This is a multicenter, open-label, single-arm, single-dose Phase I/II clinical study. It aims to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-M003 Injection in patients with Wilson's Disease (WD).
The subject fully comprehends the purpose, design, methods and possible adverse events of the study, agrees to participate voluntarily and signs the informed consent form (ICF).
Patients with confirmed diagnosis of Wilson's disease (WD).
Subjects with Wilson's disease (WD) confirmed by laboratory testing to have biallelic ATP7B gene mutation or deletion.
The subjects are treated patients with Wilson's disease (WD) who have received standard therapy (e.g., D-penicillamine or zinc acetate) continuously for at least 6 months prior to screening.
AAV8 neutralizing antibody titer > 1:10 .
History of active gastrointestinal bleeding within the past 3 months.
Decompensated liver cirrhosis or advanced liver disease presenting with portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, etc.
Subjects with other concomitant liver diseases as judged by the investigator, including autoimmune hepatitis, alcoholic liver disease, primary biliary cholangitis, primary sclerosing cholangitis, and/or drug- or toxin-induced liver disease.
Sequential
LY-M003 Injection
GeneticPhase 1: Participants receive a single, peripheral intravenous (IV) infusion of LY-M003 at dose group 1.The dose for Dose Group 1 is 4.0 × 10¹³ vg/kg.All participants are administered the drug once via intravenous injection.
LY-M003 Injection
GeneticPhase 1: Participants receive a single, peripheral intravenous (IV) infusion of LY-M003 at de-escalation dose.The dose for de-escalation dose is 2.0 × 10¹³ vg/kg.All participants are administered the drug once via intravenous injection.
LY-M003 Injection
GeneticPhase 2: Participants receive a single, peripheral intravenous (IV) infusion of LY-M003 at dose group 1.The dose for dose group 1 is 4.0 × 10¹³ vg/kg.All participants are administered the drug once via intravenous injection.
Phase I:The incidence of dose-limiting toxicity (DLT) events adjudicated by the Safety Review Committee (SRC) within at least 28 days after LY-M003 infusion.
The incidence of DLT will be assessed using CTCAE 6.0.
The incidence of treatment-emergent adverse events (AEs), adverse events of special interest (AESIs) and serious adverse events (SAEs) related to LY-M003 within 52 weeks after infusion.
Assessment will be conducted via 12-lead ECG, laboratory tests(blood routine, blood biochemistry, coagulation function, stool routine, urine routine), vital signs (blood pressure, pulse, respiratory rate, body temperature) and physical examination.
Percentage reduction in the dosage of standard of care (SoC) medications within 52 weeks after administration.
Assessment will be based on the percentage of participants who underwent standard dose reduction after infusion.
Number and proportion of subjects who discontinued standard of care (SoC) medications within 52 weeks after administration.
Assessment will be based on the number and percentage of participants who discontinued standard treatment medications.
Duration (in treatment weeks) of consecutive discontinuation of SoC medications among the above subjects.
Assessment will be based on the number of consecutive cycles during which participants discontinued standard medications.
Change in serum non-caeruloplasmin-bound copper (NCC) from baseline within 52 weeks after administration
Assessment will be based on changes in serum non-ceruloplasmin-bound copper from baseline.
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