Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia

Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia. ClinicalTrials.gov (October 17, 2013).

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.

Sponsor: Children's Hospital of Philadelphia
Collaborators: Friedreich Ataxia Research Alliance (FARA), Vidara Therapeutics Research Ltd
Information provided by (Responsible Party): Children's Hospital of Philadelphia