ALXN2050 for Liver Dysfunction

No longer recruiting at 3 trial locations

Overseen By Alexion Pharmaceuticals, Inc. (Sponsor)

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: Prophylactic antibiotics

Disqualifiers: Seizures, Brain disorder, Alcohol abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Varies by cohort

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how the new treatment, ALXN2050, behaves in the body when liver function is impaired. It seeks to provide guidance on dosing for individuals with varying levels of liver dysfunction, from mild to severe, compared to healthy individuals. The trial seeks participants with stable liver issues for over six months, such as cirrhosis from hepatitis or alcohol use, as well as those who are otherwise healthy.

As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I have to stop taking my current medications for the trial?

Participants must be on a stable medication regimen, and any additional medications need approval from Alexion unless they are common for those with impaired liver function. The protocol does not specify a need to stop current medications, but approval is required for any changes.

Is there any evidence suggesting that ALXN2050 is likely to be safe for humans?

Research has shown that ALXN2050 has undergone safety studies in humans. One study examined the safety of ALXN2050 when administered in multiple doses. Ethics committees approved this study to ensure participant safety. Although detailed results from this study remain undisclosed, it indicates that safety is closely monitored.

As ALXN2050 is in an early trial phase, researchers continue to gather safety information. This phase primarily ensures the treatment's safety for people, with careful monitoring and reporting of any side effects.

Other studies on ALXN2050 have also concentrated on safety. While detailed safety data is not publicly available, the progression of these studies suggests the treatment has been safe enough thus far. Prospective participants can expect thorough safety monitoring throughout the study. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for liver dysfunction focus on managing symptoms and slowing disease progression through lifestyle changes, medications like ursodeoxycholic acid, or in severe cases, liver transplantation. However, ALXN2050 stands out because it targets the complement system, a part of the immune system that can contribute to liver damage. This new approach could potentially offer a more direct way to reduce liver inflammation and damage. Researchers are excited about ALXN2050 because it might offer better disease management by addressing an underlying cause rather than just alleviating symptoms.

What evidence suggests that ALXN2050 might be an effective treatment for liver dysfunction?

Research has shown that ALXN2050 is under study for its potential benefits in treating conditions like generalized myasthenia gravis (gMG). In studies focused on gMG, ALXN2050 improved symptoms, as measured by the QMG score, which assesses muscle strength in people with this condition. While these results are promising for gMG, this trial also explores ALXN2050 for liver dysfunction. Although specific data for liver issues remains limited, its effectiveness in other conditions offers hope for future uses. ^{4 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for adults with varying degrees of liver dysfunction (Child-Pugh Classes A-C) and healthy controls matched by sex, race, age (+/- 10 years), and BMI (+/- 20%). Participants must not have liver cancer, be on a stable medication regimen, agree to use contraception and take antibiotics to prevent infection. Exclusions include fever or infection within 14 days before the study drug is given, recent blood donations or receipt of vaccines.

Inclusion Criteria

I am a healthy control with normal liver function, matching the sex, race, age (±10 years), and BMI (±20%) of the IHF participants.

I am healthy with normal liver function, confirmed by medical tests.

Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion Criteria

I haven't donated blood, received blood products, or had vaccines recently.

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.

Pregnant or lactating.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of ALXN2050 to assess pharmacokinetics, safety, and tolerability

Varies by cohort

Safety Review

Safety data is reviewed to determine progression to the next cohort

Not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ALXN2050

Trial Overview

The trial studies how different levels of liver impairment affect the body's handling of ALXN2050. It aims to establish dosing guidelines for patients with impaired hepatic function. The study includes both those with liver issues and healthy individuals as controls to compare results.

How Is the Trial Designed?

Group I: ALXN2050 Experimental Treatment 1 Intervention

Cohort 1: Mild IHR Cohort 2: Moderate IHR Cohort 3: Severe IHR Cohort 4: Healthy Control Participants will receive ALXN2050

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Alexion Pharmaceuticals

Lead Sponsor

Trials

231

Recruited

36,700+

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05259085

Study of ALXN2050 in Participants With Hepatic Impairment

Study Overview. This study will investigate the impact of impaired hepatic function (IHF) on the plasma pharmacokinetics of ALXN2050 in order to provide dosing ...

2.

s3.amazonaws.com

s3.amazonaws.com/ctr-med-7111/ALXN2050-MG-201/d7fb4ef2-75ad-45ff-9749-51566dbf9ca8/e95e92ff-0ed9-42a2-8de0-7caebf41efe8/ALXN2050-MG-201_Abbreviated_CSR_Synopsis_redacted-v1.pdf

SYNOPSIS

To assess the efficacy of. ALXN2050 compared with placebo in the treatment of gMG based on improvement in the. QMG total score. Change from ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5114163/

The effect of hepatic impairment on outcomes in phase 1 ...

The differential effects on hepatotoxicity suggest that underlying hepatic function may affect susceptibility to and patterns of DILI.

4.

reporter.nih.gov

reporter.nih.gov/project-details/10809651

RePORT ⟩ RePORTER

We're sorry but RePORTER doesn't work properly without JavaScript enabled. Please enable it to continue.

5.

astrazeneca.com

astrazeneca.com/content/dam/az/PDF/2023/q3/9M_and_Q3_2023_results_clinical_trials_appendix.pdf

Clinical Trials Appendix

• Data anticipated: >2024. Phase I. ALXN2050-HV-109. NCT05259085. Impaired hepatic function. 36. • Arm 1: mild IHF, 120mg vemircopan BID orally on Days 1.

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05259085

Study of ALXN2050 in Participants With Hepatic Impairment

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) ...

7.

isrctn.com

isrctn.com/pdf/14891060

A study to investigate the pharmacokinetic profile of ...

To assess the safety and tolerability of ALXN2050 following multiple oral dosing of a selected ALXN2050 MR prototype. Ethics approval required.

8.

go.drugbank.com

go.drugbank.com/conditions/DBCOND0035872

Impaired Hepatic Function (DBCOND0035872)

Associated Data ; NCT05259085. Study of ALXN2050 in Participants With Hepatic Impairment. Vemircopan ; NCT02010034. Compassionate Use of Omegaven in the Treatment ...

9.

isrctn.com

isrctn.com/ISRCTN14891060

A study to investigate the pharmacokinetic profile of ...

To determine the safety and tolerability of ALXN2050 following administration of ALXN2050 MR prototypes and the IR tablet formulation. Part 2 2.1. To assess the ...