Wilson's DiseasePhase 1/2NCT07641140
Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
A Phase 1/2 study testing LY-M003 Injection in people with Wilson's Disease, run by Lingyi Biotech Co., Ltd. at 1 site in China.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Wilson's Disease
- What is being tested?
- LY-M003 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 15 Jun 2026to 30 Dec 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
- Subjects have maintained a low-copper diet for at least 6 consecutive months prior to screening and will continue this dietary restriction throughout the study.
- Subjects must agree to refrain from donating blood, organs, tissues or cells at any time after treatment.
- Female subjects of childbearing potential (WOCBP) must have a negative pregnancy test.
- Subjects and their partners must have no plans for pregnancy from screening through 6 months after study completion, and will voluntarily use effective contraception (e.g., abstinence, condoms). Subjects shall not plan to donate sperm or ova.
Who cannot join
- 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.
- Subjects with severe hypersplenism complicated and requiring splenectomy as assessed by the investigator.
- Model for End-Stage Liver Disease (MELD) score > 13.
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- Other disorders of copper metabolism, such as chronic cholestatic liver diseases, disorders of glycosylation, copper metabolism disorders, etc.
- A history of non-compliance with copper chelators or zinc agents as assessed by the investigator within 6 months prior to screening.
- Previously treated WD subjects with ALT and/or AST levels more than 5 times the upper limit of normal (ULN).
- Subjects with severe neurological deficits or impairments that, in the investigator's judgment, compromise their safety and/or ability to participate in the study.
- Hemoglobin < 90g/L.
- Subjects with positive hepatitis B surface antigen (HBsAg), positive hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV) antibody or positive treponema pallidum antibody.
- Subjects with end-stage renal disease on dialysis (Chronic Kidney Disease Stage 3 and above), or creatinine clearance < 60 mL/min.
- Severe hyperlipidemia (triglycerides >1000 mg/dL);
- Subjects who have received or plan to undergo bone marrow transplantation, hematopoietic stem cell transplantation and/or major organ transplantation, including but not limited to liver transplantation and renal transplantation.
- Subjects with clinically diagnosed severe cardiovascular diseases or those deemed by the investigator to have such conditions (e.g., New York Heart Association [NYHA] heart failure classification ≥ Class 3).
- Subjects with uncontrolled concomitant diseases or infectious diseases as assessed by the investigator.
- Subjects who are allergic to any ingredient of LY-M003 Injection.
- Prior receipt of any type of gene therapy or cell therapy.
- Use of systemic immunosuppressants or steroids within 3 months prior to administration (except for prophylactic immunosuppressive therapy specified in the protocol).
- History of cancer within 5 years prior to screening, excluding completely resected non-melanoma skin cancer, non-metastatic prostate cancer and fully cured ductal carcinoma in situ.
- Received live attenuated vaccines within 4 months prior to screening, or planned to receive such vaccines during the clinical trial.
- Received treatment or intervention with other investigational drugs or study devices within 28 days or 5 half-lives (for drugs only) prior to screening, whichever is longer.
- Pregnant women (or women planning pregnancy) or breastfeeding women.
- Other conditions that, in the investigator's opinion, render the subject ineligible for study participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003
Chaohui YU, PhD, +86 13957161659, ych623@sina.com
Chaohui Yu, PhD
About this study
Official title: A Multicenter, Open, Single-arm, Single-dose, Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
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).
Read the detailed description
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 Phase I/II study consists of a main study phase and a long-term follow-up phase. The main study phase includes an 8-week screening period and a 52-week follow-up period after infusion. The long-term follow-up phase starts at Week 53 and lasts until 5 years post-infusion.
The study pre-specified two dose cohorts, consisting of one main dose cohort and one de-escalation dose cohort: Dose Cohort 1 (4.0 × 10¹³ vg/kg) and the de-escalation dose cohort (2.0 × 10¹³ vg/kg). Three subjects will be enrolled in each dose cohort.Dose Cohort 1 serves as the starting dose. After the first subject receives the study drug, a minimum 28-day DLT observation period must be completed to confirm safety before enrolling subsequent subjects.All three subjects in Dose Cohort 1 have completed the 28-day (Day 28) DLT observation period following LY-M003 infusion. The Safety Review Committee (SRC) will make a comprehensive assessment to determine whether to proceed to the dose expansion phase at the dose level of 4.0 × 10¹³ vg/kg.
