LTFU study of Adeno-associated Viral Vector (FLT201) in Patients with Gaucher Disease (GALILEO-2)
Research type
Research Study
Full title
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects with Gaucher Disease (GALILEO-2)
IRAS ID
1009742
Contact name
Joshua Cheal
Contact email
Sponsor organisation
Freeline Therapeutics Limited
Eudract number
2024-511172-33
Research summary
Gaucher disease is a rare genetic disorder caused by the inactivity of an enzyme, glucosylceramidase-β1 (GCase), this leads to the build-up of lipids (fats) such as glucosylsphingosine (lyso-gb1) (GB1) and other glycolipids in the liver, spleen, lungs and bone.
The standard of care for patients with Gaucher disease is long-term parenteral enzyme replacement therapy (ERT), which aims to increase the level of functional GCase enzyme to reduce the build-up of lipids, or substrate reduction therapy (SRT), which aims to reduce the amount of GB1 that is produced in the body. However, the response to these forms of treatment is varied and not always successful.
At this time, there is currently no licensed advanced therapy investigational medicinal product (ATIMP) available for the treatment of any form of Gaucher disease.
The main purpose of this FLT201-02 study is to investigate the long-term safety of the study medication, FLT-201, in patients with Gaucher Disease Type 1. This study will investigate how long a patient’s body continues to make the glucocerebrosidase (GCase) enzyme following the FLT201 treatment, how the immune system reacts to FLT201, and the health of the liver, spleen, lungs, and bone. FLT201 is an ATIMP, encoded to produce the functional GCase enzyme in the liver, this will enter the blood and break down the lipids in the liver, spleen, lungs and bone.
FLT201 has been given to animals with a form of Gaucher disease, and the animals receiving FLT201 showed an increase in functional GCase enzyme activity – leading to the breakdown of lipids in the body.
Participants who have already received the FLT201 treatment in the preceding FLT201-01 study will be able to take part in the FLT201-02 study. FLT201 will not be administered to participants in this study. Participation in this study will last up to 4 years and 3 months (5 years post-treatment of FLT-201). In addition, up to 75 participants will take part in this study at globally.REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0423
Date of REC Opinion
19 Jul 2024
REC opinion
Further Information Favourable Opinion