Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UC Davis
Dates
study started
estimated completion

Description

Summary

The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at least 1 year with standard of care (that is, trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840 will be assessed at Week 48.

Official Title

A Phase 2, Single-arm, Pathologist-blinded Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in Patients With Wilson Disease Who Are Treated With ALXN1840 for 48 Weeks Followed by an Extension Treatment Period With ALXN1840 for up to an Additional 48 Weeks

Details

Participants who complete the 48-week Treatment Period will be offered the opportunity to continue their treatment in a 48-week Extension Period that will offer additional time for evaluation of long-term efficacy and safety of ALXN1840. There will be no liver biopsies during the Extension Period.

Keywords

Wilson Disease Copper ALXN1840 Histology Liver Biopsy Hepatolenticular Degeneration Tetrathiomolybdate Choline Bis-Choline Tetrathiomolybdate

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Diagnosis of WD by Leipzig Criteria > 4.
  2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening.
  3. Body mass index < 30 kilograms/meter squared.
  4. Able to cooperate with a percutaneous liver biopsy.
  5. Willing and able to follow protocol-specified contraception requirements.
  6. Capable of giving signed informed consent.

You CAN'T join if...

  1. Decompensated cirrhosis or Model for End Stage Liver Disease score > 13.
  2. Modified Nazer score > 7.
  3. Clinically significant gastrointestinal bleed within past 3 months.
  4. Alanine aminotransferase > 2 × upper limit of normal.
  5. History of bleeding abnormality or known coagulopathy, including platelet count < 100,000, and international normalized ratio for prothrombin time ≥ 1.5.
  6. Participant unwilling to accept blood products, if required.
  7. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
  8. Hemoglobin less than lower limit of the reference range for age and sex.
  9. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease 5) or creatinine clearance < 30 milliliters/minute.
  10. . Lymphoma, leukemia, or any malignancy within the past 5 years.
  11. . Current or chronic history of liver disease not associated with WD.

Locations

  • UC Davis Health System - UC Davis Medical Center - Liver Clinic
    Sacramento California 95817 United States
  • UT Southwestern
    Dallas Texas 75390 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alexion Pharmaceuticals
ID
NCT04422431
Phase
Phase 2
Study Type
Interventional
Last Updated