Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Status Completed

Clinical Trial 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.

Clinical Trial Description

Study Objectives: Primary: • To assess the effect of Interferon Gamma-1b (IFN-g) on increasing frataxin expression and protein in children with FRDA. Secondary: - To assess the effect of IFN-g on neurological outcomes (FARS, performance measures, and hearing) in subjects with FRDA. - To assess the effectiveness of IFN-g on quality of life in subjects with FRDA. - To assess the safety and tolerability of IFN-g at the currently approved dose in the FRDA population. Study Phases: Screening - During screening, subjects will be assessed for inclusion and exclusion criteria. Intervention - Subjects will begin treatment at baseline visit and the dose of study medication will be increased to the maximum dose over four weeks. The subjects will be maintained at the maximum dose for 8 weeks. After 12 weeks, treatment will stop. Study medication will be administered via subcutaneous injections three times per week for 12 weeks. Follow-up - Follow-up visits will occur at 7 and 28 days after the subject has completed the 12 weeks of active treatment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01965327
Study type	Interventional
Source	Children's Hospital of Philadelphia
Contact
Status	Completed
Phase	Phase 2
Start date	August 2013
Completion date	October 2014

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See also
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