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

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

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.

Study Overview

• Status: Completed
• 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

• Study Type: Interventional
• Enrollment (Actual): 98
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, NW10 7EW
    • Hammersmith Medicines Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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 spirometry 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 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

Collaborators and Investigators

• Sponsor: Enterprise Therapeutics Ltd
• Investigators: Study Director: Niyati Prasad, MD, Enterprise Therapeutics

Study record dates

Study Major Dates

• Study Start (ACTUAL): June 11, 2021
• Primary Completion (ACTUAL): March 22, 2022
• Study Completion (ACTUAL): March 22, 2022

Study Registration Dates

• First Submitted: June 10, 2021
• First Submitted That Met QC Criteria: June 10, 2021
• First Posted (ACTUAL): June 15, 2021

Study Record Updates

• Last Update Posted (ACTUAL): March 24, 2022
• Last Update Submitted That Met QC Criteria: March 23, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Cystic Fibrosis
• Other Study ID Numbers: ET-ENAC-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No