NCT01233609

Trial of Oral Valproic Acid for Retinitis Pigmentosa

A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 90 (actual)
• Sponsor: Foundation Fighting Blindness · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	Valproic Acid	One to four 250mg softgels by mouth daily (dose determined by body weight)
DRUG	Placebo	Dosage per subject weight- same schedule as the active comparator

Timeline

• Start date: 2010-11-01
• Primary completion: 2015-12-01
• Completion: 2015-12-01
• First posted: 2010-11-03
• Last updated: 2017-12-02
• Results posted: 2017-12-02

Locations

6 sites across 1 country: United States