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Cystic FibrosisPhase 2NCT06478706

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

A Phase 2 study testing ETD001 in people with Cystic Fibrosis, run by Enterprise Therapeutics Ltd at 21 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 99 years
Conditions the study is for
Cystic Fibrosis
What is being tested?
ETD001
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
57 participants
Where
21 sitesin 4 countries
Study dates
26 Jun 2024to 14 Nov 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 99 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

21 sites in 4 countries.

21 sites in 4 countries

Germany6 sites

  • Charité Universtaetsmedizin

    Berlin, 13353

  • CF-Studienzentrum Universitätsklinikum Köln

    Cologne, 50924

  • Westdeutsches Lungenzentrum am Universitätsklinikum

    Essen, 45239

  • Universitätsklinikum Frankfurt

    Frankfurt, 60590

  • IKF Pneumologie

    Frankfurt, 60596

  • LMU Kinikum

    Munich, 80336

Other trials in Germany

United Kingdom6 sites

  • Belfast Health and Social Care Trust

    Belfast, BT9 7AB

  • Royal Papworth Hospital

    Cambridge, CB2 0AY

  • All Wales Adult CF Centre

    Cardiff, CF64 2XX

  • Queen Elizabeth University Hospital West of Scotland CF Service

    Glasgow, G51 4TR

  • Royal Brompton Hospital

    London, SW3 6LL

  • Southampton General Hospital

    Southampton, SO16 6YD

Other trials in United Kingdom

Italy5 sites

  • Azienda Ospedaliera Universitaria Meyer

    Florence, 50139

  • IRCCS Istituto Giannina Gaslini

    Genova, 16147

  • Fondazione IRCCS Ca' Granda- Ospedale Maggiore Policlinico

    Milan, 20122

  • Ospedale Pediatrico Bambino Gesù

    Roma, 00165

  • Azienda Ospedaliera Universitaria Integrata Verona

    Verona, 37126

Other trials in Italy

France4 sites

  • Hospices Civils de Lyon

    Lyon, 69495

  • CHU de Montpellier

    Montpellier, 34295

  • Hôpital Cochin

    Paris, 75014

  • Hôpitaux de Toulouse

    Toulouse, 31059

Other trials in France

About this study

Official title: 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 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.

Read more

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
Being tested
ETD001, Placebo
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ETD001Drug

    Twice daily doses

  • PlaceboDrug

    Twice daily doses

Study groups in NCT06478706
Study groupRoleWhat they receive
Part A - 7 day treatment period - parallel groupExperimentalTwice daily doses of ETD001/placebo for 6 days, single dose on Day 7
Receives: Drug: ETD001; Drug: Placebo
Part B - 2 x 28 day treatment period - crossoverExperimentalTwo treatment periods of twice daily doses of ETD001/placebo for 27 days, single dose on Day 28 separated by a period of 28 days
Receives: Drug: ETD001; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 28 days

  2. 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

    Measured: 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)

  1. Part A: Characterisation of plasma pharmacokinetics (PK)

    Peak plasma concentration and time observed (Cmax \& Tmax) of ETD001 following dosing.

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

  2. Part A: Characterisation of plasma pharmacokinetics (PK)

    Area under the concentration versus time curve from time 0 to last quantifiable concentration (AUC(0-t)) of ETD001 following dosing.

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

  3. Part A: Characterisation of plasma pharmacokinetics (PK)

    Area under the concentration versus time curve within a dosing interval (AUC(0-tau)) of ETD001 following dosing.

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

  4. Part A: Characterisation of plasma pharmacokinetics (PK)

    Area under the concentration versus time curve from time 0 to infinity (AUC(inf)) of ETD001 following dosing.

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

  5. Part A: Characterisation of plasma pharmacokinetics (PK)

    Apparent terminal rate constant and apparent terminal half life (λz, T1/2) of ETD001 following dosing.

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

  6. Part A: Characterisation of urine PK

    Amount ETD001 excreted in urine (Ae)

    Measured: Day 1 (0 - 6 hours)

  7. Part A: Characterisation of urine PK

    Fraction of ETD001 dose excreted (Fe)

    Measured: Day 1 (0 - 6 hours)

  8. Part A: Characterisation of urine PK

    Renal clearance of ETD001 (CLr)

    Measured: Day 1 (0 - 6 hours)

  9. Part B: Effect of repeat inhaled doses of ETD001 on other lung function assessments

    Relative change in ppFEV1, absolute change in FVC, FEV1/FVC ratio and FEF25-75 measured by spirometry, from baseline to Day 28 (for either Treatment Period 1 or Treatment Period 2), compared to placebo

    Measured: Treatment Period 1 & 2; Day 1 (pre-dose, 1, 2 & 4 hours), Day 14 (pre-dose, 1 & 2 hours), Day 28 (pre-dose, 1, 2 & 4 hours)

  10. Part B: 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

    Measured: 105 days

  11. Part B: Effect of repeat inhaled doses of ETD001 on the quality of life questionnaire, the Cystic Fibrosis Questionnaire (revised) (CFQ-R)

    Change in CFQ-R (respiratory domain) from baseline to Day 28 (for either Treatment Period 1 or Treatment Period 2), compared to placebo

    Measured: Treatment Period 1 & 2; Day 1 & Day 28 (pre-dose)

  12. Part B: Characterisation of plasma PK

    Population PK characteristics and model generated individual PK parameters (Cmax \& Tmax)

    Measured: Treatment Period 1 & 2; Day 1 (0 & 1 hour), Day 14 (0 & 2 hours), Day 28 (0 & 4 hours), Follow up (Day 105) 1 sample

  13. Part B: Characterisation of plasma PK

    Population PK characteristics and model generated individual PK parameters (AUC(0-t))

    Measured: Treatment Period 1 & 2; Day 1 (0 & 1 hour), Day 14 (0 & 2 hours), Day 28 (0 & 4 hours), Follow up (Day 105) 1 sample

  14. Part B: Characterisation of plasma PK

    Population PK characteristics and model generated individual PK parameters (AUC(0-tau))

    Measured: Treatment Period 1 & 2; Day 1 (0 & 1 hour), Day 14 (0 & 2 hours), Day 28 (0 & 4 hours), Follow up (Day 105) 1 sample

  15. Part B: Characterisation of plasma PK

    Population PK characteristics and model generated individual PK parameters (AUC(0-inf))

    Measured: Treatment Period 1 & 2; Day 1 (0 & 1 hour), Day 14 (0 & 2 hours), Day 28 (0 & 4 hours), Follow up (Day 105) 1 sample

  16. Part B: Characterisation of plasma PK

    Population PK characteristics and model generated individual PK parameters (λz, T1/2)

    Measured: Treatment Period 1 & 2; Day 1 (0 & 1 hour), Day 14 (0 & 2 hours), Day 28 (0 & 4 hours), Follow up (Day 105) 1 sample

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jun 2024
  2. Study start26 Jun 2024
  3. Primary completion14 Nov 2025
  4. Study completion14 Nov 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
57 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06478706
Sponsor's protocol ID
ET-ENAC-03
Status last verified
Nov 2025
First submitted
18 Jun 2024
Last updated
28 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ETD001 in people with Cystic Fibrosis, run by Enterprise Therapeutics Ltd at 21 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in Germany, United Kingdom, Italy, France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Safety and tolerability of repeat inhaled doses of ETD001 monitored by assessment of adverse events (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06478706.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2025.

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Clinical Trial NCT06478706 | AzTrials