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 18 years old or older at the time you give your consent to join the study.
• You have been confirmed (by central testing) to have two faulty copies of the GBA1 gene, which causes Gaucher disease.
• You have been taking an enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years, and have been on a stable, maximum tolerated dose for at least 3 months before the screening visit.
• You are able to understand and sign the consent forms and follow the study rules.
• You are male or female; however, anyone who could potentially become pregnant must use a highly effective form of birth control consistently throughout the entire study period.
• You agree not to donate blood, tissue, or organs for the entire duration of the study, including the long-term follow-up period.

Who may not be able to join:

• You have noticeable or significant problems with your nervous system, such as neurological symptoms or behavioral changes.
• You have active, worsening bone disease that doctors expect will need surgery within the next 6 months.
• You have had your entire spleen removed, or are planning to have it fully removed within the first 18 months of the study. (Having part of the spleen removed is not automatically disqualifying.)
• Your spleen is very enlarged — more than 10 times its normal size, as measured by a central MRI scan.
• You have significant liver disease, a fragile liver, or a history of exposure to substances that can damage the liver.
• Your platelet count (cells that help blood clot) is very low — below 40,000 per microliter of blood.
• You have severely high triglyceride (fat) levels in your blood — above 1,000 mg/dL.
• You currently have an unstable or serious heart or cardiovascular condition, as judged by the study doctor.
• You have been diagnosed with certain types of cancer within the past 5 years before screening (confirm with trial site for which cancers apply).
• You have another illness, condition, or treatment that the study doctor believes would put you at unacceptable risk or prevent you from completing the study.
• You are a woman who could become pregnant and are currently pregnant, breastfeeding, or planning to become pregnant during the study.
• You have used any Gaucher disease "chaperone therapy" (a specific type of treatment) within 4 weeks before screening, or may need to start one within the first 18 months of the study.
• You have previously received any type of gene therapy or cell therapy.
• You are currently taking systemic immune-suppressing medications or steroids (unless specifically required by the study protocol).
• You have taken part in another clinical drug or device study within the past 3 months, or within 5 half-lives of the last study drug — whichever is longer.
• You have high levels of antibodies against a specific virus (AAV9) used in the study, above a ratio of 1:40, as measured by a central laboratory.
• You have significantly abnormal results on blood or other laboratory tests at the time of screening.
• You are unable to have an MRI scan — for example, due to claustrophobia or having certain metal implants or a pacemaker in your body.

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