A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson's Disease

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-509998-23-00

Acronym

VTX-801_CLN_001

Enrollment

Registered

2024-04-18

Start date

2022-06-21

Completion date

2024-10-08

Last updated

2024-04-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Wilson's Disease

Brief summary

Safety and tolerability profile, including treatment-emergent adverse events (TEAE), clinical examination, changes in laboratory parameters, vital signs, ECG, brain and abdominal MRI

Detailed description

Free serum Cu, Total serum Cu, 24-hour urinary Cu, Serum ceruloplasmin activity (enzymatic assay), VTX-801 Responder status, Immune response to VTX-801

Interventions

None listed

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Safety and tolerability profile, including treatment-emergent adverse events (TEAE), clinical examination, changes in laboratory parameters, vital signs, ECG, brain and abdominal MRI	—

Secondary

Measure	Time frame
Free serum Cu, Total serum Cu, 24-hour urinary Cu, Serum ceruloplasmin activity (enzymatic assay), VTX-801 Responder status, Immune response to VTX-801	—

Countries

Denmark, Germany

Outcome results