HealthyPhase 1NCT04889690
Study of Multiple Doses of Danicopan in Healthy Participants
A Phase 1 study testing Danicopan in people with Healthy, run by Alexion Pharmaceuticals, Inc. at 1 site in New Zealand.
Trial summary
This was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (75 milligrams [mg], 200 mg, 500 mg, 800 mg) and dose-matched placebo were administered under fasted conditions. Read more about this study
- Who is it for?
- Ages 25 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy
- What is being tested?
- Danicopan
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 1 siteNew Zealand
- Study dates
- 21 May 2016to 11 Jan 2017
- 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 25 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy was defined as having no clinically relevant abnormalities identified by a detailed medical history, physical exam, blood pressure and heart rate measurements, 12-lead electrocardiogram, and clinical laboratory tests.
- Body mass index of 18 to 30 kilograms (kg)/meter squared with a minimum body weight of 50 kg.
Who cannot join
- History or clinically relevant evidence of significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease.
- Any condition possibly affecting drug absorption (including gastrectomy and cholecystectomy).
- Body temperature greater than or equal to 38°Celcius on Day -1 or Day 1, Hour 0; history of febrile illness or other evidence of infection within 14 days prior to first study drug administration.
- Current tobacco/nicotine user; consumption of any alcohol within 72 hours before first study drug administration or have a history of regular alcohol consumption exceeding 21 drinks/week within 6 months of screening; positive urine drug screen at screening or Day -1.
- Clinically significant laboratory abnormalities at either Screening or Day -1.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multiple Ascending Dose Study To Assess Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of ACH-0144471 In Healthy Volunteers
This was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (75 milligrams [mg], 200 mg, 500 mg, 800 mg) and dose-matched placebo were administered under fasted conditions.
- Conditions
- Healthy
- Being tested
- Danicopan, Placebo
- Keywords
- Danicopan, ALXN2040, ACH-0144471, Factor D Inhibitor, Pharmacokinetics, Pharmacodynamics, Ascending Dose
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Also called ACH-0144471 (formerly), ALXN2040, ACH-4471, ACH4471
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: 200 mg | Experimental | All participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period. Receives: Drug: Danicopan; Drug: Placebo |
| Cohort 2: 500 mg | Experimental | All participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period. Receives: Drug: Danicopan; Drug: Placebo |
| Cohort 3: 800 mg | Experimental | All participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period. Receives: Drug: Danicopan; Drug: Placebo |
| Cohort 4: 75 mg | Experimental | All participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period. Receives: Drug: Danicopan; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities
Measured: Day 1 through Day 42
Secondary outcomes (5)
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Measured: Up to 16 hours postdose
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Measured: Up to 16 hours postdose
Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan
Measured: Up to 16 hours postdose
Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay
Measured: Up to 16 hours postdose
Relationship Between AP Inhibition And Danicopan Plasma Concentrations
Measured: Up to 16 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2021
- Study start21 May 2016
- Primary completion11 Jan 2017
- Study completion11 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HealthyStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Achillion, a wholly owned subsidiary of Alexion
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04889690
- Sponsor's protocol ID
- ACH471-002
- Status last verified
- May 2021
- First submitted
- 12 May 2021
- Last updated
- 17 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multiple Ascending Dose Study To Assess Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of ACH-0144471 In Healthy Volunteers
- ClinicalTrials.gov ID
- NCT04889690
- Acronym / sponsor ID
- ACH471-002
- Conditions
- Healthy
- Keywords
- Danicopan, ALXN2040, ACH-0144471, Factor D Inhibitor, Pharmacokinetics, Pharmacodynamics, Ascending Dose
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Achillion, a wholly owned subsidiary of Alexion
- Study start
- 21 May 2016 (actual)
- Primary completion
- 11 Jan 2017 (actual)
- Study completion
- 11 Jan 2017 (actual)
- First posted
- 17 May 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 was a multiple ascending dose, randomized, double-blind study assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of danicopan in healthy participants. Four different doses (75 milligrams [mg], 200 mg, 500 mg, 800 mg) and dose-matched placebo were administered under fasted conditions.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: 200 mg | Experimental | Drug: Danicopan, Drug: PlaceboAll participants under fasted conditions received 200 mg of danicopan or placebo twice daily (BID) over a 14-day period. |
| Cohort 2: 500 mg | Experimental | Drug: Danicopan, Drug: PlaceboAll participants under fasted conditions received 500 mg of danicopan or placebo BID over a 14-day period. |
| Cohort 3: 800 mg | Experimental | Drug: Danicopan, Drug: PlaceboAll participants under fasted conditions received 800 mg of danicopan or placebo BID over a 14-day period. |
| Cohort 4: 75 mg | Experimental | Drug: Danicopan, Drug: PlaceboAll participants under fasted conditions received 75 mg of danicopan or placebo thrice daily (TID) over a 7-day period. |
Who may qualify
- Ages
- 25 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Healthy was defined as having no clinically relevant abnormalities identified by a detailed medical history, physical exam, blood pressure and heart rate measurements, 12-lead electrocardiogram, and clinical laboratory tests.
- Body mass index of 18 to 30 kilograms (kg)/meter squared with a minimum body weight of 50 kg.
Key exclusion criteria (5 listed)
- History or clinically relevant evidence of significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease.
- Any condition possibly affecting drug absorption (including gastrectomy and cholecystectomy).
- Body temperature greater than or equal to 38°Celcius on Day -1 or Day 1, Hour 0; history of febrile illness or other evidence of infection within 14 days prior to first study drug administration.
- Current tobacco/nicotine user; consumption of any alcohol within 72 hours before first study drug administration or have a history of regular alcohol consumption exceeding 21 drinks/week within 6 months of screening; positive urine drug screen at screening or Day -1.
- Clinically significant laboratory abnormalities at either Screening or Day -1.
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 |
|---|---|---|
| DanicopanAlso: ACH-0144471 (formerly), ALXN2040, ACH-4471, ACH4471 | Drug | Cohort 1: 200 mg, Cohort 2: 500 mg, Cohort 3: 800 mg, Cohort 4: 75 mg |
| Placebo | Drug | Cohort 1: 200 mg, Cohort 2: 500 mg, Cohort 3: 800 mg, Cohort 4: 75 mg |
Primary outcome
Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities
Time frame: Day 1 through Day 42
5 secondary outcomes
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Time frame: Up to 16 hours postdose
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Time frame: Up to 16 hours postdose
Area Under The Plasma Concentration Versus Time Curve Over The Dosing Interval (AUCtau) Of Danicopan
Time frame: Up to 16 hours postdose
Activity Of Danicopan As Measured By Alternative Pathway (AP) Wieslab Assay
Time frame: Up to 16 hours postdose
Relationship Between AP Inhibition And Danicopan Plasma Concentrations
Time frame: Up to 16 hours postdose
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 17 May 2021.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Danicopan in people with Healthy, run by Alexion Pharmaceuticals, Inc. at 1 site in New Zealand.
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 2 inclusion and 5 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 17 May 2021. 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 New Zealand. 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?
Incidence Of Serious Adverse Events, Grade 3 Or 4 Adverse Events (AEs), AEs Leading To Discontinuation, And Clinically Significant Laboratory Abnormalities And Electrocardiogram Abnormalities (time frame: Day 1 through Day 42). 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 17 May 2021.