Randomized, double-blind, placebo-controlled study of interferon-γ 1b in Friedreich Ataxia.
Ann Clin Transl Neurol
; 6(3): 546-553, 2019 03.
Article
in En
| MEDLINE
| ID: mdl-30911578
ABSTRACT
Objective:
In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN-γ 1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN-γ 1b in the treatment of Friedreich Ataxia through a double-blind, multicenter, placebo-controlled trial.Methods:
Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN-γ 1b or placebo for 6 months. The primary outcome measure was the modified Friedreich Ataxia Rating Scale (mFARS).Results:
No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open-label extension period, subjects had a more stable course than expected based on natural history data.Conclusions:
This study provides no direct evidence for a beneficial effect of IFN-γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Friedreich Ataxia
/
Interferon-gamma
Type of study:
Clinical_trials
Limits:
Adolescent
/
Adult
/
Child
/
Female
/
Humans
/
Male
Language:
En
Journal:
Ann Clin Transl Neurol
Year:
2019
Document type:
Article