A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)

Recruiting Early Phase 1 Interventional Study
Wilsons Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 and older
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
18 (estimated)
Sponsor
DSciLab Co., Ltd. · Industry
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About This Trial

This study adopted an open, single-arm, non-randomized, dose-escalation research design, aiming to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic and immunogenicity characteristics of single and multiple intravenous infusions of DSL101 in patients with Wilson's disease.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Age ≥18 years old, gender not limited. 2. Meet the diagnostic criteria f Wilson's Disease in "Guidelines for Diagnosis and Treatment of Wilson's Disease (2022 Edition)", with a Leipzig score ≥4, at least one year between diagnosis and screening; ceruloplasmin level \<0.1g/L. …
Contacts

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