Impaired Hepatic FunctionPhase 1NCT05259085
Study of ALXN2050 in Participants With Hepatic Impairment
A Phase 1 study testing ALXN2050 in people with Impaired Hepatic Function and Healthy, run by Alexion Pharmaceuticals, Inc. at 2 sites in United States.
Trial summary
This study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with varying degrees of IHF. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Impaired Hepatic Function, Healthy
- What is being tested?
- ALXN2050
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 2 sitesUnited States
- Study dates
- 7 Apr 2022to 12 Aug 2024
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Body weight must be at least 50.0 kilograms (kg) and body mass index (BMI) within the range of 18.0 - 40.0 kg/meter squared (inclusive) at the time of signing the informed consent.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Must agree to receive prophylactic antibiotics to mitigate the potential risk of meningococcal infection.
- Participants with Impaired Hepatic Function
- Aside from IHF, sufficiently healthy for study participation based upon medical history, physical examination, neurological examination, laboratory tests, vital signs, and electrocardiograms (ECGs).
- Score on the Child-Pugh scale at screening as follows:
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- Mild: Class A (Child-Pugh score ≥5 and ≤6); or
- Moderate: Class B (Child-Pugh score ≥7 and ≤9); or
- Severe: Class C (Child-Pugh score ≥10 and ≤15).
- Diagnosis of chronic (>6 months), stable (no acute episodes of illness within the previous 1 month due to deterioration in hepatic function) hepatic insufficiency.
- Must be on a stable medication regimen. Concomitant medications must be approved by Alexion unless presented in the list of common concurrent medications for participants with IHF.
- Cirrhosis, as evidenced by parenchymal liver disease by biopsy (histological diagnosis), imaging test, or other suitable imaging study, due to chronic hepatitis C virus (HCV) infection, chronic hepatitis B infection, cryptogenic, alcohol abuse, or non-alcoholic steatohepatitis.
- No evidence of hepatocellular carcinoma as documented by imaging within 6 months prior to the first dose of study intervention.
- Matched Healthy Control Participants with Normal Hepatic Function
- Must match the sex (similar ratio) and race (similar ratio of white and non-white) of participants with IHF; age must be within ± 10 years and BMI must be within ± 20% of participants with IHF at screening.
- Healthy as determined by medical evaluation including medical history, physical examination, neurological examination, laboratory tests, vital signs, and ECGs.
Who cannot join
- History or presence of seizures, head injury, head trauma, or any other brain disorder.
- History of procedures that could alter absorption or excretion of orally administered drugs.
- History of meningococcal infection or a first-degree relative with a history of meningococcal infection.
- Body temperature ≥38.0°Celcius at screening or check-in or history of febrile illness or other evidence of infection, systemic or otherwise, within 14 days prior to the first dose of study intervention.
- Classical pathway hemolysis results outside the reference ranges at screening, unless approved by Alexion.
- Significant blood loss or donation of blood within 3 months prior to the first dose of study intervention, donation of plasma within 30 days prior to the first dose of study intervention, receipt of blood products within 6 months prior to first dose of study intervention, or receipt of a vaccine within 30 days prior to the first dose of study intervention.
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- Current enrollment or past participation within the last 30 days (or 5 half-lives, whichever is longer) prior to the first dose of study intervention in the current clinical study or any other clinical study involving an investigational study intervention or any other type of medical research.
- Pregnant or lactating.
- History or presence of drug or alcohol abuse within 6 months prior to the first dose of study intervention, current tobacco user, or positive results for alcohol and/or drug screen at screening or check-in.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
Hialeah, Florida, 33014
Research Site
Orlando, Florida, 32809
About this study
Official title: A Phase 1, Open-Label, Multiple-Dose, Parallel Study to Determine the Effect of Hepatic Impairment on the Pharmacokinetics, Safety, and Tolerability of ALXN2050 in Adult Participants
This study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with varying degrees of IHF.
Read the detailed description
The study will initiate (Part 1) with participants with mild IHF (Cohort 1) and moderate IHF (Cohort 2) and their matched healthy control participants (Cohort 4). Cohort 1 will be enrolled first, and following an adequate safety review, enrollment for Cohort 2 will begin. Following data review, the study may proceed (Part 2) with participants with severe IHF (Cohort 3) if deemed necessary.
