tests, heart checks (ECG), and physical exams. They must understand the study's procedures, agree to follow them, have a BMI between 18 and 35 kg/m2, weigh at least 50 kg, and have normal lung function (FEV1 of at least 80% predicted).

Inclusion Criteria

My BMI is between 18 and 35, and I weigh at least 50 kg.

I am a healthy adult between 18-55 years old and cannot have children.

You can forcefully exhale at least 80% of the predicted volume in one second.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of inhaled RCT1100 via nebulizer

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

180 days

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• RCT1100

Trial Overview The trial is testing RCT1100 for safety and how well it's tolerated in people. It's the first time this drug is being given to humans with the goal of gathering early data that will help design future studies.

How Is the Trial Designed?

Group I: RCT1100 Experimental Treatment 1 Intervention

Drug: RCT1100 single dose

Who Is Running the Clinical Trial?

ReCode Therapeutics

Lead Sponsor

Trials

6

Recruited

390+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06633757

NCT06633757 | Study of Inhaled RCT1100 in Adults With ... This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies. Detailed ...

2. 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.

3. 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 ... Menlo Park, Calif. ... RCT1100 is a novel investigational therapy being developed to treat PCD caused by pathogenic mutations in the DNAI1 gene.

4. 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.

5. 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 ...

6. 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.

7. clinicaltrial.be clinicaltrial.be/en/details/259535?per_page=20&only_recruiting=0&only_eligible=0

Study Evaluating the Safety and Tolerability of RCT1100 i... The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via ...

8. recodetx.com recodetx.com/recode-therapeutics-announces-first-participants-dosed-in-a-phase-1-healthy-volunteer-clinical-trial-of-novel-disease-modifying-genetic-medicine-rct1100-for-the-treatment-of-primary-ciliary-dyskinesi/

Phase 1 trial to evaluate safety and ... Phase 1 trial to evaluate safety and tolerability of a single ascending dose of RCT1100, an inhaled mRNA-based genetic medicine First ...