Phase 2 Gaucher Disease Trial, Recruiting

Written by an AI model from 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 to fully understand what the study involves and sign a consent form agreeing to take part
• You have been confirmed through lab testing to have two mutated copies of the GBA1 gene, with a significant reduction in a specific enzyme (glucocerebrosidase) activity, and have been diagnosed with Gaucher disease type 1 (GD1)
• You either: (a) have been previously treated for GD1 with enzyme replacement therapy or substrate reduction therapy and your condition is currently stable, OR (b) are newly diagnosed or untreated and have at least one of the following — low but not critically low hemoglobin (a measure of red blood cells), low but not critically low platelets, an enlarged liver, or an enlarged spleen
• If you are a woman who could become pregnant, you must have a negative pregnancy test before joining
• You and your partner agree to use effective contraception (such as abstinence or condoms) from the start of the trial until 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 tested positive for a certain type of antibody (AAV8 neutralizing antibody) above a specific level
• You have been diagnosed with Gaucher disease type 2 or type 3 (which affect the brain and nervous system)
• You have active and worsening bone disease that is expected to need surgery within the next 6 months
• You have low platelets, low red blood cells, an enlarged liver or spleen, or bone thinning caused by something other than Gaucher disease (as determined by the trial doctor)
• You have taken part in another clinical trial involving an experimental drug or device within the past 28 days (or within 5 half-lives of the drug, whichever is longer)
• You have significant liver disease or liver damage, including: a severely enlarged liver, a history of moderate-to-severe liver scarring (stage 2 or above fibrosis), certain abnormal liver blood test results, a history of heavy alcohol or drug use in the past 2 years, active hepatitis B or C infection or currently taking hepatitis B medication
• You have tested positive for HIV or syphilis
• You have severely high triglycerides (a type of fat in the blood) above a specific level
• You have an uncontrolled ongoing or infectious disease (as determined by the trial doctor)
• You have previously 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 transfusions or red blood cell transfusions within the past 3 months, or platelet transfusions within the past month
• You have a serious heart condition (such as moderate-to-severe heart failure, classified as NYHA class 3 or above)
• You have a known allergy to any ingredient in the study drug (LY-M001 injection)
• You have previously received any type of gene therapy or cell therapy
• You have used immunosuppressant medications or steroids (other than those required by the study) within the past 3 months
• You have had cancer within the past 5 years or currently have an active cancer (except for certain treated skin cancers)
• You have received a live attenuated vaccine (a type of vaccine made from a weakened live virus or bacteria) within the past 4 months, or plan to receive one during the trial
• The trial doctor has determined for any other reason that the trial is not appropriate for you (confirm with trial site)

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