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

NCT04926701 | Completed Phase 1

• 1 other identifier: ET-ENAC-01
• Study Type: interventional
• Target: 98
• Locations: 1 country
• Sites: 1

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.

Conditions

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 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 ETD001

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

Study Arms (3)

Single ascending dose

EXPERIMENTAL

Single dose of inhaled ETD001/placebo on one occasion

Drug: ETD001 single dose; Drug: Placebo single dose

Multiple ascending dose (7 days)

EXPERIMENTAL

Daily doses of ETD001/placebo for 7 consecutive days

Drug: ETD001 multiple twice daily doses; Drug: Placebo multiple twice daily doses; Drug: ETD001 multiple once daily doses; Drug: Placebo multiple once daily doses

Multiple ascending dose (14 days)

EXPERIMENTAL

Daily doses of ETD001/placebo for 14 consecutive days

Drug: ETD001 multiple twice daily doses; Drug: Placebo multiple twice daily doses

Interventions

ETD001 single dose DRUG

Single ascending doses of inhaled ETD001

Single ascending dose

Placebo single dose DRUG

Single doses of inhaled placebo

Single ascending dose

ETD001 multiple twice daily doses DRUG

Ascending doses of inhaled ETD001 administered twice daily

Multiple ascending dose (14 days); Multiple ascending dose (7 days)

Placebo multiple twice daily doses DRUG

Doses of inhaled placebo administered twice daily

Multiple ascending dose (14 days); Multiple ascending dose (7 days)

ETD001 multiple once daily doses DRUG

Doses of inhaled ETD001 administered once daily

Multiple ascending dose (7 days)

Placebo multiple once daily doses DRUG

Doses of inhaled placebo administered once daily

Multiple ascending dose (7 days)

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Enterprise Therapeutics Ltd: lead

Study Sites (1)

Hammersmith Medicines Research

London, NW10 7EW, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

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

Study Officials

• Niyati Prasad, 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: June 10, 2021
• First Posted: June 15, 2021
• Study Start: June 11, 2021
• Primary Completion: March 22, 2022
• Study Completion: March 22, 2022
• Last Updated: March 24, 2022

Record last verified: 2022-03

Locations

GB (1)