A Gaucher Disease Gene Therapy Trial with FLT201 (GALILEO-3)
Research type
Research Study
Full title
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1
IRAS ID
1011569
Contact name
Joshua Cheal
Contact email
Sponsor organisation
Spur Therapeutics Limited
Research summary
Gaucher disease is caused by mutations in a gene called β-glucosylceramidase (GBA1). The mutations in the GBA1 gene stop it working properly and reduce the amount of a protein called ‘GCase’ in the body. This can cause build-up of fatty substances, resulting in problems in the organs, fatigue, increased bruising and bleeding, bone pain, and breathing problems. Existing treatments either involve frequent infusions (delivering the medicine through a vein) or taking oral medication. These treatments may have side effects, can be inconvenient, and do not cure Gaucher disease (they must be taken long-term).
FLT201 is a gene therapy that provides the body with a working copy of the GBA1 gene. The results of the previous clinical trial with FLT201 (trial number FLT201-01) showed the potential of FLT201 as a treatment for Gaucher disease type 1. This trial aims to confirm the long-term effectiveness and safety of FLT201 in adults with Gaucher disease type 1.The trial will enrol around 45 participants. All enrolled participants will receive a one-time treatment with FLT201. Participants will be followed for 5 years during which time safety and effectiveness of FLT201 will be assessed.
Participants will be 18 years of age or older with Gaucher disease type 1 and have a low level of GCase. Participants must have stable haemoglobin concentration and platelet counts and have received ERT/SRT for a least 2 years.
FLT201 will be given as a slow injection through a needle into a vein (known as an intravenous infusion). Participants will receive corticosteroids from Week 3 to Week 12 after FLT201 treatment to manage the risk of reactions (e.g., liver injury) caused by the immune system.The trial design, FLT201 dose, and immune suppression plan have been chosen to make sure the treatment works as well as possible. The overall balance of risks and potential benefits for participants in this trial is expected to be at least as favourable as existing alternative therapies.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
25/LO/0840
Date of REC Opinion
4 Feb 2026
REC opinion
Further Information Favourable Opinion