Early Phase 1 Wilson Disease Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who are able to fully understand what the study involves, including its purpose, methods, and possible side effects, and who are willing to take part and sign a consent form.
• People who have been diagnosed with Wilson Disease.
• People whose Wilson Disease has been confirmed by laboratory testing showing mutations in both copies of the ATP7B gene.
• People who have already been receiving standard Wilson Disease treatment (such as D-penicillamine or zinc acetate) for at least 6 months before the screening period.
• People who have been avoiding foods high in copper for at least 6 months before screening and are willing to continue doing so throughout the entire study.
• People who are willing to avoid donating blood, organs, tissues, or cells for the whole time they are in the study.
• Women who could potentially become pregnant must have a negative pregnancy test result.
• People (and their partners) who do not plan to have children from the screening period until 6 months after the study ends, and who are willing to use effective contraception (such as abstinence or condoms); people must also have no plans to donate sperm or eggs during this time.

Who may not be able to join:

• People with high levels of antibodies against a specific virus called AAV8 (neutralising antibody level greater than 1:10).
• People who have had active bleeding in the digestive system (gastrointestinal bleeding) within the past 3 months.
• People whose liver disease has reached an advanced or decompensated stage, showing complications such as high blood pressure in the liver's blood vessels, fluid build-up in the abdomen, an enlarged spleen, swollen veins in the oesophagus, or confusion caused by liver failure.
• People who have other liver conditions (such as autoimmune hepatitis, alcohol-related liver disease, primary biliary cholangitis, primary sclerosing cholangitis, or liver damage caused by drugs or toxins), as determined by the study doctor.
• People whose enlarged spleen is considered severe enough that removal of the spleen may be needed, as judged by the study doctor.
• People with a liver disease severity score (called the MELD score) greater than 13.
• People who have other conditions affecting how the body processes copper, such as certain chronic liver or metabolic disorders.
• People who have not consistently followed their prescribed copper-lowering treatment in the 6 months before screening, as determined by the study doctor.
• People who have been on Wilson Disease treatment and whose liver enzyme levels (ALT and/or AST) are more than 5 times the upper limit of the normal range.
• People with severe neurological (brain and nervous system) symptoms that require urgent intensive hospital care, as judged by the study doctor.
• People with a haemoglobin level below 90 g/L (a measure of low red blood cell count).
• People who have had epileptic seizures or other conditions that might affect their ability to follow study procedures within the 6 months before screening.
• People who test positive for hepatitis B, hepatitis C, HIV, or syphilis.
• People with severe kidney disease who are on dialysis, have chronic kidney disease at stage 3 or above, or whose kidney filtration rate is below 60 mL/min.
• People with severely elevated triglycerides (blood fats) above 1000 mg/dL.
• People who have had, or are planning to have, a bone marrow transplant, stem cell transplant, or major organ transplant (such as a liver or kidney transplant).
• People with serious heart disease, such as heart failure classified as grade 3 or higher according to a standard medical scale (NYHA classification), as determined by the study doctor.
• People with other uncontrolled ongoing illnesses or active infections, as judged by the study doctor.
• People who have a known allergy or sensitivity to any ingredient in the study treatment (LY-M003 injection).
• People who have previously received any type of gene therapy or cell therapy.
• People who have taken immune-suppressing medications or steroid treatments (other than any specifically allowed by the study plan) within 3 months before receiving the study treatment.
• People who have had a cancer diagnosis within the past 5 years, with the exception of certain fully treated skin cancers, non-spreading prostate cancer, or fully cured early-stage breast duct cancer.
• People who have received a live attenuated vaccine within 4 months before screening, or who plan to receive one during the trial.
• People who have taken part in another clinical trial or used another experimental drug or device within 28 days (or 5 half-lives of the drug, whichever is longer) before screening.
• Women who are pregnant, planning to become pregnant, or who are currently breastfeeding.
• People whom the study doctor considers unsuitable to participate for any other reason.

Important: Always verify eligibility with the trial site directly before applying.