- Conditions
- Wilson's Disease
- Being tested
- LY-M003 Injection
- Keywords
- Wilson's Disease, LY-M003 Injection, Gene Therapy
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY-M003 InjectionGenetic
Phase 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 InjectionGenetic
Phase 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 InjectionGenetic
Phase 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.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: LY-M003 Dose group 1 | Experimental | Not described Receives: Genetic: LY-M003 Injection |
| Phase 1: LY-M003 de-escalation dose | Experimental | Not described Receives: Genetic: LY-M003 Injection |
| Phase 2: LY-M003 Dose group 1 | Experimental | Not described Receives: Genetic: LY-M003 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Within 28 days after infusion of LY-M003 Injection
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.
Measured: 5 years
Secondary outcomes (30)
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.
Measured: Within 52 weeks after administration
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.
Measured: Within 52 weeks after administration
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.
Measured: Within 52 weeks after administration
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.
Measured: Within 52 weeks after administration
Change in serum ceruloplasmin level from baseline within 52 weeks after administration
Assessment will be based on changes in serum ceruloplasmin level from baseline.
Measured: Within 52 weeks after administration
Change in serum ceruloplasmin activity level from baseline within 52 weeks after administration
Assessment will be based on changes in erum ceruloplasmin activity level from baseline.
Measured: Within 52 weeks after administration
Change in 24-hour urinary copper content from baseline at each follow-up time point within 52 weeks after administration
Assessment will be based on changes in 24-hour urinary copper content from baseline.
Measured: Within 52 weeks after administration
Change in serum total copper from baseline within 52 weeks after administration
Assessment will be based on changes in serum total copper from baseline
Measured: Within 52 weeks after administration
Change from baseline in total score of the neurological subscale of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Assess the change from baseline in the total score of Part I of the Unified Wilson's Disease Rating Scale (UWDRS). Part I of the UWDRS is the neurological subscale, with a total score ranging from 0 to 208. Higher scores indicate more severe disease and poorer functional status.
Measured: Within 52 weeks after administration
Change from baseline in hepatic subscale score of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Assess the change from baseline in the total score of Part II of the Unified Wilson's Disease Rating Scale (UWDRS). Part II is the hepatic subscale, with a total score ranging from 0 to 36. Higher scores indicate more severe disease and poorer functional status.
Measured: Within 52 weeks after administration
Change from baseline in psychiatric subscale score of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Assess the change from baseline in the total score of Part III of the Unified Wilson's Disease Rating Scale (UWDRS). Part III is the psychiatric subscale, with a total score ranging from 0 to 76. Higher scores indicate more severe disease and poorer functional status.
Measured: Within 52 weeks after administration
Change from baseline in individual item/subscale scores (speech, handwriting, standing and gait) of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Assessment will be based on changes from baseline in individual item/subscale scores (speech, handwriting, standing and gait) of the Unified Wilson's Disease Rating Scale (UWDRS).The total score for speech, writing, standing and gait each ranges from 0 to 4. Higher scores indicate more severe conditions.
Measured: Within 52 weeks after administration
Change in ALT and AST from baseline within 52 weeks after administration
Assessment will be based on changes in ALT and AST from baseline
Measured: Within 52 weeks after administration
Change in liver stiffness from baseline within 52 weeks after administration
Assessment will be based on changes in liver stiffness from baseline.Liver stiffness is measured by liver and gallbladder ultrasonography combined with ultrasound elastography, with the unit of kPa.
- <5.8 kPa: No liver fibrosis (F0)
- 5.8 ~ 6.8 kPa: Mild liver fibrosis (F1)
- 6.8 ~ 9.1 kPa: Moderate liver fibrosis (F2)
- 9.1 ~ 10.3 kPa: Severe liver fibrosis (F3)
- >10.3 kPa: Liver cirrhosis (F4) Higher values indicate more advanced liver fibrosis or cirrhosis.
