UNASSIGNED: @entity7960 is an @entity74 caused by deficiency of the protein @entity5602 . The most common genetic cause is a homozygotic expansion of GAA triplets within intron 1 of the @entity5602 gene leading to impaired transcription. Preclinical in vivo and in vitro studies have shown that interferon gamma (IFNy) is able to up-regulate the expression of @entity5602 gene in multiple cell types. We designed a phase IIa clinical trial, the first in Italy, aimed at assessing both safety and tolerability of IFNy in Friedreich's @entity1 and ability to increase @entity5602 levels in peripheral blood mononuclear cells. Nine @entity1 (6 female and 3 males aged 21-38  years) with genetically confirmed disease were given 3 subcutaneous escalating doses (100, 150 and 200   g) of IFNy ( @entity1 recombinant interferon 1 @entity6434 , trade name IMUKIN(  )), over 4  weeks. The primary end-point was the assessment of the safety and tolerability of IFNy by means of standard clinical and hematological criteria. The secondary end-point was the detection of changes of @entity5602 levels in peripheral blood mononuclear cells after each single escalating dose of the drug. IFNy was generally well tolerated, the main adverse event was @entity221 /fever. Although, increases in @entity5602 levels could be detected in a minority of @entity1 , these changes were not significant. A large phase III multicenter, randomized clinical trial with IFNy in @entity7960 @entity1 is currently ongoing. This study is expected to conclusively address the clinical efficacy of IFNy therapy in @entity1 with @entity7960 .

GIFT-1, a phase IIa clinical trial to test the safety and efficacy of IFNy administration in @entity7960 XXXX .

@entity7960 :: ('MESH:D005621', 'Disease') :: ["Friedreich's ataxia"] @entity1 :: ('9606', 'Species') :: ['human', 'patients'] @entity6434 :: ('347962', 'Species') :: ['b gamma'] @entity74 :: ('MESH:D030342', 'Disease') :: ['autosomal recessive progressive degenerative disorder'] @entity5602 :: ('2395', 'Gene') :: ['frataxin'] @entity221 :: ('MESH:D005334', 'Disease') :: ['hyperthermia']
@entity1 :: (9606,Species) :: ['patients']
