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

Sponsor

Enterprise Therapeutics Ltd (Industry)

Overall Status

Completed

CT.gov ID

NCT04926701

Collaborator

(none)

Enrollment

Location

Arms

9.3

Actual Duration (Months)

10.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	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	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

98 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, 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

Actual Study Start Date :

Jun 11, 2021

Actual Primary Completion Date :

Mar 22, 2022

Actual Study Completion Date :

Mar 22, 2022

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

Plasma concentrations of ETD001 [Day 1 pre-dose and at multiple time-points (up to 14 days) post final dose]
Blood levels of ETD001 measured after dosing

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 50 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hammersmith Medicines Research	London		United Kingdom	NW10 7EW

Sponsors and Collaborators

Enterprise Therapeutics Ltd

Investigators

Study Director: Niyati Prasad, MD, Enterprise Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Enterprise Therapeutics Ltd

ClinicalTrials.gov Identifier:

NCT04926701

Other Study ID Numbers:

ET-ENAC-01

First Posted:

Jun 15, 2021

Last Update Posted:

Mar 24, 2022

Last Verified:

Mar 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Cystic Fibrosis

Study Results

No Results Posted as of Mar 24, 2022