Hepatic ImpairmentPhase 1NCT03555539
Study of Danicopan in Participants With Hepatic Impairment
A Phase 1 study testing Danicopan in people with Hepatic Impairment, run by Alexion Pharmaceuticals, Inc. at 2 sites in United States.
Trial summary
The purpose of this study was to evaluate the safety, tolerability, and pharmacokinetics (PK) of ACH-0144471 (danicopan) in participants with hepatic impairment (HI) as compared to healthy matched participants. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Hepatic Impairment
- What is being tested?
- Danicopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 May 2018to 21 Sep 2018
- 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 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Body mass index range of 18.0 to 40.0 kilograms (kg)/square meter with a minimum body weight of 50 kg at Screening.
- Females must be of non-childbearing potential.
- Males must agree to abstinence or use a highly effective method of contraception.
- HI Participants:
- Be sufficiently healthy for study participation.
- Diagnosis of chronic (>6 months) stable hepatic insufficiency.
Show all 14Show fewer
- A stable medication regimen.
- In good general health at Screening and check-in, allowing for the concurrent illnesses associated with HI.
- Evidence of cirrhosis due to hepatitis C virus (HCV), hepatitis B virus (HBV) infection, cryptogenic, alcohol abuse, or nonalcoholic steatohepatitis.
- No evidence of hepatocellular carcinoma.
- Have HI as assessed by a Child-Pugh classification score at Screening.
- Healthy Participants:
- Participants must be demographically matched to a hepatically impaired participant.
- Medically healthy and without a clinically significant medical history.
Who cannot join
- Evidence of any other clinically significant deviation from normal in a clinical laboratory for the match-controlled participants and laboratory findings beyond those that are consistent with the degree of HI from hepatic participants.
- History of any medical or psychiatric condition or disease.
- Any previous procedure that could alter the absorption or excretion of orally administered drugs.
- Body temperature greater than or equal to 38 degrees centigrade on Day -1 or Day 1 prior to dosing; history of febrile illness or other evidence of infection within 14 days prior to dosing.
- History or presence of drug or alcohol abuse within 6 months prior to dosing; current tobacco/nicotine user; positive for alcohol and/or drugs-of-abuse at Screening or check in.
- Participants who have received eculizumab at any dose or interval within the past 75 days.
Show all 21Show fewer
- Participation in any other investigational study drug trial 30 days before dosing.
- Donation of whole blood from 3 months before dosing or of plasma from 30 days before dosing; receipt of blood products within 6 months before dosing.
- HI Participants:
- Any acute or chronic non-hepatic condition that would limit the participant's ability to participate in this study.
- Any other unspecified reason that would make the participant unsuitable for enrollment.
- Any screening laboratory evaluation outside the laboratory reference ranges not related to HI.
- Fluctuating or rapidly deteriorating hepatic function within the screening period and up to 30 days prior to Day 1.
- History of chronic liver disease due to Wilson's disease.
- History of liver or other solid organ transplants.
- Healthy Participants:
- Clinical laboratory evaluations outside of the reference range at Screening or check-in.
- Evidence of acute or chronic liver disease.
- Use of any prescription medications/products within 14 days prior to dosing.
- Use of over-the-counter, nonprescription preparations within 7 days prior to dosing.
- Evidence of chronic HBV or chronic HCV infection.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Clinical Trial Site
Miami, Florida, 33136
Clinical Trial Site
Orlando, Florida, 32809
About this study
Official title: A Phase 1, 2-Part, Open-Label, Single-Dose, Parallel Group Study to Determine the Effect of Hepatic Impairment on the Safety, Tolerability, and Pharmacokinetics of ACH-0144471 in Adult Subjects
The purpose of this study was to evaluate the safety, tolerability, and pharmacokinetics (PK) of ACH-0144471 (danicopan) in participants with hepatic impairment (HI) as compared to healthy matched participants.
- Conditions
- Hepatic Impairment
- Being tested
- Danicopan
- Keywords
- Hepatic Impairment, Danicopan, ALXN2040, ACH-0144471, Pharmacokinetics, Factor D Inhibitor, Safety
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanicopanDrug
Also called ALXN2040, ACH-0144471 (formerly), ACH-4471, ACH4471
Oral tablet.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Healthy Match | Experimental | Single 200-milligram (mg) dose of danicopan on Day 1 in healthy participants (matched control group with normal hepatic function). Receives: Drug: Danicopan |
| Part 1: Moderate HI | Experimental | Single 200-mg dose of danicopan on Day 1 in participants with moderate HI. Receives: Drug: Danicopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under The Plasma Concentration Versus Time Curve From Time 0 Extrapolated To Infinity (AUC0-inf) Of Danicopan
Measured: Up to 72 hours postdose
Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Measurable Concentration (AUC0-t) Of Danicopan
Measured: Up to 72 hours postdose
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Measured: Up to 72 hours postdose
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Measured: Up to 72 hours postdose
Secondary outcomes (1)
Participants Experiencing Treatment-emergent Adverse Events
Measured: Day 1 (postdose) through follow-up visit (10 [+/- 2] days after last study drug administration)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2018
- Study start1 May 2018
- Primary completion21 Sep 2018
- Study completion21 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatic ImpairmentStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- This study was to be conducted sequentially in 2 parts. Part 1: Participants with moderate HI (based on Child-Pugh scores) were compared to demographically matched participants with normal hepatic function. Following a review of PK and safety data from Part 1, a decision was to be made by the principle investigators and the sponsor whether to conduct Part 2 of this study, which was planned to enroll participants with severe and mild HI.
