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

NCT06478706 | Completed Phase 2 DMC

• 1 other identifier: ET-ENAC-03
• Study Type: interventional
• Target: 57
• Locations: 4 countries
• Sites: 21

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

Outcome Measures

Primary Outcomes (2)

• Part A: Safety and tolerability of repeat inhaled doses of ETD001 monitored by assessment of adverse events

  Incidence of treatment emergent adverse events(AE)/serious AE), withdrawals due to AE

  28 days

• Part B: Effect of repeat inhaled doses of ETD001 on percent predicted forced expiratory volume in 1 second (ppFEV1)

  Change in ppFEV1 measured by spirometry from baseline to Day 28 (for either Treatment Period 1 or Treatment Period 2), compared to placebo

  Treatment Period 1 & 2 - Day 1 (pre-dose, 1, 2 & 4 hours post dose), Day 14 (pre-dose, 1 & 2 hours post), Day 28 (at 0, 1, 2 & 4 hours post dose)

Secondary Outcomes (16)

• Part A: Characterisation of plasma pharmacokinetics (PK)

  Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours)

• Part A: Characterisation of plasma pharmacokinetics (PK)

  Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours, Day 7 (pre-dose), Day 28 (single sample)

• Part A: Characterisation of plasma pharmacokinetics (PK)

  Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours, Day 7 (pre-dose)

• Part A: Characterisation of plasma pharmacokinetics (PK)

  Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours, Day 7 (pre-dose) Day 28 (single sample)

• Part A: Characterisation of plasma pharmacokinetics (PK)

  Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours, Day 7 (pre-dose), Day 28 (single sample)

Study Arms (2)

Part A - 7 day treatment period - parallel group

EXPERIMENTAL

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

Drug: ETD001; Drug: Placebo

Part B - 2 x 28 day treatment period - crossover

EXPERIMENTAL

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

Drug: ETD001; Drug: Placebo

Interventions

ETD001 DRUG

Twice daily doses

Part A - 7 day treatment period - parallel group; Part B - 2 x 28 day treatment period - crossover

Placebo DRUG

Twice daily doses

Part A - 7 day treatment period - parallel group; Part B - 2 x 28 day treatment period - crossover

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Enterprise Therapeutics Ltd: lead

Study Sites (21)

Hospices Civils de Lyon

Lyon, 69495, France

Location

CHU de Montpellier

Montpellier, 34295, France

Location

Hôpital Cochin

Paris, 75014, France

Location

Hôpitaux de Toulouse

Toulouse, 31059, France

Location

Charité Universtaetsmedizin

Berlin, 13353, Germany

Location

CF-Studienzentrum Universitätsklinikum Köln

Cologne, 50924, Germany

Location

Westdeutsches Lungenzentrum am Universitätsklinikum

Essen, 45239, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt, 60590, Germany

Location

IKF Pneumologie

Frankfurt, 60596, Germany

Location

LMU Kinikum

Munich, 80336, Germany

Location

Azienda Ospedaliera Universitaria Meyer

Florence, 50139, Italy

Location

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

Location

Fondazione IRCCS Ca' Granda- Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

Azienda Ospedaliera Universitaria Integrata Verona

Verona, 37126, Italy

Location

Belfast Health and Social Care Trust

Belfast, BT9 7AB, United Kingdom

Location

Royal Papworth Hospital

Cambridge, CB2 0AY, United Kingdom

Location

All Wales Adult CF Centre

Cardiff, CF64 2XX, United Kingdom

Location

Queen Elizabeth University Hospital West of Scotland CF Service

Glasgow, G51 4TR, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6LL, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Renu Gupta, MD

  Enterprise Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2024
• First Posted: June 27, 2024
• Study Start: June 26, 2024
• Primary Completion: November 14, 2025
• Study Completion: November 14, 2025
• Last Updated: November 28, 2025

Record last verified: 2025-11

Locations

GB (6); FR (4); DE (6); IT (5)