- Conditions
- Impaired Hepatic Function, Healthy
- Being tested
- ALXN2050
- Keywords
- Pharmacokinetics, Safety, Factor D inhibitor, ALXN2050
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN2050Drug
Also called ACH-0145228 (formerly)
ALXN2050 (120 milligrams) will be administered orally twice daily on Days 1 through 3, with an additional dose (120 milligrams) administered orally on the morning of Day 4.
| Study group | Role | What they receive |
|---|---|---|
| ALXN2050 | Experimental | Cohort 1: Mild IHR Cohort 2: Moderate IHR Cohort 3: Severe IHR Cohort 4: Healthy Control
Participants will receive ALXN2050 Receives: Drug: ALXN2050 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under The Concentration-time Curve From Time 0 To The 12-hour Time Point (AUC0-12) Of Plasma ALXN2050 After Steady-state
Measured: Up to 72 hours postdose
Maximum (Peak) Steady-state Plasma Concentration Of ALXN2050 (Cmax,ss)
Measured: Up to 72 hours postdose
Time To Reach Maximum (Peak) Plasma Concentration Following ALXN2050 Administration At Steady-state (Tmax,ss)
Measured: Up to 72 hours postdose
Secondary outcomes (1)
Number Of Participants Receiving ALXN2050 With Treatment-emergent Adverse Events
Measured: Day 1 (postdose) through follow-up (30 [+/- 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 posted28 Feb 2022
- Study start7 Apr 2022
- Primary completion12 Aug 2024
- Study completion12 Aug 2024
Why it stopped: Sponsor decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Impaired Hepatic FunctionStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05259085
- Sponsor's protocol ID
- ALXN2050-HV-109
- Status last verified
- Feb 2025
- First submitted
- 5 Nov 2020
- Last updated
- 19 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Multiple-Dose, Parallel Study to Determine the Effect of Hepatic Impairment on the Pharmacokinetics, Safety, and Tolerability of ALXN2050 in Adult Participants
- ClinicalTrials.gov ID
- NCT05259085
- Acronym / sponsor ID
- ALXN2050-HV-109
- Conditions
- Impaired Hepatic Function, Healthy
- Keywords
- Pharmacokinetics, Safety, Factor D inhibitor, ALXN2050
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision.
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Apr 2022 (actual)
- Primary completion
- 12 Aug 2024 (actual)
- Study completion
- 12 Aug 2024 (actual)
- First posted
- 28 Feb 2022 (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 study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing recommendations for future indications in individuals with varying degrees of IHF.
Detailed description
The study will initiate (Part 1) with participants with mild IHF (Cohort 1) and moderate IHF (Cohort 2) and their matched healthy control participants (Cohort 4). Cohort 1 will be enrolled first, and following an adequate safety review, enrollment for Cohort 2 will begin. Following data review, the study may proceed (Part 2) with participants with severe IHF (Cohort 3) if deemed necessary.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALXN2050 | Experimental | Drug: ALXN2050Cohort 1: Mild IHR Cohort 2: Moderate IHR Cohort 3: Severe IHR Cohort 4: Healthy Control Participants will receive ALXN2050 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Body weight must be at least 50.0 kilograms (kg) and body mass index (BMI) within the range of 18.0 - 40.0 kg/meter squared (inclusive) at the time of signing the informed consent.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Must agree to receive prophylactic antibiotics to mitigate the potential risk of meningococcal infection.
- Participants with Impaired Hepatic Function
- Aside from IHF, sufficiently healthy for study participation based upon medical history, physical examination, neurological examination, laboratory tests, vital signs, and electrocardiograms (ECGs).
Key exclusion criteria (9 listed)
- History or presence of seizures, head injury, head trauma, or any other brain disorder.
- History of procedures that could alter absorption or excretion of orally administered drugs.
- History of meningococcal infection or a first-degree relative with a history of meningococcal infection.
- Body temperature ≥38.0°Celcius at screening or check-in or history of febrile illness or other evidence of infection, systemic or otherwise, within 14 days prior to the first dose of study intervention.
- Classical pathway hemolysis results outside the reference ranges at screening, unless approved by Alexion.
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 |
|---|---|---|
| ALXN2050Also: ACH-0145228 (formerly)ALXN2050 (120 milligrams) will be administered orally twice daily on Days 1 through 3, with an additional dose (120 milligrams) administered orally on the morning of Day 4. | Drug | ALXN2050 |
Primary outcome
Area Under The Concentration-time Curve From Time 0 To The 12-hour Time Point (AUC0-12) Of Plasma ALXN2050 After Steady-state
Time frame: Up to 72 hours postdose
Maximum (Peak) Steady-state Plasma Concentration Of ALXN2050 (Cmax,ss)
Time frame: Up to 72 hours postdose
Time To Reach Maximum (Peak) Plasma Concentration Following ALXN2050 Administration At Steady-state (Tmax,ss)
Time frame: Up to 72 hours postdose
1 secondary outcome
Number Of Participants Receiving ALXN2050 With Treatment-emergent Adverse Events
Time frame: Day 1 (postdose) through follow-up (30 [+/- 2] days after last study drug administration)
Results and safety
The study was stopped early (reason given: Sponsor decision.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2025.
- Individual participant dataSharing planned: No
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Hepatic Impairment
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 ALXN2050 in people with Impaired Hepatic Function and Healthy, 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 16 inclusion and 9 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: stopped early, as of the sponsor's last update on 19 Mar 2025. 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 was stopped early (reason given: Sponsor decision.). Results are not available in the source record.
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 Concentration-time Curve From Time 0 To The 12-hour Time Point (AUC0-12) Of Plasma ALXN2050 After Steady-state (time frame: Up to 72 hours postdose). The study lists 3 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 19 Mar 2025.