Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A Phase 1b study of inhaled RCT1100 in Adults with Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance

We are conducting a small early-stage study to test a new inhaled medicine called RCT1100. This medicine is for adults who have a rare and often serious lung condition called Primary Ciliary Dyskinesia, or PCD. Specifically, we're focusing on people whose PCD is caused by a change in a gene called DNAI1. The main goal is to find out if this new medicine can help improve how well their lungs clear away mucus. Better mucus clearance can help reduce infections and breathing problems. This is an early study to understand how the medicine works in the body and if it's safe, rather than seeing if it cures the condition.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Recode Therapeutics Inc.

Enrolment target

Start

28 May 2024

Adults with Primary Ciliary Dyskinesia

What is this study about?

Imagine your airways are like tiny pipes with little hair-like structures called cilia that constantly sweep out dirt and germs. In people with Primary Ciliary Dyskinesia (PCD), these cilia don't work properly, meaning mucus and other things get stuck, leading to infections and breathing difficulties. This can be a challenging condition to manage.

This study is looking at a new medicine called RCT1100, which you would breathe in. It's designed to help the ciliary 'sweepers' work better. We are testing it in adults who have a specific type of PCD caused by a change in a gene called DNAI1. Understanding how this medicine works at an early stage is a crucial step in developing new treatments.

The main aim of this study is to measure something called 'mucociliary clearance.' This simply means how effectively your lungs are clearing away mucus. We want to see if RCT1100 can improve this process in people with this particular type of PCD. This knowledge helps us understand if the medicine has a good chance of helping patients in the future.

Key takeaways

This is an early study for a new inhaled medicine (RCT1100) for PCD.
It's specifically for adults with PCD caused by a DNAI1 gene problem.
The main goal is to measure how well the lungs clear mucus.
Participation involves clinic visits, assessments, and close medical monitoring.
Your safety and understanding are paramount; all questions will be answered.

Who may be eligible?

This study is for adults, meaning you must be 18 years old or older to take part. There is no upper age limit, so older adults are welcome too. This study is open to everyone, whether you are male or female.

To be eligible, you must have a confirmed diagnosis of Primary Ciliary Dyskinesia (PCD). Crucially, your PCD must be specifically caused by a particular problem in a gene called DNAI1. We will need to check your medical records to confirm this.

There might be other health conditions or medications that would prevent you from joining, as the researchers need to make sure it's safe for everyone involved and that the results of the study are clear.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with Primary Ciliary Dyskinesia (PCD)?
Has your doctor told you your PCD is due to a problem with the DNAI1 gene?
Are you able to attend regular clinic appointments?

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

What does participation involve?

If you decide to join, you would be given the study medicine, RCT1100, to breathe in. The study involves several visits to the clinic where doctors and nurses will monitor your health closely. They will conduct various tests, including checking your lung function and measuring how well your lungs clear mucus. What this involves and how long it takes will be explained in full.

You'll be carefully watched to see how your body reacts to the medicine and if you experience any side effects. You will have regular contact with the study team. The full schedule of visits and assessments will be explained to you before you agree to take part, so you know exactly what to expect. The total duration of your participation in the study will also be made clear.

Potential risks and benefits

Taking part in a study like this could offer potential benefits, such as receiving a new medicine that might help improve your lung health, or getting extra medical attention and monitoring. However, as with any medicine, there's always a chance of side effects or that the medicine might not help you. All known potential risks will be fully explained before you decide to take part. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

—
Denmark
—
Germany

Common questions

What is Primary Ciliary Dyskinesia (PCD)?

PCD is a rare lung condition where tiny hair-like structures in your airways, called cilia, don't work properly, leading to mucus build-up and breathing problems.

What does 'inhaled medicine' mean?

It means you breathe the medicine in directly, usually through a special device, so it goes straight to your lungs.

What is the DNAI1 gene?

It's one of many genes that can cause PCD. If there's a problem with it, it can affect how your cilia work.

Will this medicine cure my PCD?

This is an early study to see if the medicine can help, but it's not designed to be a cure for PCD.

What does a 'Phase 1b study' mean?

It's an early stage of human testing, mainly to check if a new medicine is safe and how it works in the body, rather than focusing on its full effectiveness yet.