Cystic FibrosisPhase 1NCT04926701
A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects
A Phase 1 study testing ETD001 single dose, ETD001 multiple twice daily doses and ETD001 multiple once daily doses in people with Cystic Fibrosis, run by Enterprise Therapeutics Ltd at 1 site in United Kingdom.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ETD001 single dose, ETD001 multiple twice daily doses and ETD001 multiple once daily doses
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 98 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 11 Jun 2021to 22 Mar 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- ETD001 single dose, Placebo single dose, ETD001 multiple twice daily doses, Placebo multiple twice daily doses, ETD001 multiple once daily doses
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ETD001 single doseDrug
Single ascending doses of inhaled ETD001
Placebo single doseDrug
Single doses of inhaled placebo
ETD001 multiple twice daily dosesDrug
Ascending doses of inhaled ETD001 administered twice daily
Placebo multiple twice daily dosesDrug
Doses of inhaled placebo administered twice daily
ETD001 multiple once daily dosesDrug
Doses of inhaled ETD001 administered once daily
Placebo multiple once daily dosesDrug
Doses of inhaled placebo administered once daily
| Study group | Role | What they receive |
|---|---|---|
| Single ascending dose | Experimental | Single dose of inhaled ETD001/placebo on one occasion Receives: Drug: ETD001 single dose; Drug: Placebo single dose |
| Multiple ascending dose (7 days) | Experimental | Daily doses of ETD001/placebo for 7 consecutive days Receives: 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 Receives: Drug: ETD001 multiple twice daily doses; Drug: Placebo multiple twice daily doses |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants reporting one or more treatment emergent adverse event (TEAE)
Measured: Baseline to Week 8
Number of participants who discontinue due to an adverse event (AE)
Measured: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for 12-lead ECG assessment at least once post dose
Measured: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose
Measured: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for laboratory assessments at least once post dose
Measured: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose
Measured: Baseline to Week 8
Secondary outcomes (1)
Plasma concentrations of ETD001
Blood levels of ETD001 measured after dosing
Measured: Day 1 pre-dose and at multiple time-points (up to 14 days) post final dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2021
- Study start11 Jun 2021
- Primary completion22 Mar 2022
- Study completion22 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 98 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enterprise Therapeutics Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Niyati Prasad, MD, Study director, Enterprise Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04926701
- Sponsor's protocol ID
- ET-ENAC-01
- Status last verified
- Mar 2022
- First submitted
- 10 Jun 2021
- Last updated
- 24 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04926701
- Acronym / sponsor ID
- ET-ENAC-01
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 98 participants (actual)
- Lead sponsor
- Enterprise Therapeutics LtdIndustry (pharma, biotech, devices)
- Study start
- 11 Jun 2021 (actual)
- Primary completion
- 22 Mar 2022 (actual)
- Study completion
- 22 Mar 2022 (actual)
- First posted
- 15 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Single ascending dose | Experimental | Drug: ETD001 single dose, Drug: Placebo single doseSingle dose of inhaled ETD001/placebo on one occasion |
| Multiple ascending dose (7 days) | Experimental | Drug: ETD001 multiple twice daily doses, Drug: Placebo multiple twice daily doses, Drug: ETD001 multiple once daily doses, Drug: Placebo multiple once daily dosesDaily doses of ETD001/placebo for 7 consecutive days |
| Multiple ascending dose (14 days) | Experimental | Drug: ETD001 multiple twice daily doses, Drug: Placebo multiple twice daily dosesDaily doses of ETD001/placebo for 14 consecutive days |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ETD001 single doseSingle ascending doses of inhaled ETD001 | Drug | Single ascending dose |
| Placebo single doseSingle doses of inhaled placebo | Drug | Single ascending dose |
| ETD001 multiple twice daily dosesAscending doses of inhaled ETD001 administered twice daily | Drug | Multiple ascending dose (14 days), Multiple ascending dose (7 days) |
| Placebo multiple twice daily dosesDoses of inhaled placebo administered twice daily | Drug | Multiple ascending dose (14 days), Multiple ascending dose (7 days) |
| ETD001 multiple once daily dosesDoses of inhaled ETD001 administered once daily | Drug | Multiple ascending dose (7 days) |
| Placebo multiple once daily dosesDoses of inhaled placebo administered once daily | Drug | Multiple ascending dose (7 days) |
Primary outcome
Number of participants reporting one or more treatment emergent adverse event (TEAE)
Time frame: Baseline to Week 8
Number of participants who discontinue due to an adverse event (AE)
Time frame: Baseline to Week 8
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
Number of participants who meet the markedly abnormal criteria for vital signs assessments at least once post dose
Time frame: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for laboratory assessments at least once post dose
Time frame: Baseline to Week 8
Number of participants who meet the markedly abnormal criteria for spirometry assessments at least once post dose
Time frame: Baseline to Week 8
1 secondary outcome
Plasma concentrations of ETD001
Time frame: Day 1 pre-dose and at multiple time-points (up to 14 days) post final dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Niyati Prasad, MD study director
Enterprise Therapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ETD001 single dose, ETD001 multiple twice daily doses and ETD001 multiple once daily doses in people with Cystic Fibrosis, run by Enterprise Therapeutics Ltd at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants reporting one or more treatment emergent adverse event (TEAE) (time frame: Baseline to Week 8). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2022.