RecruitingPHASE2INTERVENTIONAL

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

This study is looking at a new inhaled medicine called RCT2100. It's the very first time this treatment is being given to people. The main goal is to see if it's safe and how well people tolerate it. The study will happen in three parts: First, healthy adults will try a single dose of RCT2100. Then, people with cystic fibrosis (CF) will receive multiple doses. Finally, some people with CF will take RCT2100 along with an existing CF medicine called ivacaftor. All participants will use a nebuliser to take the medicine. This information will help researchers plan future studies for this potential new treatment.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

ReCode Therapeutics

Enrolment target

192

Start

01 Feb 2024

Estimated completion

31 Dec 2026

Cystic Fibrosis

What is this study about?

This research is looking at a new medicine called RCT2100, which is delivered as a mist you breathe in using a nebuliser. This is the very first time this medication will be given to people, so the main focus is to find out if it's safe and how well people can tolerate it. This early step is really important for developing new treatments before they can be tested more widely.

The study is split into three main parts. In the first part, healthy adult volunteers will receive a single dose of RCT2100. This helps the researchers understand how the body reacts to the medicine for the first time in a safe way. After that, the study will move on to people who have cystic fibrosis (CF). In the second part, these participants will receive several doses of RCT2100. Finally, in the third part, some people with CF will take RCT2100 at the same time as another CF medicine called ivacaftor.

The information gathered from all three parts – about safety, how the medicine moves through the body, and any side effects – will be used to help scientists decide if and how to continue developing RCT2100 as a potential new treatment for cystic fibrosis. It’s all about making sure new medicines are as safe and effective as possible.

Key takeaways

This is an early study of a new inhaled medicine, RCT2100, for cystic fibrosis.
The main goal is to check its safety and how well people tolerate it.
It involves healthy volunteers and people with cystic fibrosis.
The medicine is given using a nebuliser.
It will also look at using RCT2100 with an existing CF medicine (ivacaftor).
Participation includes medical checks and careful monitoring.

Who may be eligible?

This study is looking for both healthy adult volunteers and adults with cystic fibrosis. All participants need to be between 18 and 60 years old.

If you're a healthy volunteer, you'd need to be generally well, not smoke, and have healthy lungs and other body functions. Your weight should also be within a certain range.

For those with cystic fibrosis, you need to have a confirmed diagnosis and your lung function should be within certain limits. You might be eligible if other CF medicines (called CFTR modulators) don't work for your specific type of CF, or if you can't take them due to side effects or other reasons. Certain health conditions, like severe liver problems or a recent chest infection, would mean you couldn't take part.

Quick self-check

Are you between 18 and 60 years old?
Do you have a diagnosis of cystic fibrosis, or are you a healthy adult?
Is your lung function (FEV1) within the ranges specified by the study (if you have CF)?
Are you able to use an inhaled nebuliser treatment?
Do you have no severe liver problems or other serious health issues that would exclude you?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you take part in this study, you'll be given the study medicine, RCT2100, or a dummy medicine (placebo) if you're in the first part of the study, using a nebuliser to breathe it in. You'll have several visits to the clinic, which will include physical check-ups, blood and urine tests, heart checks (ECGs), and lung function tests. Researchers will also monitor you closely for any side effects.

The number of visits and the full duration of your participation will depend on which part of the study you are in. Throughout the study, you'll be monitored regularly by the study team. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Potential risks and benefits

Participating in this study might offer some potential benefits, such as contributing to the development of new treatments for cystic fibrosis. For some with CF, it could mean early access to a new medicine. However, there are also potential risks, as this is a new medicine and not all side effects are known. You might experience discomfort from tests or side effects from the study medicine. It's important to remember that you can stop participating at any time.

Locations (23)

The University of Alabama at Birmingham
Birmingham, United States· Recruiting
University of Arizona
Tucson, United States· Recruiting
Stanford University
Palo Alto, United States· Recruiting
UCSD
San Diego, United States· Recruiting
National Jewish Health
Denver, United States· Recruiting
Emory University
Atlanta, United States· Recruiting
Boston Children's Hospital
Boston, United States· Recruiting
New York Medical College
Valhalla, United States· Recruiting
The University of North Carolina at Chapel Hill
Chapel Hill, United States· Recruiting
Oregon Health & Science University
Portland, United States· Recruiting
University of Pittsburgh
Pittsburgh, United States· Recruiting
UT Southwestern Medical Center
Dallas, United States· Recruiting

+11 more sites — see the official record for the full list.

Common questions

What is RCT2100?

RCT2100 is a new medicine that's breathed in through a nebuliser. This study is the first time it will be given to people.

What is a nebuliser?

A nebuliser is a device that turns liquid medicine into a fine mist, which you can then breathe in through a mask or mouthpiece.

What does 'Phase 2' mean for a study?

Phase 2 means that the study is mainly focused on checking the safety of a new medicine and seeing how well people tolerate it, after it has already been tested in labs and sometimes in very small groups of people.

Will I know if I'm getting the real medicine or a placebo?

In some parts of the study, participants might receive a placebo (a dummy medicine with no active ingredients). This is important for comparing effects fairly. You usually won't know which you're getting until after the study has finished.

Can I take part if I already take other CF medicines?

Some people with CF might be able to take part even if they're on other CF medicines. The study specifically looks at taking RCT2100 with ivacaftor, but details would depend on your specific situation and the study rules.

How to find out more

Priya Ryali, MBA

clinicaltrials@recodetx.com 650-629-7900 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.