Gaucher 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:

• You are between 18 and 60 years old (male or female)
• You are willing and able to understand what the study involves, including possible side effects, and sign a consent form
• You have been diagnosed with Type 1 Gaucher disease, confirmed by lab tests showing two mutations in the relevant gene and reduced enzyme activity to less than 30% of normal levels
• If you have previously received treatment specifically for Gaucher disease, enough time has passed since your last dose (based on the medication's half-life)
• If you are a woman who could become pregnant, you have a negative pregnancy test before joining
• You and your partner agree to use effective contraception during the study and for 6 months after it ends, and you do not plan to donate sperm or eggs during this time
• You agree not to donate blood during the study and for at least 1 year after it ends

Who may not be able to join:

• You have a strongly positive test result for a certain type of antibody against AAV8 (a component used in the therapy)
• You are suspected to have Type 2 or Type 3 Gaucher disease
• You have active, worsening bone disease that is likely to need surgery within the next 6 months
• You have low platelets, anaemia, an enlarged liver or spleen, or osteoporosis that is not related to Gaucher disease
• You have taken part in another clinical trial or used an experimental drug or device within roughly the past 28 days or longer (depending on the drug)
• You have signs of significant liver disease, including an enlarged liver, liver scarring (stage 2 or higher), abnormal liver blood test results, a history of alcohol or drug misuse in the past 2 years, or certain hepatitis B or C infections
• You test positive for HIV or syphilis
• You have severely high triglyceride (fat) levels in your blood (above 1000 mg/dL)
• You have another serious or uncontrolled illness or active infection (as judged by the trial doctor)
• You have had your spleen removed, or are scheduled to have it removed during the study
• Your general health and ability to perform daily activities falls below a certain level on a standard medical scoring scale (confirm with trial site)
• You 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)
• You have received certain blood-boosting treatments or blood transfusions within the past 1 to 3 months before screening
• You have been diagnosed with significant heart disease, such as moderate-to-severe heart failure
• You have a known allergy to any ingredient in the study treatment (LY-M001 Injection)
• You have previously received any type of gene therapy or cell therapy
• You have used immunosuppressant medications or steroids (not related to trial requirements) within the past 3 months
• You have had a cancer diagnosis within the past 5 years (with some exceptions for certain fully treated skin cancers — confirm with trial site)
• You have received a live vaccine within the past 4 months, or plan to receive one during the trial
• The trial doctor has another reason to believe this study is not right for you

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