Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Completed · Phase 2
Conditions studied: Friedreich Ataxia
In brief
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.
Key facts
- Study ID
- NCT01965327
- Run by
- Children's Hospital of Philadelphia
- People needed
- 12
- Starts
- 2013-08-01
- Expected to finish
- 2014-10-01
- Last updated by the study team
- 2021-04-13
Who can join
Age: 5 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Full record on ClinicalTrials.gov
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