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Tests treatment safety and results for Wilson Disease
Official title A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
ClinicalTrials.gov ID: NCT06650319
What this study is testing
What is LY-M003?
LY-M003 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for wilson disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 10 to 60
You may be able to join if
- The subject must be able to fully understood the purpose, nature, method, and possible adverse effects of the study, must be able to voluntarily...
- Patients diagnosed with Wilson Disease .
- Wilson Disease (WD) patients confirmed by laboratory tests to have biallelic mutations in the ATP7B gene.
- people must be treatment-experienced to WD who have received standard treatment (eg, D-penicillamine or zinc acetate) for at least 6 months prior to...
- people must restrict food with high copper content for at least 6 months prior to screening and continue this restriction during the entire duration...
You likely can't join if
- 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...
- people with other liver diseases as determined by the investigator, such as immune hepatitis, alcoholic liver disease, primary biliary cholangitis...
- people considered as complicated with severe hypersplenism and requiring splenectomy as judged by the investigator.
- Model for End-Stage Liver Disease (MELD) Score \> 13.
See the full eligibility criteria
- 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...
- Patients diagnosed with Wilson Disease .
- Wilson Disease (WD) patients confirmed by laboratory tests to have biallelic mutations in the ATP7B gene.
- people 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.
- people must restrict food with high copper content for at least 6 months prior to screening and continue this restriction during the entire duration of study participation.
- people must be willing to refrain from donating blood, organs, tissues or cells during study participation.
- Negative pregnancy test in women of childbearing potential (WOCBP).
- people and their partners who have no childbearing plans from the screening period to 6 months after the end of the study and are willing to adopt effective contraceptive measures (e.g., abstinence, condoms, etc.)...
- 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.
- people 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
- people considered as complicated with severe hypersplenism and requiring splenectomy as judged by the investigator.
- Model for End-Stage Liver Disease (MELD) Score \> 13.
- Other disorders of copper metabolism, such as chronic cholestatic liver diseases, disorders of glycosylation, copper metabolism disorders, etc.
- History of noncompliance with copper chelators or zinc agents within 6 months prior to screening, as determined by the investigator.
- people with treatment-experienced WD who have ALT and/or AST 5 times greater than the upper limit of normal (ULN).
- Severe central nervous system symptoms urgent for intensive hospitalization judged by the investigator.
- Hemoglobin \< 90 g/L.
- A history of epileptic seizures or other diseases that may potentially affect compliance with study procedures within 6 months prior to the screening period.
- Hepatitis B surface antigen (HBsAg) positive, hepatitis C virus (HCV) antibody positive, human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
- people with end-stage renal disease receiving dialysis (chronic kidney disease stage 3 and above) or creatinine clearance \< 60 mL/min.
- Severe hyperlipidemia (triglycerides \> 1000 mg/dL).
- Subject received or plans to receive bone marrow transplantation, hematopoietic stem cell transplantation and/or major organ transplantation, including but not limited to liver transplantation, kidney transplantation...
- Clinically diagnosed or judged as serious cardiovascular disease by the investigator (eg, classification of heart failure ≥ 3 according to New York Heart Association [NYHA]).
- Patients with uncontrolled concomitant diseases or infectious diseases as judged by the investigator.
- people who have hypersensitivity to any component of LY-M003 injection.
- people who have previously received gene therapy or cell therapy of any kind.
- people who use systemic immunosuppressive agents or receive steroid therapy within 3 months prior to dosing (except for prophylactic immunosuppressive therapy as specified in protocol).
- people with history of cancer within 5 years prior to screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer and completely cured ductal carcinoma in situ.
- people who have vaccinated with attenuated live vaccine within 4 months prior to screening or plan to receive a live attenuated vaccine during the clinical trial.
- people who have received treatment or disposition with another investigational drug or investigational device within 28 days or 5 half-lives (drug only), whichever is longer, prior to screening.
- Pregnant women (or women planning to become pregnant) or lactating women.
- Other circumstances in which the investigator deems the subject inappropriate for study participation.
The study team makes the final eligibility decision.
Where it's taking place
- Hangzhou, Zhejiang, China
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 10 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hangzhou, Zhejiang, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.