Gene therapy trial for rare heart condition halted before it began

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participant has both a clinical diagnosis of Friedreich ataxia (FA) and a documented history of genetic diagnosis of FA with either a guanine-adenine-adenine (GAA) trinucleotide repeat (TNR) expansion in intron 1 of both gene for frataxin (FXN) alleles or a GAA TNR expansion of intron 1 of one FXN allele and a pathogenic variant in the other FXN allele. * Participant has a resting LVEF ≥ 40% and \< 55% as measured at screening by ECHO. * Participant has a body mass index range of 17.0 to 30.0 kg/m2. * Participant agrees not to begin omaveloxolone treatment during the 52-week period after receiving study intervention. * Participants on omaveloxolone, who opt to discontinue omaveloxolone, may enroll if they stop omaveloxolone for 3 weeks and pass study screening, including LFTs. * Participants on omaveloxolone, who opt to stay on omaveloxolone will need to have been on it for a minimum of 3 months, with LFTs that pass diagnostic assessments exclusion criteria at screening and prior to dosing with ASP2016. Prior elevation(s) in AST/ALT associated with omaveloxolone use must be discussed with the sponsor medical monitor. If there is a liver function test (LFT) elevation after treatment, participant agrees to stop omaveloxolone treatment until 52 weeks. * Participants on omaveloxolone will need to discontinue strong or moderate cytochrome P450 3A4 (CYP3A4) inducers and inhibitors. * Woman of Child Bearing Potential (WOCBP) on omaveloxolone must use a nonhormonal, highly effective methods of contraception (e.g., nonhormonal intrauterine device system), as omaveloxolone may interfere with hormonal methods of contraception. Exclusion Criteria: * Participant has late-onset FA, defined as symptom onset after the age of 25 years. * Participant is unable to complete cardiopulmonary exercise testing (CPET) procedure. * Participant has a contraindication to endomyocardial biopsy or cardiac catheterization. * Participant has a contraindication to cardiac magnetic resonance imaging (CMRI) with contrast, including hypersensitivity to gadolinium contrast agent, cardiac pacemaker or implantable cardiac defibrillator. * Participant has an elevated titer of anti-AAV8 total antibodies, as determined by central testing. * Participant has significant fibrosis on CMRI, defined as late gadolinium enhancement of \> 15% left ventricular myocardial mass.