Measured: Within 52 weeks after administration
Change in Kayser-Fleischer (K-F) rings from baseline within 52 weeks after administration
Assessment will be based on changes in Kayser-Fleischer (K-F) rings from baseline
Measured: within 52 weeks after administration
Long-term follow-up study: Percentage reduction in standard of care (SoC) medication dosage after 52 weeks of administration
Assessment will be based on the percentage of participants who underwent standard dose reduction after infusion
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Number and proportion of subjects who discontinued standard of care (SoC) medications after 52 weeks of treatment
Assessment will be based on the number and percentage of participants who discontinued standard treatment medications.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Number of consecutive treatment weeks during which the above-mentioned subjects remained off SoC medications
Assessment will be based on the number of consecutive cycles during which participants discontinued standard medications.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in serum ceruloplasmin activity levels from baseline after 52 weeks of treatment
Assessment will be based on changes in erum ceruloplasmin activity level from baseline.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in serum ceruloplasmin concentration levels from baseline following 52 weeks of treatment
Assessment will be based on changes in serum ceruloplasmin level from baseline.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in non-ceruloplasmin bound copper (NCC) in serum from baseline after 52 weeks of treatment
Assessment will be based on changes in serum non-ceruloplasmin-bound copper from baseline
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in total serum copper from baseline after 52 weeks of treatment
Assessment will be based on changes in serum total copper from baseline
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes of 24-hour urinary copper levels from baseline at all follow-up visits after 52 weeks of treatment
Assessment will be based on changes in 24-hour urinary copper content from baseline.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in quantitative neurological function scores of the Unified Wilson's Disease Rating Scale (UWDRS) from baseline after 52 weeks of treatment
Assess the change from baseline in the total score of Part I of the Unified Wilson's Disease Rating Scale (UWDRS). Part I of the UWDRS is the neurological subscale, with a total score ranging from 0 to 208. Higher scores indicate more severe disease and poorer functional status.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in hepatic subscale quantitative scores of the UWDRS from baseline after 52 weeks of treatment
Assess the change from baseline in the total score of Part II of the Unified Wilson's Disease Rating Scale (UWDRS). Part II is the hepatic subscale, with a total score ranging from 0 to 36. Higher scores indicate more severe disease and poorer functional status.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in psychiatric symptom subscale quantitative scores of the UWDRS from baseline after 52 weeks of treatment
Assess the change from baseline in the total score of Part III of the Unified Wilson's Disease Rating Scale (UWDRS). Part III is the psychiatric subscale, with a total score ranging from 0 to 76. Higher scores indicate more severe disease and poorer functional status.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes from baseline in individual item and subscale scores (Speech, Handwriting, Standing and Gait) of the quantitative UWDRS after 52 weeks of treatment
Assessment will be based on changes from baseline in individual item/subscale scores (speech, handwriting, standing and gait) of the Unified Wilson's Disease Rating Scale (UWDRS).The total score for speech, writing, standing and gait each ranges from 0 to 4. Higher scores indicate more severe conditions.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in ALT and AST levels from baseline after 52 weeks of treatment
Assessment will be based on changes in ALT and AST from baseline
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in liver stiffness measurements from baseline after 52 weeks of treatment
Assessment will be based on changes in liver stiffness from baseline.Liver stiffness is measured by liver and gallbladder ultrasonography combined with ultrasound elastography, with the unit of kPa.
- <5.8 kPa: No liver fibrosis (F0)
- 5.8 ~ 6.8 kPa: Mild liver fibrosis (F1)
- 6.8 ~ 9.1 kPa: Moderate liver fibrosis (F2)
- 9.1 ~ 10.3 kPa: Severe liver fibrosis (F3)
- >10.3 kPa: Liver cirrhosis (F4) Higher values indicate more advanced liver fibrosis or cirrhosis.
Measured: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in Kayser-Fleischer (K-F) ring grading from baseline after 52 weeks of treatment
Assessment will be based on changes in Kayser-Fleischer (K-F) rings from baseline
Measured: Weeks 53 to 5 years after LY-M003 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2026
- Study start15 Jun 2026 (estimated)
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lingyi Biotech Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chaohui Yu, PhD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07641140
- Sponsor's protocol ID
- LY-M003-WD-002
- Status last verified
- Jun 2026
- First submitted
- 2 Jun 2026
- Last updated
- 2 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open, Single-arm, Single-dose, Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
- ClinicalTrials.gov ID
- NCT07641140
- Acronym / sponsor ID
- LY-M003-WD-002
- Conditions
- Wilson's Disease
- Keywords
- Wilson's Disease, LY-M003 Injection, Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Lingyi Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Jun 2026 (estimated)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 30 Dec 2032 (estimated)
- First posted
- 11 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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 Phase I/II study consists of a main study phase and a long-term follow-up phase. The main study phase includes an 8-week screening period and a 52-week follow-up period after infusion. The long-term follow-up phase starts at Week 53 and lasts until 5 years post-infusion.
