Conditions: Retinitis Pigmentosa
Keywords: retinitis pigmentosa valproic acid visual field
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 90
Healthy volunteers: no

Brief summary:
The objectives of this study are to evaluate the efficacy of Valproic Acid (VPA) to both slow the progression of visual function loss and/or to restore visual function in patients with Autosomal Dominant Retinitis Pigmentosa (RP) and to collect safety and tolerability information.

Detailed description:
Retinitis Pigmentosa (RP) is an incurable and untreatable group of heterogeneous retinal degenerative diseases that cause severe visual loss. There is currently no therapeutic that substantially slows the progression of this disease, and certainly none that can restore vision in RP patients. The Valproic Acid (VPA) study is designed as a six-site, interventional, prospective, randomized, placebo controlled, double-blinded study of 90 participants to evaluate the efficacy of oral Valproic Acid to both slow the progression of visual function loss and/or to restore visual function in patients with an Autosomal Dominant RP genetic mutation and to collect safety and tolerability information. Patients that participate in the study will be randomized to either placebo or VPA in a 1:1 ratio.

Arms:
arm 1: Subjects who receive valproic acid arm 2: Subjects who receive placebo

Interventions: intervention 1: Valproic Acid intervention 2: Placebo
Intervention descriptions: intervention 1: One to four 250mg softgels by mouth daily (dose determined by body weight) intervention 2: Dosage per subject weight- same schedule as the active comparator

Protocol document: