Priya Kishnani, MD, Professor and Chief of the Division of Medical Genetics at Duke University, discusses the GALILEO-3 clinical trial of FLT201 in adults with Gaucher disease type 1 (GD1).
GD1 is the most common form of Gaucher disease. It is caused by a deficiency of glucocerebrosidase (GBA), an important enzyme that breaks down a fatty chemical called glucocerebroside. Because the body cannot break down this chemical, fat-filled Gaucher cells build up in areas like the spleen, liver and bone marrow. Unlike type 2 and 3, GD1 does not usually involve the central nervous system. Symptoms of GD1 include enlarged spleen and liver, low blood cell counts, bleeding problems and bone disease. Gaucher disease is caused by changes in the GBA gene.
It was recently announced that the first patient has been dosed in the GALILEO-3 clinical trial (NCT07223944). This study is a phase 3 non-randomized, global, multicenter study evaluating the safety and efficacy of FLT201in adult patients with GD1 currently on stable standard-of-care therapies.
The study hopes to enroll approximately 45 adults with GD1 who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years. Participants will receive a single intravenous infusion of FLT201 alongside a prophylactic immunosuppressive regimen.
The primary endpoint is the proportion of participants maintaining stable hemoglobin concentration at week 52. Secondary endpoints include safety and tolerability, stability of platelet counts, spleen and liver volumes, biomarker changes, bone marrow burden and mineral density changes, and patient-reported quality-of-life outcomes.
FLT201 is a proprietary, liver-tropic capsid and an engineered beta-glucocerebrosidase (GCase85), designed to deliver continuous, sustained enzyme exposure beyond what is achievable with ERT. This allows for a low vector genome dose, necessary for a favorable safety profile. FLT201 has both Orphan Drug and RMAT (regenerative medicine advanced therapy) designations from the FDA.
Clinical trial eligibility is as follows:
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
To learn more about GD1 and other rare metabolic conditions, visit https://checkrare.com/diseases/metabolic-disorders/