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

• ClinicalTrials.gov Identifier: NCT04488705
• Recruitment Status: Completed
• First Posted: July 28, 2020
• Last Update Posted: May 21, 2021
• Sponsor: Enterprise Therapeutics Ltd
• Information provided by (Responsible Party): Enterprise Therapeutics Ltd

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Healthy	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	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: August 13, 2020
• Actual Primary Completion Date: May 19, 2021
• Actual Study Completion Date: May 19, 2021

Arms and Interventions

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)

Outcome Measures

Primary Outcome Measures :

1. Number of participants reporting one or more treatment emergent adverse event (TEAE) [ Time Frame: Baseline to Week 8 ]
2. Number of participants who discontinue due to an adverse event (AE) [ Time Frame: Baseline to Week 8 ]
3. Number of participants who meet the markedly abnormal criteria for 12-lead ECG assessment at least once post dose [ Time Frame: Baseline to Week 8 ]
4. Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose [ Time Frame: Baseline to Week 8 ]
5. Number of participants who meet the markedly abnormal criteria for safety laboratory assessments at least once post dose [ Time Frame: Baseline to Week 8 ]
6. Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose [ Time Frame: Baseline to Week 8 ]

Secondary Outcome Measures :

1. Plasma concentrations of ETD002 [ Time Frame: Day 1 pre-dose and at multiple time points (up to 14 days) post final dose ]
   Blood levels of ETD002 measured after dosing

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 50 Years (Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Criteria

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

Contacts and Locations

Locations

United Kingdom

Hammersmith Medicines Research

London, United Kingdom, NW10 7EW

Sponsors and Collaborators

Enterprise Therapeutics Ltd

Investigators

• Study Director: David Morris, MD; Enterprise Therapeutics

More Information

• Responsible Party: Enterprise Therapeutics Ltd
• ClinicalTrials.gov Identifier: NCT04488705
• Other Study ID Numbers: ET-TMEM-01; 2020-001080-92 ( EudraCT Number )
• First Posted: July 28, 2020
• Last Update Posted: May 21, 2021
• Last Verified: May 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Enterprise Therapeutics Ltd:

Cystic fibrosis

Additional relevant MeSH terms:

Albuterol; Bronchodilator Agents; Autonomic Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Anti-Asthmatic Agents; Respiratory System Agents; Tocolytic Agents; Reproductive Control Agents; Adrenergic beta-2 Receptor Agonists; Adrenergic beta-Agonists; Adrenergic Agonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action