U.S., March 25 -- ClinicalTrials.gov registry received information related to the study (NCT06891443) titled 'Study to Evaluate Sepofarsen in Subjects with Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)' on Feb. 03.
Brief Summary: The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290.
Study Start Date: March, 2025
Study Type: INTERVENTIONAL
Condition:
Leber Congenital Amaurosis 10
Blindness
Leber Congenital Amaurosis
Sensation Disorders
Vision Disorder
Neurological Manifestations
Eye Diseases, Hereditary
Eye Diseases
Eye Disor...