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

Launched by ENTERPRISE THERAPEUTICS LTD · Jun 24, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new inhaled medication called ETD001 in people with cystic fibrosis (CF). The study has two parts. In the first part, researchers want to see if ETD001 is safe for people with CF by giving it to a small group of participants for 7 days. In the second part, they will check if taking ETD001 for 28 days helps improve lung function compared to a placebo, which is a dummy medicine that looks like the real drug but has no active ingredients. The total study duration for participants can be up to 140 days, with several visits to the clinic for health assessments, including breathing tests and physical exams.

To join the study, participants need to be at least 18 years old and have a confirmed diagnosis of cystic fibrosis. They should also be in stable health and able to perform certain breathing tests. Some people may not be eligible if they have certain health issues or have recently changed their CF treatments. This trial is a great opportunity for those who meet the criteria and are interested in helping researchers learn more about potential new treatments for cystic fibrosis.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * 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
• Exclusion Criteria:
• • 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