EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2013
The aim of the research is to investigate the safety and efficacy of EPI-743 treatment in patients with Cbl-C defect and related visual and neurological impairment. Primary Endpoints will be the improvement in visual function as assessed by visual acuity and eye-hand coordination and manual dexterity. Secondary Endpoints will be the improvement in neurologic function, evaluated by a battery of age-appropriated psychophysical tests, and/or in objective electrophysiological tests such as Visual Evoked potentials (VEP) and Electroretinogram (ERG) and/or the change in serum markers of redox state.
Epistemonikos ID: 1eeb940419139752068b6ca0fc183fb3b7a3ce25
First added on: May 11, 2024