Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

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Brief Title

Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

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

Brief 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.
    

Detailed Description

      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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change From Baseline At Week 48 In Liver Cu Concentration

Secondary Outcome

 Change From Baseline At Week 48 In Liver Histopathology As Assessed by Liver Biopsy

Condition

Wilson Disease

Intervention

Bis-Choline Tetrathiomolybdate

Study Arms / Comparison Groups

 ALXN1840
Description:  Participants will receive ALXN1840.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

28

Start Date

June 2020

Completion Date

June 2022

Primary Completion Date

June 2021

Eligibility Criteria

        Inclusion Criteria:

          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.

        Exclusion Criteria:

          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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 855-752-2356, [email protected]

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT04422431

Organization ID

ALXN1840-WD-205

Secondary IDs

2019-003711-60

Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

, , 


Verification Date

June 2020