A Fabry Disease Gene Therapy Study

Phase 1/2 Terminated 3 enrolled Spur Therapeutics

NaSingle-groupOpen-labelTreatment

Summary

This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.

Timeline

Start: 2019-07-08
Primary completion: 2023-05-02
Completion: 2023-05-02

Outcome

Outcome not reported

Stopped: “Freeline has decided to pause development of FLT190 in Fabry disease to focus its resources on advancing FLT201.”

Publications

Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	FLT190	Gene therapy (AAV / viral vector)	—	Intravenous