A Phase 2 interventional study of ETD001 and Placebo in Cystic Fibrosis, sponsored by Enterprise Therapeutics Ltd. Completed at 21 sites in 4 countries. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2025-11-28.
Sponsored by Enterprise Therapeutics Ltd · Phase 2, Interventional, and Treatment
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 57 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Enterprise Therapeutics Ltd is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
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;
Exclusion Criteria:
Twice daily doses of ETD001/placebo for 6 days, single dose on Day 7
Drug: ETD001 · Drug: Placebo
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
Twice daily doses
Twice daily doses
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
Time frame: 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
Time frame: 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)
Part A: Characterisation of plasma pharmacokinetics (PK)
Peak plasma concentration and time observed (Cmax \& Tmax) of ETD001 following dosing.
Time frame: Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours)
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.
Time frame: 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)
Area under the concentration versus time curve within a dosing interval (AUC(0-tau)) of ETD001 following dosing.
Time frame: Day 1 (pre-dose, 0.25, 0.5, 1, 2, 4 & 6 hours, Day 7 (pre-dose)
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.
Time frame: 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)
Apparent terminal rate constant and apparent terminal half life (λz, T1/2) of ETD001 following dosing.
Time frame: 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 urine PK
Amount ETD001 excreted in urine (Ae)
Time frame: Day 1 (0 - 6 hours)
Part A: Characterisation of urine PK
Fraction of ETD001 dose excreted (Fe)
Time frame: Day 1 (0 - 6 hours)
Part A: Characterisation of urine PK
Renal clearance of ETD001 (CLr)
Time frame: Day 1 (0 - 6 hours)
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
Time frame: 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)
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
Time frame: 105 days
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
Time frame: Treatment Period 1 & 2; Day 1 & Day 28 (pre-dose)
Part B: Characterisation of plasma PK
Population PK characteristics and model generated individual PK parameters (Cmax \& Tmax)
Time frame: 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
Part B: Characterisation of plasma PK
Population PK characteristics and model generated individual PK parameters (AUC(0-t))
Time frame: 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
Part B: Characterisation of plasma PK
Population PK characteristics and model generated individual PK parameters (AUC(0-tau))
Time frame: 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
Part B: Characterisation of plasma PK
Population PK characteristics and model generated individual PK parameters (AUC(0-inf))
Time frame: 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
Part B: Characterisation of plasma PK
Population PK characteristics and model generated individual PK parameters (λz, T1/2)
Time frame: 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
This study is completed, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.
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Enterprise Therapeutics Ltd