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Study on the Effects of Inhaled ETD001 for People with Cystic Fibrosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Cystic Fibrosis, a genetic condition that affects the lungs and digestive system. The trial will test a new treatment called ETD001, which is administered as a nebulizer solution. A nebulizer is a device that turns liquid medicine into a mist, making it easier to inhale into the lungs. The study will compare the effects of ETD001 with a placebo to understand its safety and effectiveness.

The purpose of the study is to evaluate how well ETD001 works and how safe it is for people with Cystic Fibrosis. Participants will use the ETD001 treatment or a placebo over a period of time, and researchers will monitor their lung function and overall health. The study is divided into two parts: the first part focuses on the safety and tolerability of the treatment, while the second part assesses its impact on lung function, specifically measuring the forced expiratory volume in one second (FEV1), which is a common test to check how well the lungs are working.

Throughout the study, participants will use the eFlow® Nebulizer System, a quiet and lightweight device that helps deliver the medication directly to the lungs. The trial aims to provide valuable information on the potential benefits of ETD001 for individuals living with Cystic Fibrosis, contributing to the development of new treatment options for this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, you will attend an initial visit. During this visit, you will undergo various assessments to confirm your eligibility. These assessments include a review of your medical history, a physical examination, and tests to measure your lung function.

    You will be asked to provide written informed consent, confirming your willingness to participate in the study and comply with the study procedures.

  2. Step 2

    Baseline assessments

    Before starting the treatment, baseline assessments will be conducted. These assessments include measuring your lung function, vital signs, and other clinical laboratory tests.

    These baseline measurements are important to compare with future results to evaluate the effects of the treatment.

  3. Step 3

    Treatment period

    You will be randomly assigned to receive either the study medication ETD001 or a placebo. The medication is administered as an inhaled solution using a nebulizer.

    The treatment involves repeat doses, and you will be required to use the nebulizer as instructed by the study team. The exact dosage and frequency will be provided to you during the study.

  4. Step 4

    Follow-up visits

    Throughout the study, you will attend regular follow-up visits. During these visits, your health will be monitored, and additional assessments will be conducted to evaluate the safety and effectiveness of the treatment.

    These visits are crucial to ensure your well-being and to gather data on the study medication's impact on your condition.

  5. Step 5

    Final visit

    At the end of the treatment period, you will have a final visit. This visit will include a comprehensive assessment similar to the initial visit, including lung function tests and other clinical evaluations.

    The final visit helps determine the overall effects of the treatment and ensures that you are in good health before concluding your participation in the study.

Who can join the trial?

14 criteria

  • All genders aged 18 years or older can participate.
  • Women who can have children must agree to use birth control from 28 days before the first dose until the end of the study.
  • Women who cannot have children include those who have not had a period for over 12 months and show signs of menopause, or those who have had surgery to remove the uterus or ovaries.
  • Men must agree to use birth control from the first dose until the end of the study.
  • Participants must be able to use a nebuliser (a device that turns liquid medicine into a mist to be inhaled).
  • Body mass index (BMI) must be between 16 and 30 kg/m².
  • Must have a confirmed diagnosis of Cystic Fibrosis (CF) with specific test results or genetic mutations.
  • Must have a lung function test result (FEV1) between 40% and 90% of what is expected for their age, gender, and height.
  • Must be able to perform lung function tests correctly.
  • Must have stable CF lung disease, meaning no significant worsening of symptoms or lung function in the last 28 days.
  • Routine CF treatments must not have changed in the last 28 days.
  • Must provide written consent to participate in the study.
  • Must be able and willing to follow study instructions and procedures.
  • Females must have a negative pregnancy test at the start of the study.

Who cannot join the trial?

7 criteria

  • People who do not have Cystic Fibrosis cannot participate.
  • Children under the age of 3 cannot participate.
  • People who are not willing or able to follow the study procedures cannot participate.
  • People who have other serious health conditions that might interfere with the study cannot participate.
  • Women who are pregnant or breastfeeding cannot participate.
  • People who have participated in another clinical trial recently may not be able to participate.
  • People who are allergic to any of the study medications cannot participate.
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Investigated drugs

ETD001 is an experimental medication being tested in this clinical trial for people with Cystic Fibrosis. It is administered through inhalation, which means you breathe it in so it can directly reach your lungs. The main goal of using ETD001 in this study is to see if it is safe and well-tolerated by patients. Additionally, the trial aims to find out if ETD001 can help improve lung function, specifically by measuring how much air you can forcefully exhale in one second. This is an important measure for people with Cystic Fibrosis, as it can indicate how well the lungs are working.

What is already known about the treatment

ETD001 – ETD001 is administered as an inhaled medication, designed specifically for individuals with Cystic Fibrosis. Currently, it is being studied in clinical trials to evaluate its safety, tolerability, and effectiveness. The main therapeutic indication for ETD001 is to improve lung function in people with Cystic Fibrosis, as measured by the percent predicted forced expiratory volume in one second (ppFEV1). At the molecular level, ETD001 works by targeting specific pathways in the lungs to help clear mucus and improve breathing. It is classified pharmacologically as a respiratory agent.

Investigated diseases

Cystic Fibrosis – Cystic Fibrosis is a genetic disorder that affects the respiratory, digestive, and reproductive systems. It is characterized by the production of thick and sticky mucus that can clog the airways and lead to respiratory issues. Over time, this mucus buildup can cause persistent lung infections and damage to the lungs. In the digestive system, it can obstruct the pancreas, preventing the release of digestive enzymes necessary for breaking down food. This can lead to malnutrition and poor growth. The disease progresses with increasing respiratory difficulties and complications in other organs due to mucus accumulation.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-504092-25-00Protocol codeET-ENAC-03Estimated enrolment60 patientsSponsorEnterprise Therapeutics Limited

sourced from the EU Clinical Trials Register and site verification

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