Safety and efficacy study of repeated inhaled doses of ETD001 in people with cystic fibrosis

• Research type

  Research Study

• Full 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

• IRAS ID

  1008079

• Contact name

  Paul Russell

• Contact email

  paul@enterprisetherapeutics.com

• Sponsor organisation

  Enterprise Therapeutics Ltd,

• ISRCTN Number

  Not available

• Clinicaltrials.gov Identifier

  Not available

• Research summary

  Cystic fibrosis (CF) is an inherited disorder that causes sticky mucus to build up in the lungs and digestive system. Current treatments help control the symptoms, but they don’t work for everyone, and some treatments are burdensome. The study drug (called ETD001) works by loosening the mucus in the lungs so it is less sticky making breathing easier and helping to reduce chest infections. ETD001 is given using a device called a nebuliser that turns the liquid medicine into a mist so that it may be breathed in.
  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. Most of the study medicine will be taken at home.

  Thirty people are needed to complete the study, the study will be run at sites in the United Kingdom, France and Germany.

  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, 22 people will take part. Participants will take up to 133 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.

• REC name

  East Midlands - Leicester South Research Ethics Committee

• REC reference

  23/EM/0180

• Date of REC Opinion

  14 Sep 2023

• REC opinion

  Further Information Favourable Opinion