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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
90
One to four 250mg softgels by mouth daily (dose determined by body weight)
Dosage per subject weight- same schedule as the active comparator
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
University of Michigan, Ann Arbor
Ann Arbor, Michigan, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Tennessee, Hamilton Eye Institute
Memphis, Tennessee, United States
Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the III4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
Time frame: baseline to week 52
Mean Change in Visual Field Area From Baseline to 52 Weeks--I4e Isopter
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the I4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
Time frame: baseline to week 52
Static Perimetry by Treatment Arm--Full Field Hill of Vision
Mean change from baseline at week 52 for Full field Hill of Vision (Static perimetry)
Time frame: baseline to week 52
Static Perimetry Volume--30 Degree Hill of Vision
Mean Change from baseline to week 52 for Static Perimetry Volume --30 Degree Hill of Vision. Full field static perimetry protocol was followed using the Octopus 900 (Haag-Streit) for a single session for each eye.
Time frame: baseline to week 52
Mean Change From Baseline in Best Corrected Visual Acuity
Mean change in best corrected visual acuity as assessed by ETDRS (Early Treatment Diabetic Retinopathy Study) method from baseline to week 52
Time frame: baseline to week 52
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Retina Foundation of the Southwest
Dallas, Texas, United States
University of Utah School of Medicine, Moran Eye Center
Salt Lake City, Utah, United States