Beyond loss of frataxin: the complex molecular pathology of Friedreich ataxia; Evans-Galea MV, Lockhart PJ, Galea CA, Hannan AJ, Delatycki MB, Discovery Medicine [2014, 17(91):25-35]
A review about diverse array of molecular events that have been shown to influence clinical outcome in FRDA. The authors also examine additional pathogenic factors from other trinucleotide repeat diseases which could be potentially important in FRDA.
Wednesday, January 15, 2014
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients. ClinicalTrials.gov
Sponsor: Azienda Policlinico Umberto I
The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Detailed Description:
This is a Phase 2 clinical trial. A total of 10 FRDA patients will be recruited All subjects will be treated with a dose of 100-150-200-micrograms of IFN gamma 1b (Imukin®) subcutaneously, with an interval of 14 days, for a total of 3 injections.
Sponsor: Azienda Policlinico Umberto I
The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Detailed Description:
This is a Phase 2 clinical trial. A total of 10 FRDA patients will be recruited All subjects will be treated with a dose of 100-150-200-micrograms of IFN gamma 1b (Imukin®) subcutaneously, with an interval of 14 days, for a total of 3 injections.
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