Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

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Brief Title

Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Official Title

A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

Brief Summary

      This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and
      pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson
      disease (WD).

Detailed Description

      Participants who complete the 48 weeks of treatment in Period 1 will have the option to
      receive ALXN1840 for 24 weeks in Period 2 (open-label extension).

      Safety will be monitored throughout the study.

Study Phase

Phase 3

Study Type


Primary Outcome

Percentage Change From Baseline To Week 48 In Non-ceruloplasmin-bound Copper (NCC) In Plasma

Secondary Outcome

 Area Under The Effect Versus Time Curve (AUEC) For NCC And Plasma Total Copper


Wilson Disease



Study Arms / Comparison Groups

Description:  ALXN1840 will be administered at one of two starting doses, with incremental dose increases permitted.


* 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


Estimated Enrollment


Start Date

October 6, 2021

Completion Date

May 31, 2024

Primary Completion Date

October 31, 2023

Eligibility Criteria

        Key Inclusion Criteria:

          1. Diagnosis of Wilson Disease by Leipzig Score ≥ 4.

          2. Adequate venous access to allow collection of required blood samples.

          3. Able to swallow intact ALXN1840 tablets or mini-tablets.

          4. Willing to avoid intake of foods and drinks with high contents of copper.

          5. Willing and able to follow protocol-specified contraception requirements.

        Key Exclusion Criteria:

          1. Decompensated hepatic cirrhosis or MELD score > 13 (ages 12 to <18) or PELD score > 13
             (ages 3 to < 12).

          2. Modified Nazer score > 7.

          3. Clinically significant gastrointestinal bleed within past 3 months.

          4. Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) for participants
             treated for > 28 days with WD therapy or ALT > 5 × ULN for treatment-naïve
             participants or participants who have been treated for ≤ 28 days.

          5. Marked neurological disease requiring either nasogastric feeding tube or intensive
             inpatient medical care.

          6. Hemoglobin less than lower limit of the reference range for age and sex.

          7. History of seizure activity within 6 months prior to informed consent/assent.

          8. Participants in renal failure, defined as in end-stage renal disease on dialysis
             (chronic kidney disease stage 5) or estimated glomerular filtration rate < 30
             milliliters/minute/1.73 meter squared.




3 Years - 17 Years

Accepts Healthy Volunteers



Eugene S. Swenson, MD, PhD, +1 855-752-2356, [email protected]

Location Countries


Location Countries


Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Alexion Pharmaceuticals

Study Sponsor

Eugene S. Swenson, MD, PhD, Study Director, Alexion Pharmaceuticals

Verification Date

June 2022