NCT06478706

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Enterprise Therapeutics Ltd · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	ETD001	Twice daily doses
DRUG	Placebo	Twice daily doses

Timeline

• Start date: 2024-06-26
• Primary completion: 2025-11-14
• Completion: 2025-11-14
• First posted: 2024-06-27
• Last updated: 2025-11-28

Locations

21 sites across 4 countries: France, Germany, Italy, United Kingdom