ALXN1840 for Wilson's Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called ALXN1840 (also known as Bis-Choline Tetrathiomolybdate) for individuals with Wilson Disease, a genetic disorder causing excess copper buildup in the body, particularly the liver. Researchers aim to determine if this treatment can reduce liver copper levels over 48 weeks and assess its safety and effectiveness. The trial includes adults who have managed their Wilson Disease with standard treatments like penicillamine, trientine, or zinc for at least a year and do not have severe liver damage. Participants must be able to undergo a liver biopsy and adhere to specific guidelines during the trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in Wilson Disease treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop your current medications, but it mentions that participants must have been on standard treatments like trientine, penicillamine, or zinc for at least a year before joining. It seems likely you will continue these medications during the trial.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research has shown that bis-choline tetrathiomolybdate, also called ALXN1840, is generally safe. Previous studies found that people with Wilson Disease, a condition where copper accumulates in the body, can take this treatment once a day without major side effects. Over six years, data presented at a major liver conference confirmed its safety and effectiveness. This suggests that ALXN1840 could be a promising option for patients, with a low risk of serious side effects.12345
Why do researchers think this study treatment might be promising for Wilson Disease?
Unlike the standard treatments for Wilson disease, which often include zinc or chelating agents like penicillamine and trientine, ALXN1840, also known as Bis-Choline Tetrathiomolybdate, offers a novel approach. This investigational drug works by uniquely binding copper and reducing its toxicity in the body, potentially offering a more direct and efficient way to manage copper accumulation compared to existing treatments. Researchers are excited about ALXN1840 because it could provide a more targeted mechanism of action, potentially leading to improved outcomes for patients with Wilson disease.
What evidence suggests that ALXN1840 might be an effective treatment for Wilson Disease?
Research has shown that ALXN1840, also known as bis-choline tetrathiomolybdate, might help treat Wilson Disease by controlling copper levels in the body. In one study, 71% of participants experienced a significant drop in harmful copper levels after 24 weeks of treatment. Another study found that ALXN1840 quickly improved copper balance without causing neurological issues. Participants generally tolerated it well, with most side effects being mild to moderate. These findings suggest that ALXN1840 could be a promising option for managing Wilson Disease by reducing excess copper.14567
Who Is on the Research Team?
Eugene S. Swenson, MD, PhD
Principal Investigator
Alexion Pharmaceuticals, Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ALXN1840 for 48 weeks to evaluate the change in liver copper concentration and other histopathologic changes
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Participants may continue treatment with ALXN1840 for an additional 48 weeks to evaluate long-term efficacy and safety
What Are the Treatments Tested in This Trial?
Interventions
- Bis-Choline Tetrathiomolybdate
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
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
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