A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 with Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants with Cystic Fibrosis
Cystic fibrosis is an inherited condition that causes thick, sticky mucus to build up in the lungs and gut, making breathing difficult and leading to frequent chest infections. This early-stage study is testing an experimental medicine called RCT2100 to find out if it is safe and suitable for people. Because this is the first time RCT2100 is being tested in humans, the trial has two parts. Healthy adults will receive single, increasing doses to check basic safety. Adults living with cystic fibrosis will then receive repeated doses over several weeks. Some participants may also take an existing cystic fibrosis medicine called Kalydeco. The main goal is to monitor for any side effects and see how the body absorbs and clears the medicine. Researchers will also check whether the treatment helps improve breathing and day-to-day quality of life for people with cystic fibrosis.
At a glance
What is this study about?
Cystic fibrosis is a lifelong genetic condition that affects thousands of people across the UK. In people with cystic fibrosis, a faulty gene stops salt and water from moving properly in and out of cells. This creates thick mucus that clogs the lungs, making it hard to breathe and easier for serious chest infections to develop.
This trial is investigating a new potential treatment called RCT2100. It is a Phase 1 and 2 trial, which means it is an early-stage study. In fact, this is the first time the medicine is being given to human volunteers. Because safety is the top priority, doctors start with very small doses and slowly increase them while keeping a very close eye on how volunteers respond.
The study involves both healthy volunteers and adults with cystic fibrosis. For the healthy participants, the aim is to check general safety and understand how the drug moves through the bloodstream. For participants who have cystic fibrosis, researchers want to see if the medicine is safe when taken repeatedly, and whether it shows promising early signs of clearing the airways and making breathing easier.
Finding new ways to treat cystic fibrosis is vital. While current treatments have improved many lives, not all medicines work for every person. Early trials like this are the crucial first step towards discovering whether new treatments might offer safe, practical help in the future.
Key takeaways
- This is a Phase 1/2 trial testing a new medicine, RCT2100, in humans for the first time.
- The primary focus is finding out if the drug is safe, well-tolerated, and free from serious side effects.
- The study includes both healthy adults and adults living with cystic fibrosis.
- Participants with cystic fibrosis will be monitored over 4 to 12 weeks to see if their lung function improves.
- Taking part is completely voluntary and you can withdraw at any time.
Who may be eligible?
This study is open to adults aged 18 and older. It includes both healthy volunteers and people who have been diagnosed with cystic fibrosis. Men and women can both take part.
Healthy volunteers will be needed for the earliest part of the trial, where single small doses are tested. To join this group, you must generally be in good health with no significant ongoing medical conditions.
The second part of the trial is specifically for adults with a confirmed diagnosis of cystic fibrosis. Your care team will review your general health, your lung function levels, and any other medications you take—such as Kalydeco—to decide if taking part is medically suitable and safe for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 years or older.
- I am either in good general health, OR I have a confirmed diagnosis of cystic fibrosis.
- I am willing to attend regular hospital or clinic appointments for monitoring.
- I am comfortable having routine blood tests and simple breathing checks.
- I am happy to discuss my current medications with the study doctors.
What does participation involve?
Taking part involves regular visits to a specialist study clinic. Healthy volunteers will take a single dose of the study medicine and stay under observation while medical staff monitor their response. Volunteers with cystic fibrosis will take multiple doses over a period of 4 to 12 weeks.
Throughout the study, you will have regular health checks. These include simple physical examinations, routine blood tests to see how the medicine travels through your body, and heart tracings (ECGs). Participants with cystic fibrosis will also do regular blowing tests (spirometry) to measure lung capacity, and fill out short questionnaires about their daily breathing symptoms.
You will have planned follow-up visits or phone calls with the trial doctors after your final dose to ensure you remain well. The study team will provide a detailed calendar of all appointments before you agree to join.
Potential risks and benefits
Locations (2)
- —UnverifiedFrance
- —UnverifiedNetherlands
Common questions
What is the purpose of this clinical trial?
The main goal is to find out if the experimental drug RCT2100 is safe to use in humans, how the body absorbs it, and whether it shows early promise in improving breathing for people with cystic fibrosis.
Why are healthy people being asked to take part?
Healthy volunteers help researchers understand how a new medicine behaves in the human body without the effects of an underlying illness, providing an important baseline for safety.
What is Kalydeco and why is it used in this study?
Kalydeco is an approved, widely used tablet for certain types of cystic fibrosis. In this study, it may be used alongside RCT2100 to see how the two treatments work together safely.
Will taking part cure my cystic fibrosis?
No. This is an early-stage study designed mainly to check safety and drug behaviour. While researchers hope it will benefit patients in the future, it is not expected to cure the condition.
Can I leave the study if I change my mind?
Yes. Taking part is entirely your choice. You are free to withdraw at any point, and doing so will never affect your relationship with your regular doctors or your standard medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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