Cystic Fibrosis
Conditions
Brief summary
This is a first in human study of ETD001 a new drug being developed for the treatment of cystic fibrosis. The study is a randomised, placebo-controlled, double-blind interventional study to assess the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled ETD001in healthy male and female subjects.
Interventions
DRUGETD001 single dose
Single ascending doses of inhaled ETD001
Single doses of inhaled placebo
DRUGETD001 multiple twice daily doses
Ascending doses of inhaled ETD001 administered twice daily
DRUGPlacebo multiple twice daily doses
Doses of inhaled placebo administered twice daily
DRUGETD001 multiple once daily doses
Doses of inhaled ETD001 administered once daily
DRUGPlacebo multiple once daily doses
Doses of inhaled placebo administered once daily
Sponsors
Enterprise Therapeutics Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
Yes
Inclusion criteria
* Males and females using suitable methods of contraception, or females of non-childbearing potential * Consent to study participation * Body weight ≥ 50 kg and body mass index 19 - 30 kg/m2 * Vital sign assessments within the normal ranges * Healthy as determined following physical and laboratory examinations at screening visit * Spirometry readings (FEV1 and FVC) to be ≥ 80% of predicted value
Exclusion criteria
* Acute or chronic illness detected at the screening visit * Respiratory tract infection within 4 weeks of the screening visit * Use of prescribed or OTC medication within 14 days of the screening visit * History of regular alcohol use over the recommended limits within 6 months of screening, or history/evidence of alcohol or drug abuse * Smoker or use of tobacco products within 6 months of screening * Abnormal blood or urine laboratory test results at screening * Recent participation (within 3 months) in another clinical trial * Current, or history of, allergy that may be contraindicated
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants who meet the markedly abnormal criteria for 12-lead ECG assessment at least once post dose | Baseline to Week 8 |
| Number of participants reporting one or more treatment emergent adverse event (TEAE) | Baseline to Week 8 |
| Number of participants who discontinue due to an adverse event (AE) | Baseline to Week 8 |
| Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose | Baseline to Week 8 |
| Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose | Baseline to Week 8 |
| Number of participants who meet the markedly abnormal criteria for laboratory assessments at least once post dose | Baseline to Week 8 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma concentrations of ETD001 | Day 1 pre-dose and at multiple time-points (up to 14 days) post final dose | Blood levels of ETD001 measured after dosing |
Countries
United Kingdom
Outcome results
None listed