Interferon Gamma-1b in Friedreich Ataxia (FRDA)

• Status: Completed
• Trial end date: 2014-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Philadelphia
• Collaborators: Friedreich's Ataxia Research Alliance; Vidara Therapeutics Research Ltd
• Treatments: Interferon-gamma; Interferons

Criteria

Inclusion Criteria:

• Subjects with FRDA confirmed by genetic testing with 2 expanded Guanine-adenine-adenine repeats

• Females who are not pregnant or breast feeding, and who do not intend to become pregnant. Females of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at screening

• Stable doses of all medications, vitamins and supplements for 30 days prior to study entry and for the duration of the study

• Parent/guardian permission (informed consent) and child assent

Exclusion Criteria:

• Any unstable illness that in the investigator's opinion precludes participation in this study

• Use of any investigational product within 30 days prior to enrollment

• Subjects with a history of substance abuse

• Presence of clinically significant cardiac disease

• History of hypersensitivity to IFN-g or E. coli derived products

• Presence of severe renal disease or hepatic disease

• Clinically significant abnormal White blood cell count, hemoglobin or platelet count

• Any subject planning a scheduled surgical procedure during the study