Wilson Disease Patients to evaluate efficacy and safety of WTX101
Research type
Research Study
Full title
A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older
IRAS ID
169744
Contact name
Aftab Ala
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2014-001703-41
Duration of Study in the UK
1 years, 0 months, 17 days
Research summary
This is a study of an investigational drug, called WTX101 (bischoline
TETRATHIOMOLYBDATE capsules), an investigational drug being developed by Wilson Therapeutics, for the treatment of the Wilson Disease.
This study will assess the safety and effectiveness of WTX101 at different doses, administered with a proton pump inhibitor (PPI), in treating patients with Wilson Disease newly diagnosed during a period of 24 weeks.About 30 patients are expected to take part from approximately 2 to 8 hospitals in Europe and to 2 to 8 hospitals the United States.
After undergoing certain screening procedures, suitable patients will be enrolled to receive the study drug WTX101 during 24 weeks. As the study is open label, all the patients will be treated with the investigational drug during the study.
Whilst in the study, patients will undergo 6 treatment cycles and have the following study procedures performed: physical examinations, vital signs recorded, ECG, and blood (including pharmacokinetic and pharmacodynamic samples PK/PD), blood sampling for safety, urine samples taken for laboratory tests. Participants will also be required to complete various scales and various questionnaires, some scalings which will be completed by the investigator (Unified Wilson Disease Rating Scale (UWDRS), at regular intervals throughout the study.
The number of treatment cycles a patient may receive is 6, and so a patient’s length of participation in the study will depend on how they respond to the study treatment. At the end of the study, patients who experience clinical benefit from the study treatment may be offered the opportunity to continue the study medication in an extension phase.
REC name
Scotland B REC
REC reference
15/SS/0030
Date of REC Opinion
11 May 2015
REC opinion
Further Information Favourable Opinion