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This non-randomized, multicenter Phase 3 study examines the gene therapy FLT201 in adults with Gaucher disease type 1 who have been on stable enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The aim is to confirm the efficacy and safety of FLT201 after discontinuation of ERT/SRT. Participants must be 18 or older with a clinical diagnosis of Gaucher disease type 1 and stable hemoglobin and platelet levels at baseline.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * Aged ≥18 years at time of screening. * Clinical diagnosis of Gaucher disease type 1 * Stable hemoglobin concentration at baseline * Stable platelet count at baseline * Receiving ERT or SRT without interruption for at least 2 years Key Exclusion Criteria: * Diagnosed or suspected Gaucher disease type 2 or type 3 * Positive for AAVS3 neutralizing antibodies. * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Positive pregnancy test or lactating * History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant. * History of receiving any gene therapy or cell therapy. * History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply