NCT06663878

An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease

An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.

Detailed description

This is an open-label, dose escalation study to evaluate the tolerability, safety, and preliminary efficacy of MWAV201 in patients with Wilson disease. Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201. The dose escalation plan and the number of dose levels may be adjusted during study period.

Conditions

• Wilson Disease

Interventions

Type	Name	Description
GENETIC	MWAV201	Nonreplicating, recombinant gene transfer vector

Timeline

• Start date: 2024-10-01
• Primary completion: 2026-03-01
• Completion: 2031-05-01
• First posted: 2024-10-29
• Last updated: 2024-10-29

Locations

1 site across 1 country: China