A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD002 in Healthy Subjects

A First in Human, Randomised, Double Blind, Placebo-controlled, Three-part Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses (SAD & MAD) of Inhaled ETD002 in Healthy Male and Female Subjects

This is a first in human study of ETD002 a new drug being developed for the treatment of cystic fibrosis.The study is a randomised, double-blind, placebo-controlled, interventional study to assess the safety and tolerability of ascending single and repeat doses of inhaled ETD002 in healthy male and female subjects.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: ETD002 - single dose; Drug: Placebo - single dose; Drug: ETD002 - 7 day repeat dose; Drug: Placebo - 7 day repeat dose; Drug: ETD002 - 14 day repeat dose; Drug: Placebo - 14 day repeat dose; Drug: Salbutamol

• Study Type: Interventional
• Enrollment (Actual): 80
• 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: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Males & females using suitable methods of contraception or females of non-childbearing potential
• Consent to study participation
• Body weight ≥ 50 kg and body mass index within the range 19 - 30 kg/m2
• Vital signs assessments within normal ranges
• Healthy as determined following physical examination at screening visit
• Spirometry readings (FEV1 and FVC) to be ≥ 80% of predicted value

Exclusion Criteria:

• Acute or chronic illness detected at screening visit
• Respiratory tract infection within 4 weeks of the screening visit
• Use of prescription or OTC medication within 14 days of the screening visit
• History of regular alcohol consumption over recommended limits within 6 months of the study, or history/evidence of alcohol or drug abuse
• Smoking or use of tobacco products within 6 months of screening
• Abnormal blood/urine laboratory screening test results
• 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 Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single ascending dose	Drug: ETD002 - single dose; Single ascending doses of ETD002; Drug: Placebo - single dose; Single doses of placebo
Experimental: Repeat dose - 7 days	Drug: ETD002 - 7 day repeat dose; Twice daily doses of ETD002 for 7 days; Drug: Placebo - 7 day repeat dose; Twice daily doses of Placebo for 7 days
Experimental: Repeat dose - 14 days	Drug: ETD002 - 14 day repeat dose; Twice daily doses of ETD002 for 14 days; Drug: Placebo - 14 day repeat dose; Twice daily doses of Placebo for 14 days
Experimental: Repeat dose - 7 days with SABA	Drug: ETD002 - 7 day repeat dose; Twice daily doses of ETD002 for 7 days; Drug: Placebo - 7 day repeat dose; Twice daily doses of Placebo for 7 days; Drug: Salbutamol; Twice daily doses of salbutamol for 3 days (Days 5, 6 & 7)

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 safety 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 ETD002	Blood levels of ETD002 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: David Morris, MD, Enterprise Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): August 13, 2020
• Primary Completion (Actual): May 19, 2021
• Study Completion (Actual): May 19, 2021

Study Registration Dates

• First Submitted: July 23, 2020
• First Submitted That Met QC Criteria: July 23, 2020
• First Posted (Actual): July 28, 2020

Study Record Updates

• Last Update Posted (Actual): May 21, 2021
• Last Update Submitted That Met QC Criteria: May 20, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Keywords: Cystic fibrosis
• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Autonomic Agents; Peripheral Nervous System Agents; Adrenergic Agonists; Bronchodilator Agents; Anti-Asthmatic Agents; Respiratory System Agents; Reproductive Control Agents; Adrenergic beta-2 Receptor Agonists; Adrenergic beta-Agonists; Tocolytic Agents; Albuterol
• Other Study ID Numbers: ET-TMEM-01; 2020-001080-92 (EudraCT Number)

Drug and device information, study documents

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