Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Summary

Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy. Recently, a study reported that interferon gamma (IFN-g) could raise frataxin protein levels in both cell lines derived from patients with Friedreich ataxia and in a mouse model with Friedreich ataxia. The present study will test whether IFN-g is safe, tolerated and potentially efficacious in a heterogeneous cohort of children with FRDA.

Timeline

Start

2013-08

Primary completion

2014-03

Completion

2014-10

Drugs

Indications