Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
Summary
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation Period. In addition, efficacy and safety will be evaluated during an optional 60-month Extension Period.
Timeline
- Start
- 2018-02-22
- Primary completion
- 2023-06-30
- Completion
- 2023-06-30
Outcome
Met primary endpoint
Stopped (Business): “Sponsor decision to terminate the program”
release “Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN1840, an investigational once-daily, oral medicine, met its primary endpoint” astrazeneca.com ↗
release “These analyses build upon the previously reported Phase 3 FoCus results demonstrating ALXN1840 met its primary endpoint of superior copper mobilization versus standard of care.” monopartx.com ↗
release “ALXN1840 met the primary endpoint by demonstrating rapid and sustained copper mobilization significantly greater than standard of care over 48 weeks” monopartx.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | TIOMOLIBDATE CHOLINE | Small molecule | 15 mg | Oral |
| Subject | TIOMOLIBDATE CHOLINE | Small molecule | 60 mg | Oral |
| Comparator | Zinc Sulfate | Unknown | — | — |
| Comparator | penicillamine | Unknown | — | — |
| Comparator | trientine | Unknown | — | — |