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CompletedPhase IIInterventional

A two-part study to assess the effectiveness, safety, and blood levels of repeat doses of inhaled ETD001 in people with cystic fibrosis

Cystic fibrosis causes thick, sticky mucus to build up in the lungs, making breathing difficult and leading to frequent chest infections. This clinical trial is testing a new inhaled medicine called ETD001 to find out if it can safely loosen this mucus and help clear the airways. The medicine is taken as a mist through a nebuliser twice a day, mostly at home. The trial takes place in two parts. Part A checks the safety of the medicine over one week. Part B tests whether it improves lung health over four weeks, comparing it against a dummy medicine (placebo). Around 30 adults with cystic fibrosis across the UK and Europe will take part. Participants continue taking their usual cystic fibrosis therapies throughout the study. Doctors will closely monitor lung health, heart activity, and blood levels to check how well the treatment works.

At a glance

Status
Completed
Phase
Phase II
Sponsor
Enterprise Therapeutics Ltd
Enrolment target
60
Start
26 Jun 2024
Estimated completion
15 Aug 2025
Cystic fibrosis

What is this study about?

Cystic fibrosis is an inherited condition that causes the body to produce thick, sticky mucus. This mucus builds up in the lungs and digestive system, making it harder to breathe and increasing the risk of chest infections. While modern treatments have greatly improved life for many people with cystic fibrosis, existing medicines do not work for everyone, and daily treatment routines can be challenging to maintain.

This study is testing an experimental medicine called ETD001. The drug is designed to thin the mucus in the lungs, making it less sticky and easier to cough up. If successful, this could help protect lung health and reduce chest infections. The medicine is inhaled directly into the airways as a fine mist using an ordinary nebuliser device.

Because this is the first time ETD001 is being given to people with cystic fibrosis, the study is divided into two steps. The first step tests small numbers of patients to ensure the drug is safe and well tolerated. The second step measures whether the drug actually improves breathing and lung function over several weeks. The findings from this trial will help researchers decide if ETD001 could become a valuable new treatment option in the future.

Key takeaways

  • Phase II clinical trial testing an inhaled medicine called ETD001 for adults with cystic fibrosis.
  • The treatment aims to loosen sticky lung mucus to make breathing easier and prevent infections.
  • The drug is taken twice a day via a nebuliser at home alongside your standard daily medicines.
  • The trial involves clinic visits for breathing tests, heart checks, and blood samples over two to four months.
  • Taking part is entirely voluntary, and you can withdraw at any time.

Who may be eligible?

This trial is open to adults aged 18 and over who have a confirmed medical diagnosis of cystic fibrosis. To take part, your lung health must be relatively stable, meaning you have not had a sudden chest flare-up, high fever, or significant drop in your breathing test scores in the past four weeks.

Your breathing capacity (measured by a standard test called FEV1) must be between 40% and 90% of what is normally expected for your age, sex, and height. You also need to be able to blow reliably into a breathing machine so doctors can accurately track any changes in your lungs.

Because the effects of ETD001 on an unborn baby are unknown, male and female participants who could have children must agree to use reliable contraception throughout the study. You will not be asked to stop taking your regular cystic fibrosis treatments.

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.

  1. I am aged 18 or older.
  2. I have a confirmed diagnosis of cystic fibrosis.
  3. My chest and breathing have been stable with no sudden flare-ups in the past four weeks.
  4. My lung function (FEV1) is between 40% and 90% of the predicted level.
  5. I am able and willing to use reliable contraception during the trial if applicable.
Answer every question to see your result.

What does participation involve?

Taking part involves inhaling the study medicine twice a day using a nebuliser, with most doses taken at home. The trial has two different stages. In Part A, eight participants take either ETD001 or a dummy medicine for 7 days, attending five clinic visits over about two months. In Part B, 32 participants take ETD001 for 28 days and a dummy medicine for another 28 days, attending eight clinic visits over around four to five months.

During hospital visits, the study team will carry out standard health checks. These include physical examinations, blood and urine tests, heart traces (ECGs), and breathing tests (spirometry). You will also complete questionnaires about how you feel. Your health will be monitored closely throughout the trial, and you can contact the medical team at any time.

Potential risks and benefits

You may not get any direct health benefit from taking part, but your involvement could help improve future treatments for people with cystic fibrosis. As with all new medicines, there is a risk of side effects. In earlier tests with healthy volunteers, ETD001 was generally well tolerated, but some people reported mild headaches, chest discomfort, coughing, breathlessness, or a sore throat. Routine tests like blood draws may cause brief stinging or bruising, and breathing tests might leave you feeling briefly tired or dizzy. Taking part is completely voluntary, and you are free to leave the trial at any point without it affecting your regular NHS medical care.

Locations (21)

  • Royal Brompton Hospital
    Approximate
    London, England
  • Queen Elizabeth University Hospital West of Scotland CF Service
    City only
    Glasgow, Scotland
  • Southampton General Hospital
    Approximate
    Southampton, England
  • Belfast Health and Social Care Trust
    City only
    Belfast, Northern Ireland
  • Royal Papworth Hospital NHS Foundation Trust
    City only
    Cambridge, England
  • All Wales Adult CF Centre
    Unverified
    Cardiff, Wales
  • CF-Studienzentrum
    Unverified
    Köln, Germany
  • Universitätsklinikum Frankfurt
    Unverified
    Frankfurt am Main, Germany
  • LMU Klinikum
    Unverified
    München, Germany
  • IKH Pneumologie GmbH & Co.KG
    Unverified
    Frankfurt am Main, Germany
  • Westdeutsches Lungenzentrum
    Unverified
    Essen, Germany
  • Charité Universtaetsmedizin Berlin
    Unverified
    Berlin, Germany

Common questions

Will I have to stop taking my normal cystic fibrosis medicines?

No. The study medicine is designed to be taken alongside your usual prescribed cystic fibrosis therapies.

How do I take the study drug?

You inhale it as a liquid mist twice a day using a nebuliser machine, which you will use at home.

What is a placebo?

A placebo is a dummy medicine that looks and feels identical to the real drug but contains no active ingredients. It helps researchers fairly measure the true effects of the study drug.

Can I leave the study if I change my mind?

Yes. You can leave the study at any time without giving a reason, and your regular medical care will not be affected.

Where does the trial take place?

The study is running at specialist hospital clinics across the UK, France, Germany, and Italy.

How to find out more

Kathy Woodward

Official trial record

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

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