A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects

Study Overview
=================
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.

Official Title
-----------------
A First in Human Randomised, Double Blind, Placebo-controlled, Two-part Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses (SAD and MAD) of Inhaled ETD001 in Healthy Male and Female Subjects

Conditions
-----------------
Cystic Fibrosis

Intervention / Treatment
-----------------
* Drug: ETD001 single dose
* Drug: Placebo single dose
* Drug: ETD001 multiple twice daily doses
* Drug: Placebo multiple twice daily doses
* Drug: ETD001 multiple once daily doses
* Drug: Placebo multiple once daily doses


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
Accepts Healthy Volunteers

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single ascending dose<br>Single dose of inhaled ETD001/placebo on one occasion | Drug: ETD001 single dose<br>* Single ascending doses of inhaled ETD001<br>Drug: Placebo single dose<br>* Single doses of inhaled placebo<br>|
| Experimental: Multiple ascending dose (7 days)<br>Daily doses of ETD001/placebo for 7 consecutive days | Drug: ETD001 multiple twice daily doses<br>* Ascending doses of inhaled ETD001 administered twice daily<br>Drug: Placebo multiple twice daily doses<br>* Doses of inhaled placebo administered twice daily<br>Drug: ETD001 multiple once daily doses<br>* Doses of inhaled ETD001 administered once daily<br>Drug: Placebo multiple once daily doses<br>* Doses of inhaled placebo administered once daily<br>|
| Experimental: Multiple ascending dose (14 days)<br>Daily doses of ETD001/placebo for 14 consecutive days | Drug: ETD001 multiple twice daily doses<br>* Ascending doses of inhaled ETD001 administered twice daily<br>Drug: Placebo multiple twice daily doses<br>* Doses of inhaled placebo administered twice daily<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 12-lead ECG assessment 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 | 
| Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose |  | Baseline to Week 8 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma concentrations of ETD001 | Blood levels of ETD001 measured after dosing | Day 1 pre-dose and at multiple time-points (up to 14 days) post final dose |