PK Study of Oral ALXN1840 in Japanese and Non-Japanese Adult HV
Research type
Research Study
Full title
An Open-Label, Two-Single-Dose, Two-Period, Parallel Group Study to Assess the Pharmacokinetics and Safety of ALXN1840 in Healthy Adult Japanese and non-Japanese Subjects
IRAS ID
258032
Contact name
Ulrike Lorch
Contact email
Sponsor organisation
Alexion Pharma GmbH
Eudract number
2018-004483-57
Duration of Study in the UK
0 years, 1 months, 19 days
Research summary
We are conducting a clinical trial with the investigational drug bis-choline tetrathiomolybdate (ALXN1840). This is being developed by Alexion Pharmaceuticals, Inc. for the treatment of Wilson’s Disease (WD). This study will assess the pharmacokinetics and safety of ALXN1840 in healthy Japanese and non-Japanese subjects, to evaluate the appropriateness of testing ALXN1840 in patients with WD in Japan.\n\nWD is a genetic disorder that causes people to have higher levels of copper in their body than normal. Increased levels of copper can be toxic and cause damage to the liver, brain and other organs. Currently there are very few treatment options available for individuals with WD.\n\nALXN1840 is a novel first-in-class protein binding agent that rapidly forms virtually irreversible, stable copper-protein complexes. These complexes are then removed by biliary excretion into faeces. It also acts directly on the liver to remove excess copper. It is hoped that ALXN1840 will yield greater compliance compared to current treatments, as it is administered once daily rather than in multiple daily doses.\n\nALXN1840 has been evaluated in 28 patients with WD. Results showed that ALXN1840 monotherapy reduced mean serum copper by 72% after 24 weeks compared to baseline. Liver status also improved in the majority of patients. ALXN1840 was generally well tolerated. It was also tolerated in previous healthy volunteer studies.\n\n24 volunteers will participate in the study in 2 cohorts, each lasting 56 days (27 days for screening and 29 days for dosing and follow-up). Cohort 1 will contain 12 Japanese volunteers. Cohort 2 will contain 12 non-Japanese volunteers. Both cohorts will consist of 2 periods. In Period 1 they will be given one 15mg tablet. In Period 2 they will be given four 15mg tablets.\n\nWe will assess safety parameters including physical examination, vital signs, laboratory evaluations, electrocardiogram and monitoring of adverse events.
REC name
London - London Bridge Research Ethics Committee
REC reference
19/LO/0048
Date of REC Opinion
29 Jan 2019
REC opinion
Favourable Opinion