The study pre-specified two dose cohorts, consisting of one main dose cohort and one de-escalation dose cohort: Dose Cohort 1 (4.0 × 10¹³ vg/kg) and the de-escalation dose cohort (2.0 × 10¹³ vg/kg). Three subjects will be enrolled in each dose cohort.Dose Cohort 1 serves as the starting dose. After the first subject receives the study drug, a minimum 28-day DLT observation period must be completed to confirm safety before enrolling subsequent subjects.All three subjects in Dose Cohort 1 have completed the 28-day (Day 28) DLT observation period following LY-M003 infusion. The Safety Review Committee (SRC) will make a comprehensive assessment to determine whether to proceed to the dose expansion phase at the dose level of 4.0 × 10¹³ vg/kg.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase 1: LY-M003 Dose group 1 | Experimental | Genetic: LY-M003 Injection |
| Phase 1: LY-M003 de-escalation dose | Experimental | Genetic: LY-M003 Injection |
| Phase 2: LY-M003 Dose group 1 | Experimental | Genetic: LY-M003 Injection |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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.
- Subjects have maintained a low-copper diet for at least 6 consecutive months prior to screening and will continue this dietary restriction throughout the study.
Key exclusion criteria (25 listed)
- 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.
- Subjects with severe hypersplenism complicated and requiring splenectomy as assessed by the investigator.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LY-M003 InjectionPhase 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. | Genetic | Phase 1: LY-M003 Dose group 1 |
| LY-M003 InjectionPhase 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. | Genetic | Phase 1: LY-M003 de-escalation dose |
| LY-M003 InjectionPhase 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. | Genetic | Phase 2: LY-M003 Dose group 1 |
Primary outcome
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.
Time frame: Within 28 days after infusion of LY-M003 Injection
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.
Time frame: 5 years
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.
30 secondary outcomes
Percentage reduction in the dosage of standard of care (SoC) medications within 52 weeks after administration.
Time frame: Within 52 weeks after administration
Number and proportion of subjects who discontinued standard of care (SoC) medications within 52 weeks after administration.
Time frame: Within 52 weeks after administration
Duration (in treatment weeks) of consecutive discontinuation of SoC medications among the above subjects.
Time frame: Within 52 weeks after administration
Change in serum non-caeruloplasmin-bound copper (NCC) from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in serum ceruloplasmin level from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in serum ceruloplasmin activity level from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in 24-hour urinary copper content from baseline at each follow-up time point within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in serum total copper from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change from baseline in total score of the neurological subscale of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change from baseline in hepatic subscale score of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change from baseline in psychiatric subscale score of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change from baseline in individual item/subscale scores (speech, handwriting, standing and gait) of the Unified Wilson's Disease Rating Scale (UWDRS) within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in ALT and AST from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in liver stiffness from baseline within 52 weeks after administration
Time frame: Within 52 weeks after administration
Change in Kayser-Fleischer (K-F) rings from baseline within 52 weeks after administration
Time frame: within 52 weeks after administration
Long-term follow-up study: Percentage reduction in standard of care (SoC) medication dosage after 52 weeks of administration
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Number and proportion of subjects who discontinued standard of care (SoC) medications after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Number of consecutive treatment weeks during which the above-mentioned subjects remained off SoC medications
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in serum ceruloplasmin activity levels from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in serum ceruloplasmin concentration levels from baseline following 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in non-ceruloplasmin bound copper (NCC) in serum from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in total serum copper from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes of 24-hour urinary copper levels from baseline at all follow-up visits after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in quantitative neurological function scores of the Unified Wilson's Disease Rating Scale (UWDRS) from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in hepatic subscale quantitative scores of the UWDRS from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in psychiatric symptom subscale quantitative scores of the UWDRS from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes from baseline in individual item and subscale scores (Speech, Handwriting, Standing and Gait) of the quantitative UWDRS after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in ALT and AST levels from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in liver stiffness measurements from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Long-term follow-up study: Changes in Kayser-Fleischer (K-F) ring grading from baseline after 52 weeks of treatment
Time frame: Weeks 53 to 5 years after LY-M003 infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chaohui Yu, PhD principal investigator
First Affiliated Hospital of Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LY-M003 Injection in people with Wilson's Disease, run by Lingyi Biotech Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rihong He (+8619121572057, rihong.he@lingyimed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: Within 28 days after infusion of LY-M003 Injection). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2026.