- Masking
- None (open label)
- Enrolment
- 16 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
- NCT03555539
- Sponsor's protocol ID
- ACH471-012
- Status last verified
- Aug 2021
- First submitted
- 1 Jun 2018
- Last updated
- 20 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, 2-Part, Open-Label, Single-Dose, Parallel Group Study to Determine the Effect of Hepatic Impairment on the Safety, Tolerability, and Pharmacokinetics of ACH-0144471 in Adult Subjects
- ClinicalTrials.gov ID
- NCT03555539
- Acronym / sponsor ID
- ACH471-012
- Conditions
- Hepatic Impairment
- Keywords
- Hepatic Impairment, Danicopan, ALXN2040, ACH-0144471, Pharmacokinetics, Factor D Inhibitor, Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Achillion, a wholly owned subsidiary of Alexion
- Study start
- 1 May 2018 (actual)
- Primary completion
- 21 Sep 2018 (actual)
- Study completion
- 21 Sep 2018 (actual)
- First posted
- 13 Jun 2018 (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.
The purpose of this study was to evaluate the safety, tolerability, and pharmacokinetics (PK) of ACH-0144471 (danicopan) in participants with hepatic impairment (HI) as compared to healthy matched participants.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This study was to be conducted sequentially in 2 parts. Part 1: Participants with moderate HI (based on Child-Pugh scores) were compared to demographically matched participants with normal hepatic function. Following a review of PK and safety data from Part 1, a decision was to be made by the principle investigators and the sponsor whether to conduct Part 2 of this study, which was planned to enroll participants with severe and mild HI.
| Arm | Type | Receives |
|---|---|---|
| Part 1: Healthy Match | Experimental | Drug: DanicopanSingle 200-milligram (mg) dose of danicopan on Day 1 in healthy participants (matched control group with normal hepatic function). |
| Part 1: Moderate HI | Experimental | Drug: DanicopanSingle 200-mg dose of danicopan on Day 1 in participants with moderate HI. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Body mass index range of 18.0 to 40.0 kilograms (kg)/square meter with a minimum body weight of 50 kg at Screening.
- Females must be of non-childbearing potential.
- Males must agree to abstinence or use a highly effective method of contraception.
- Be sufficiently healthy for study participation.
- Diagnosis of chronic (>6 months) stable hepatic insufficiency.
Key exclusion criteria (21 listed)
- Evidence of any other clinically significant deviation from normal in a clinical laboratory for the match-controlled participants and laboratory findings beyond those that are consistent with the degree of HI from hepatic participants.
- History of any medical or psychiatric condition or disease.
- Any previous procedure that could alter the absorption or excretion of orally administered drugs.
- Body temperature greater than or equal to 38 degrees centigrade on Day -1 or Day 1 prior to dosing; history of febrile illness or other evidence of infection within 14 days prior to dosing.
- History or presence of drug or alcohol abuse within 6 months prior to dosing; current tobacco/nicotine user; positive for alcohol and/or drugs-of-abuse at Screening or check in.
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: ALXN2040, ACH-0144471 (formerly), ACH-4471, ACH4471Oral tablet. | Drug | Part 1: Healthy Match, Part 1: Moderate HI |
Primary outcome
Area Under The Plasma Concentration Versus Time Curve From Time 0 Extrapolated To Infinity (AUC0-inf) Of Danicopan
Time frame: Up to 72 hours postdose
Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Last Measurable Concentration (AUC0-t) Of Danicopan
Time frame: Up to 72 hours postdose
Maximum Observed Plasma Concentration (Cmax) Of Danicopan
Time frame: Up to 72 hours postdose
Time To Maximum Observed Plasma Concentration (Tmax) Of Danicopan
Time frame: Up to 72 hours postdose
1 secondary outcome
Participants Experiencing Treatment-emergent Adverse Events
Time frame: Day 1 (postdose) through follow-up visit (10 [+/- 2] days after last study drug administration)
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 20 Aug 2021.
- Individual participant dataSharing planned: Yes
Related trials for Hepatic Impairment
Not yet recruitingPhase 1
A Study to Assess the Pharmacokinetics and Safety of Revumenib in Participants With Advanced Cancer and Hepatic or Renal Impairment
Relapsed/Refractory (R/R) Hematologic Malignancies, Acute Leukemia
RecruitingPhase 1
A Study of Efimosfermin Alfa in Adults With Hepatic Impairment
Non-alcoholic Fatty Liver Disease
Not yet recruitingPhase 1
A Study of Zovaglutide (ZT002 Injection) in Participants With Hepatic Impairment and Healthy Participants
Overweight, Obesity
Not yet recruitingPhase 1
A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Tacabrutideg (BGB-16673) in Participants With Moderate and Severe Hepatic Impairment
Hepatic Impairment, Healthy Adult Participants
RecruitingPhase 1
Investigation of Pharmacokinetics, Safety of HSK31858 in Subjects With Hepatic Impairment
Non-cystic Fibrosis Bronchiectasis (NCFBE)
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.
2 sites 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 Hepatic Impairment, run by Alexion Pharmaceuticals, Inc. at 2 sites in United States.
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 14 inclusion and 21 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 20 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
Area Under The Plasma Concentration Versus Time Curve From Time 0 Extrapolated To Infinity (AUC0-inf) Of Danicopan (time frame: Up to 72 hours postdose). The study lists 4 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 20 Aug 2021.