ALXN2050 for Liver Dysfunction
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.12345Why 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.46789
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive multiple doses of ALXN2050 to assess pharmacokinetics, safety, and tolerability
Safety Review
Safety data is reviewed to determine progression to the next cohort
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ALXN2050
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
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
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
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