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

NCT04488705 | Completed Phase 1

• 2 other identifiers: ET-TMEM-012020-001080-92
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy

Keywords

Cystic fibrosis

Outcome Measures

Primary Outcomes (6)

• 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 Outcomes (1)

• Plasma concentrations of ETD002

  Day 1 pre-dose and at multiple time points (up to 14 days) post final dose

Study Arms (4)

Single ascending dose

EXPERIMENTAL

Drug: ETD002 - single dose; Drug: Placebo - single dose

Repeat dose - 7 days

EXPERIMENTAL

Drug: ETD002 - 7 day repeat dose; Drug: Placebo - 7 day repeat dose

Repeat dose - 14 days

EXPERIMENTAL

Drug: ETD002 - 14 day repeat dose; Drug: Placebo - 14 day repeat dose

Repeat dose - 7 days with SABA

EXPERIMENTAL

Drug: ETD002 - 7 day repeat dose; Drug: Placebo - 7 day repeat dose; Drug: Salbutamol

Interventions

ETD002 - single dose DRUG

Single ascending doses of ETD002

Single ascending dose

Placebo - single dose DRUG

Single doses of placebo

Single ascending dose

ETD002 - 7 day repeat dose DRUG

Twice daily doses of ETD002 for 7 days

Repeat dose - 7 days; Repeat dose - 7 days with SABA

Placebo - 7 day repeat dose DRUG

Twice daily doses of Placebo for 7 days

Repeat dose - 7 days; Repeat dose - 7 days with SABA

ETD002 - 14 day repeat dose DRUG

Twice daily doses of ETD002 for 14 days

Repeat dose - 14 days

Placebo - 14 day repeat dose DRUG

Twice daily doses of Placebo for 14 days

Repeat dose - 14 days

Salbutamol DRUG

Twice daily doses of salbutamol for 3 days (Days 5, 6 \& 7)

Repeat dose - 7 days with SABA

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Enterprise Therapeutics Ltd: lead

Study Sites (1)

Hammersmith Medicines Research

London, NW10 7EW, United Kingdom

Location

Related Publications (1)

• Miller P, Repplinger D, Zhu R, Chen Y, Lewin-Koh N, Morris D, Russell P, She G, Singh N, White R, Wilkes D, Chen H, Galanter J. Randomized, phase I studies to evaluate the safety, tolerability, and pharmacokinetics of an inhaled, TMEM16A potentiator, GDC-6988, in healthy subjects. Ther Adv Respir Dis. 2025 Jan-Dec;19:17534666251393346. doi: 10.1177/17534666251393346. Epub 2025 Nov 29.

  PMID: 41318901 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Albuterol

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Phenethylamines; Ethylamines

Study Officials

• David Morris, MD

  Enterprise Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 23, 2020
• First Posted: July 28, 2020
• Study Start: August 13, 2020
• Primary Completion: May 19, 2021
• Study Completion: May 19, 2021
• Last Updated: May 21, 2021

Record last verified: 2021-05

Locations

GB (1)