Evaluating a new inhaled treatment for cystic fibrosis
A Randomised, Double-Blind, Placebo Controlled, Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of a Repeat Dose of Inhaled ETD001 in People With Cystic Fibrosis
This study is testing a new inhaled medication for cystic fibrosis to see if it can improve lung function and is looking for people with the condition to participate.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | Enterprise Therapeutics Ltd Industry-sponsored |
| Locations | 21 sites (Lyon and 20 other locations) |
| Trial ID | NCT06478706 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of ETD001, an inhaled medication, in individuals with cystic fibrosis (CF). The study is divided into two parts: Part A focuses on the safety of ETD001 over a 7-day period, while Part B assesses its impact on lung function over 28 days, with a placebo comparison. Participants will undergo various assessments including physical exams, breathing tests, and health questionnaires throughout the study duration. The trial aims to determine if ETD001 can improve lung function in patients with stable CF.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of cystic fibrosis and stable lung disease.
Not a fit: Patients with severe liver or renal dysfunction, or those who have recently received CFTR modulator therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve lung function and overall health for patients with cystic fibrosis.
How similar studies have performed: While this approach is novel for ETD001, similar inhaled therapies have shown promise in improving lung function in cystic fibrosis patients.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male \& female ≥ 18 years of age, who fit one of the following criteria: Women of childbearing potential using permitted contraception a minimum of 28 days before dosing until completion of the final follow up visit; Women of non-childbearing potential; Men using contraception from the time of the first dose, until completion of the final follow up visit; * Confirmed diagnosis of CF * FEV1 ≥ 40% and ≤ 90% of predicted normal for age, gender, and height * Able to reproducibly perform spirometry manoeuvres * Clinically stable CF lung disease * Routine CF therapy has not changed within 28 days prior to screening. * Provided written informed consent. * Body mass index (BMI) \> 16 and \< 30 kg/m2 Exclusion Criteria: * Abnormal liver function * Abnormal renal function * History of solid organ transplant * Chest x-ray within the past 12 months with abnormalities suggesting unstable pulmonary disease other than CF * Received CFTR modulator therapy in the 60 days before screening * Changes in bronchodilator, corticosteroid or other anti-inflammatory medications 14 days before screening * Unable to withhold use of long-acting bronchodilators 24 hours or short-acting bronchodilators 6 hours before spirometry assessments * Unable to withhold use of anti-cholinergics within 24 hours of spirometry * Started dornase alfa, hypertonic saline, or other airway clearing therapy less than 28 days before screening * Using inhaled antibiotics for less than 2 complete cycles and unable to complete the entire study during the off or on cycle. * Changes in inhaled or oral antibiotic use within 14 days of screening * Taking oral corticosteroids in excess of 10 mg/day or 20 mg every other day within 14 days of screening * Use of diuretics, or renin-angiotensin aldosterone system antihypertensive drugs , drospirenone, or trimethoprim in the 28 days before screening * Presence of co-morbidities and medical history in the opinion of the investigator, may pose additional risk by participating in the study, or may confound the results of the study
Where this trial is running
Lyon and 20 other locations
- Hospices Civils de Lyon — Lyon, France (Not_yet_recruiting)
- CHU de Montpellier — Montpellier, France (Recruiting)
- Hôpital Cochin — Paris, France (Recruiting)
- Hôpitaux de Toulouse — Toulouse, France (Recruiting)
- Charité Universtaetsmedizin — Berlin, Germany (Recruiting)
- CF-Studienzentrum Universitätsklinikum Köln — Cologne, Germany (Recruiting)
- Westdeutsches Lungenzentrum am Universitätsklinikum — Essen, Germany (Recruiting)
- Universitätsklinikum Frankfurt — Frankfurt, Germany (Recruiting)
- IKF Pneumologie — Frankfurt, Germany (Recruiting)
- LMU Kinikum — Munich, Germany (Recruiting)
- Azienda Ospedaliera Universitaria Meyer — Florence, Italy (Recruiting)
- IRCCS Istituto Giannina Gaslini — Genova, Italy (Recruiting)
- Fondazione IRCCS Ca' Granda- Ospedale Maggiore Policlinico — Milano, Italy (Recruiting)
- Ospedale Pediatrico Bambino Gesù — Roma, Italy (Recruiting)
- Azienda Ospedaliera Universitaria Integrata Verona — Verona, Italy (Recruiting)
- Belfast Health and Social Care Trust — Belfast, United Kingdom (Recruiting)
- Royal Papworth Hospital — Cambridge, United Kingdom (Recruiting)
- All Wales Adult CF Centre — Cardiff, United Kingdom (Recruiting)
- Queen Elizabeth University Hospital West of Scotland CF Service — Glasgow, United Kingdom (Recruiting)
- Royal Brompton Hospital — London, United Kingdom (Recruiting)
- Southampton General Hospital — Southampton, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Enterprise Information
- Email: info@enterprisetherapeutics.com
- Phone: +44 1273 234667
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.