An upcoming study will assess the benefit of continued treatment with ALCN1840 in patients with Wilson disease (WD).

Researchers have initiated an expanded access study for patients with WD that could receive a treatment scheme with a novel drug named ALXN1840. The intended sample of this clinical trial is cataloged as an intermediate-size population.

This open-label, multicenter, single-arm expanded access program aims to offer early access to treatment with ALXN1840 for eligible individuals.

Read more about WD therapies

The trial will include children and adults of 3 years of age or older, of both sexes, with a previously confirmed diagnosis of WD. All participants must have completed either the ALXN1840-WD-205, WTX101-301, ALXN1840-WD-302, or other ALXN1840 studies in the past.

Each patient’s treating physician must agree that the possible benefits outweigh the potential risks for ALXN1840. In the case of females of childbearing age, they must also comply with contraception methods. All individuals or their legal representatives, according to their age, must understand and consent or assent to this expanded access program.

On the other hand, patients eligible to participate in an Alecion-sponsored study of ALXN1840 could not participate in this protocol. Likewise, being unable to comply with the treatment plan, current pregnancy, known hypersensitivity to the drug or excipients, decompensated hepatic cirrhosis, a score of 13 points or more in the Model for End-Stage Liver Disease scale, a Modified Nazer Score of 7 or more points, end-stage renal disease cataloged as chronic kidney disease stage 5 (CKD 5), or with a creatinine clearance of 30 ml/min or less, are part of the exclusion criteria.

ALXN1840, also known as bis-choline tetrathiomolybdate, tiomolibdic acid, or tiomolibdate choline (WTX101), is an investigational medicinal product that is still waiting to receive marketing authorization.


Early access program for ALXN1840 in patients with Wilson disease. Accessed January 24, 2023.