RCT2100 for Cystic Fibrosis

Recruiting at 23 trial locations

Overseen By Priya Riyali, MBA

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: ReCode Therapeutics

Must not be taking: CFTR modulators

Disqualifiers: Hypertension, Smoking, Hepatic impairment, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

192 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called RCT2100 for individuals with cystic fibrosis (CF), a condition that affects the lungs and makes breathing difficult. The study aims to determine if RCT2100 is safe and tolerable. Participants will receive either a single dose, multiple doses of the treatment, or a placebo (a substance with no active drug). Suitable candidates have a confirmed CF diagnosis and are not currently using CFTR modulator therapies due to intolerance or other reasons. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires that participants not use CFTR modulators (specific cystic fibrosis medications) within 12 weeks before screening. Other medications are not specifically mentioned, so it's best to discuss your current medications with the study team.

Is there any evidence suggesting that RCT2100 is likely to be safe for humans?

Research shows that RCT2100 is currently being tested for safety and tolerance in people with cystic fibrosis. This marks the first human trial of RCT2100, so no previous human data exists for this treatment. The trial primarily aims to understand how individuals respond to the treatment and identify potential side effects.

As an early-stage study, it seeks to gather initial information on the body's reaction to RCT2100. The trials are designed to identify any possible risks or negative effects. While early trials involve careful safety monitoring, more data from this study will be needed to fully understand the safety of RCT2100. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about RCT2100 for cystic fibrosis because it offers a fresh approach to managing this condition. Unlike current treatments, which often focus on managing symptoms and improving lung function, RCT2100 is designed to provide more targeted and potentially effective relief with a unique dosing strategy. The treatment involves both single and multiple doses, which could offer flexibility and improved outcomes over time. This innovative approach has the potential to enhance the quality of life for people with cystic fibrosis by addressing the condition in a new way.

What evidence suggests that RCT2100 might be an effective treatment for cystic fibrosis?

Research has shown that RCT2100, an inhaled treatment using mRNA technology, aims to help people with cystic fibrosis by improving the function of the CFTR protein, which often malfunctions in individuals with this condition. This protein plays a crucial role in controlling the movement of salt and water in and out of cells. Correcting this protein could alleviate many symptoms of cystic fibrosis. In this trial, participants will receive either a single or multiple doses of RCT2100, or a placebo. Although RCT2100 is being tested in humans for the first time, it is being developed to potentially aid those who do not benefit from current treatments. Early results suggest that this new approach could be promising for individuals who do not respond well to existing options. ^{1 2 3 4 6}

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, weighing at least 50 kg with a BMI of 16-32. Participants must have good lung function (FEV1 of at least 80% predicted) and be in overall good health as determined by various medical assessments. They need to understand the study procedures and agree to follow the protocol.

Inclusion Criteria

Understands the study procedures in the informed consent form (ICF) and is willing and able to comply with the protocol

Forced expiratory volume in one second (FEV1) of at least 80% predicted

I am a healthy adult between 18 and 55 years old.

Exclusion Criteria

Supine blood pressure (BP) >150 mm Hg (systolic) or >90 mm Hg (diastolic) after at least 5 minutes of rest

Abnormal clinical laboratory tests at screening as assessed by the study-specific laboratory

Arterial oxygen saturation on room air less than 94% at screening

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants

4 weeks

1 visit (in-person)

Treatment Part 2

Multiple ascending doses of inhaled RCT2100 administered to participants with CF

12 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• RCT2100

Trial Overview The trial is testing RCT2100, a new medication, on both healthy participants and those with Cystic Fibrosis. It aims to assess safety and tolerability. Some people will receive RCT2100 while others will get a placebo (a substance with no therapeutic effect) for comparison.

How Is the Trial Designed?

Group I: RCT2100 (Part 2) 4 week Experimental Treatment 1 Intervention

RCT2100 multiple dose

Group II: RCT2100 (Part 2) 12 week Experimental Treatment 1 Intervention

RCT2100 multiple dose

Group III: RCT2100 (Part 1) Experimental Treatment 1 Intervention

RCT2100 single dose

Group IV: Experimental: RCT2100 (Part 3) 6 week Experimental Treatment 2 Interventions

RCT2100 multiple dose

Group V: Placebo (Part 1) Placebo Group 1 Intervention

Placebo single dose

Who Is Running the Clinical Trial?

ReCode Therapeutics

Lead Sponsor

Trials

6

Recruited

390+

Citations

1. recodetx.com recodetx.com/recode-therapeutics-doses-first-patient-in-phase-1b-clinical-study-of-rct2100-for-the-treatment-of-cystic-fibrosis/

ReCode Therapeutics Doses First Patient in Phase 1b ... The first patient has been dosed in a Phase 1b study evaluating RCT2100, an investigational inhaled mRNA therapy for people with cystic fibrosis (CF).

2. cf-clinical-studies.com cf-clinical-studies.com/

CF Phase 1b RCT2100 This clinical study is exploring a potential new inhaled messenger RNA (mRNA) treatment called RCT2100 that may improve the function of the CFTR protein in the ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06237335

NCT06237335 | A Phase 1 Study Evaluating Safety and ... This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to ...

4. recodetx.com recodetx.com/recode-therapeutics-receives-u-s-fda-orphan-drug-designation-for-rct2100-for-the-treatment-of-cystic-fibrosis/

Cystic Fibrosis “We are currently enrolling patients in a Phase 1b study evaluating RCT2100 in people with CF who do not respond to or are intolerant of current ...

5. medrac.web.unc.edu medrac.web.unc.edu/2025/05/cystic-fibrosis-and-mrna-therapy/

The Current Cystic Fibrosis Treatment Landscape and the ... This is the first-in-human study of RCT2100 mRNA in SORT LNP formulation. In part 1, healthy participants of any gender from 18-55 years of age with BMI of ...

6. cysticfibrosis.org.uk cysticfibrosis.org.uk/get-involved/clinical-trials/trialstracker/tt014421

A trial investigating the safety and tolerability of a new ... An early phase study evaluating the safety and tolerability of a new mRNA based therapy (RCT2100) with single doses in people without Cystic Fibrosis.