Assessing ALXN1840 in Patients with Wilsons Disease
Research type
Research Study
Full 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
IRAS ID
1003343
Contact name
Aftab Ala
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc
Eudract number
2019-003711-60
Research summary
his study is being conducted to assess the effect of the investigational drug ALXN1840
on changes in liver copper (Cu) concentration and liver histopathology, in patients with
Wilson disease (WD). WD is a rare genetic disorder that causes Cu to build up in the
liver, which is the primary storage organ in the body for Cu. When the Cu storage
capacity of the liver cells is exhausted, it will then spill into the bloodstream and deposit
in other parts of the body, such as the brain. Consequently, untreated or inadequately
treated WD disease may cause damage to the liver, as well as the central nervous system.
There are standard of care (SoC) medications (trientine, penicillamine and zinc)
available to treat WD, but they primarily help control excess Cu in the blood and do not
consistently help reduce Cu overload in the liver. ALXN1840 is being developed, with its
active ingredient Tetrathiomolybdate having previously been shown to reduce Cu in the
liver in animals. The principal aim of this study is therefore to evaluate the effect of
ALXN1840 on reduction of liver Cu concentration at Week 48 of treatment, when
compared with adult patients with WD treated with SoC for at least 1 year. This study
will include a Screening Period up to 4 weeks, followed by a 48-week Treatment Period
that starts on the day of first dose of ALXN1840, and an optional 48-week Extension
Period. The total planned duration is approximately 100 weeks when including the option
Extension Period. Study procedures are summarised further the in patient information
sheet.REC name
London - Westminster Research Ethics Committee
REC reference
20/LO/0939
Date of REC Opinion
25 Aug 2020
REC opinion
Further Information Favourable Opinion