Clinical Trial: Trial of Oral Valproic Acid for Retinitis Pigmentosa

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary: 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.
Sponsor: Foundation Fighting Blindness Clinical Research Institute

Current Primary Outcome: Mean change in visual field area from baseline to 52 weeks. [ Time Frame: Screening, baseline, week 26, and week 52 ]

Mean change in visual field area from baseline to 52 weeks. Visual field area will be measured using semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haig-Strait).


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Static perimetry measurements in the full visual field [ Time Frame: Screening, baseline, week 26, and week 52 ]
    Static perimetry measurements in the full visual field, including the central 30 field.
  • Mean change in best corrected visual acuity. [ Time Frame: Baseline to Week 52 ]
    Mean change in best corrected visual acuity as measured by Electronic Visual Acuity-Early Treatment Diabetic Retinopathy Study (EVA-ETDRS) visual acuity test.
  • Color contrast sensitivity [ Time Frame: Baseline to 52 weeks ]
    Color contrast sensitivity as measured by ChromaTest.
  • Mean change in peak Electroretinogram (ERG) amplitude [ Time Frame: Baseline to 52 weeks ]
    Mean change in peak Electroretinogram (ERG) amplitude
  • Mean change in central macular thickness and macular volume [ Time Frame: Baseline to 52 weeks ]
    Mean change in central macular thickness and mean change in macular volume as measured by Optical Coherence Tomography (OCT).
  • Change in fundus appearance [ Time Frame: Baseline to 52 weeks ]
    Change in fundus appearance as judged by color retinal photography.
  • Mean change in total score on vision-related quality of life. [ Time Frame: Baseline to 52 weeks ]
    Mean change in total score on vision-related quality of life as measured by the National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25).
  • Changes in clinical laboratory data [ Time Frame: Baseline through 15 months ]
    Changes in clinical laboratory data
  • Changes in physical examination findings [ Time Frame: Baseline through 15 months ]
    Changes in physical examination findings.


Original Secondary Outcome:

  • Static perimetry measurements in the full visual field [ Time Frame: Screening, baseline, week 26, and week 52 ]
    Static perimetry measurements in the full visual field, including the central 30 field.
  • Mean change in best corrected visual acuity. [ Time Frame: Baseline to Week 52 ]
    Mean change in best corrected visual acuity as measured by EVA-ETDRS.
  • Color contrast sensitivity [ Time Frame: Baseline to 52 weeks ]
    Color contrast sensitivity as measured by ChromaTest.
  • Mean change in peak ERG amplitude [ Time Frame: Baseline to 52 weeks ]
    Mean change in peak ERG amplitude
  • Mean change in central macular thickness and macular volume [ Time Frame: Baseline to 52 weeks ]
    Mean change in central mauclar thickness and mean change in macular volume as measured by OCT.
  • Change in fundus appearance [ Time Frame: Baseline to 52 weeks ]
    Change in fundus appearance as judged by color retinal photography.
  • Mean change in total score on vision-related quality of life. [ Time Frame: Baseline to 52 weeks ]
    Mean change in total score on vision-related quality of life as measured by the NEI-VFQ25.
  • Changes in clinical laboratory data [ Time Frame: Baseline through 15 months ]
    Cahnges in clinical laboratory data
  • Changes in physical examination findings [ Time Frame: Baseline through 15 months ]
    Cahnges in physical examination findings.


Information By: Foundation Fighting Blindness Clinical Research Institute

Dates:
Date Received: November 1, 2010
Date Started: November 2010
Date Completion:
Last Updated: March 21, 2016
Last Verified: March 2016