A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
ConditionWilson Disease
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age10-60
SponsorChaohui Yu
About this trial
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, LY-M003 Injection is a gene therapy products with rAAV8 vector. After a single intravenous infusion, LY-M003 can be transduced to the target organ of liver and express the ATP7B in hepatocytese.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
The subject must be able to fully understood the purpose, nature, method, and possible adverse effects of the study, must be able to voluntarily participate in the study and voluntarily able to provide the written informed consent form (ICF).
Patients diagnosed with Wilson Disease .
Wilson Disease (WD) patients confirmed by laboratory tests to have biallelic mutations in the ATP7B gene.
Subjects must be treatment-experienced to WD who have received standard treatment (eg, D-penicillamine or zinc acetate) for at least 6 months prior to the screening period.
Disqualifiers
AAV8 neutralizing antibody titer > 1:10 .
Active gastrointestinal bleeding within the past 3 months.
Decompensated cirrhosis or advanced hepatic disease, manifested as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, etc.
Subjects with other liver diseases as determined by the investigator, such as immune hepatitis, alcoholic liver disease, primary biliary cholangitis, primary sclerosing cholangitis, and/or drug or toxic liver disease
Trial design
Design model
Single group
Treatments tested in this trial
LY-M003
GeneticA single peripheral intravenous (IV) infusion of LY-M003
Treatment groups
18 Participants
are divided into 5 treatment groupsGroup A: LY-M003 Dose group 1-Adult CohortExperimental treatment 1 intervention
Group B: LY-M003 Dose group 2-Adult CohortExperimental treatment 1 intervention
Group C: LY-M003 Dose group 3-Adult CohortExperimental treatment 1 intervention
Group D: LY-M003 Dose group 4-Adult CohortExperimental treatment 1 intervention
Group E: LY-M003-Pediatric CohortExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 52 weeks after the injection of LY-M003
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Time frame
From enrollment to 52 weeks after administration2
Incidence of dose-limiting toxicity (DLT) events assessed within at least 28 days following LY-M003 infusion
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.The adverse events defined as dose-limiting toxicity (DLT) have been clearly specified in the protocol.
Time frame
From enrollment to 52 weeks after administrationSecondary outcomes
1
Percentage decrease in standard of care (SoC) medication use within 52 weeks after administration
To assess the reduction in standard of care (SoC) medication use in subjects who completed administration of LY-M003 injection.
Time frame
From enrollment to 52 weeks after administration2
Number and proportion of subjects who discontinue standard of care medication within 52 weeks after administration
To evaluate the number and proportion of subjects who complete the administration of LY-M003 injection and discontinued standard of care (SOC) drugs.
Time frame
From enrollment to 52 weeks after administration3
Change from baseline in serum ceruloplasmin content level through 52 weeks after administration
To assess from baseline in serum ceruloplasmin content level through 52 weeks afterinjection of LY-M003.
Time frame
From enrollment to 52 weeks after administration4
Change from baseline in total serum copper level through 52 weeks after administrationfrom
To assess change from baseline in total serum copper level through 52 weeks after injection of LY-M003.
Time frame
From enrollment to 52 weeks after administrationSponsors and contacts
Click on the lead sponsor to view all of their trials.
Chaohui Yu
Lead sponsor
First Affiliated Hospital of Zhejiang University
Sponsor institution
Source ClinicalTrials.gov