A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
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.
• Age ≥18 years old, gender not limited.
• 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.
• Patinets with Wilson's disease confirmed by laboratory tests to have double-chromosome mutations in the ATP7B gene.
• Low copper diet for at least six months befoer screening and willing to continue low copper diet during study.
• Fertile subjects agreed to adopt reliable contrraceptive methods from the screening until 6 months after the last administration.
• The subjects are atable patients with WD who have been trasted for at least six months without drug or dose changes for at least 6 momths at the time of screening , and have continuously used standard treatments \[SOC, such as D-penicillamine, sodiu dihydroxypropane sulfonate, dimercaptosuccinic acid, trientine, and zinc preparations (zinc acetate, zinc gluconate, zinc sulfate)\] for at least 6 months screening, and allowed subjects to continue with their prior SOC treatment.
• The subject's condition was fully controlled after treatment, and its definition must meet all of the following conditions:
‣ Serum NCC level ≥ 25 μg/L and ≤ 150 μg/L;
⁃ Urinary copper ≥100 μg/24 hours and ≤900 μg/24 hours;;
⁃ ALT \< 2 times of upper limit of normal value (ULN);
⁃ The investigator believes that no other laboratory values or clinical symptoms would stop current standard therapy;
• Subjects with good compliance, who can understand and cooperate to complete the requirements of protocol.
• The subjects voluntarily participated in the trial and signed the informed consent form.