A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis

NCT ID: NCT06478706

Last Updated: 2025-11-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 57 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-26
• Study Completion Date: 2025-11-14

Brief Summary

This study is the first to give ETD001 to people with CF. The study will be run in two parts. Part A will assess if ETD001 is safe to give to people with CF, and Part B will assess if ETD001 improves lung function. The study drug is taken twice a day, in Part A it is taken for 7 days and in Part B for 28 days. In Part B there will be a separate period where dummy medicine is given for 28 days so the treatments can be compared.

In Part A participants will receive 13 doses of either ETD001 or placebo, 8 people will take part. Participants will take up to 56 days to finish the study and make 5 outpatient visits.

In Part B participants will receive 55 doses of ETD001 and 55 doses of placebo, 32 people will take part. Participants will take up to 140 days to finish the study and will make 8 outpatient visits.

Study assessments include physical examinations, vital signs, heart traces, blood/urine samples, breathing tests and health questionnaires.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Part A - 7 day treatment period - parallel group

Twice daily doses of ETD001/placebo for 6 days, single dose on Day 7

Group Type: EXPERIMENTAL

ETD001

Intervention Type: DRUG

Twice daily doses

Placebo

Intervention Type: DRUG

Twice daily doses

Part B - 2 x 28 day treatment period - crossover

Two treatment periods of twice daily doses of ETD001/placebo for 27 days, single dose on Day 28 separated by a period of 28 days

Group Type: EXPERIMENTAL

ETD001

Intervention Type: DRUG

Twice daily doses

Placebo

Intervention Type: DRUG

Twice daily doses

Interventions

ETD001

Twice daily doses

Intervention Type: DRUG

Placebo

Twice daily doses

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Enterprise Therapeutics Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Renu Gupta, MD

Role: STUDY_DIRECTOR

Enterprise Therapeutics

Locations

Hospices Civils de Lyon

Lyon, , France

CHU de Montpellier

Montpellier, , France

Hôpital Cochin

Paris, , France

Hôpitaux de Toulouse

Toulouse, , France

Charité Universtaetsmedizin

Berlin, , Germany

CF-Studienzentrum Universitätsklinikum Köln

Cologne, , Germany

Westdeutsches Lungenzentrum am Universitätsklinikum

Essen, , Germany

Universitätsklinikum Frankfurt

Frankfurt, , Germany

IKF Pneumologie

Frankfurt, , Germany

LMU Kinikum

Munich, , Germany

Azienda Ospedaliera Universitaria Meyer

Florence, , Italy

IRCCS Istituto Giannina Gaslini

Genova, , Italy

Fondazione IRCCS Ca' Granda- Ospedale Maggiore Policlinico

Milan, , Italy

Ospedale Pediatrico Bambino Gesù

Roma, , Italy

Azienda Ospedaliera Universitaria Integrata Verona

Verona, , Italy

Belfast Health and Social Care Trust

Belfast, , United Kingdom

Royal Papworth Hospital

Cambridge, , United Kingdom

All Wales Adult CF Centre

Cardiff, , United Kingdom

Queen Elizabeth University Hospital West of Scotland CF Service

Glasgow, , United Kingdom

Royal Brompton Hospital

London, , United Kingdom

Southampton General Hospital

Southampton, , United Kingdom

Countries

France; Germany; Italy; United Kingdom

Other Identifiers

ET-ENAC-03

Identifier Type: -

Identifier Source: org_study_id