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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Oncopole Claudius Regaud	Toulouse	France

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH	Essen	Germany
Centre Hospitalier Lyon Sud	Pierre Benite	France
IKF Pneumologie GmbH & Co. KG	Frankfurt	Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Universita’ Degli Studi Di Verona	Verona	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Ludwig Maximilian University Of Munich	Munich	Germany
Universita’ Politecnica Delle Marche	Ancona	Italy
A.O.U. Policlinico G. Martino Di Messina	Messina	Italy
Hopital Beaujon	Clichy	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Ubqsinfpfu Hrxcjblg Cdcjdje	Cologne	Germany
Gcjqyb Uyxxtsnhbx Ffhlwscbo	Frankfurt	Germany

Trial status

Country	Status	Recruitment Start
France	Not recruiting	02.11.2023
Germany	Not recruiting	02.11.2023
Italy	Not recruiting	02.11.2023

Trial locations

Investigated drugs:

3-Amino-N-{[5-[4-[Bis[(2S,3R,4R,5R)-2,3,4,5,6-Pentahydroxyhexyl]Amino]Piperidine-1-Carbonyl]-1,3-Diethyl-Benzimidazol-1-Ium-2-Yl]Methyl}-5H-Pyrrolo[2,3-B]Pyrazine-2-Carboxamide Dihydrochloride Chloride

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.

Investigated diseases:

Cystic fibrosis

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 ID:

2023-504092-25-00

Protocol code:

ET-ENAC-03

Trial Phase:

Therapeutic exploratory (Phase II)

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

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