Clinical Trial: Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

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

Official Title:

Brief Summary:

The purpose of this study is to evaluate the efficacy and safety of oral valproic acid to slow the progression of visual function and/or to improve the visual function in patients with retinitis pigmentosa (RP).

Enrolled subjects in valproic acid group will be treated with oral valproic acid 500mg daily for 48 weeks. Visual function and safety will be assess before and after treatment (48 weeks) between valproic acid and control groups.


Detailed Summary: This study is designed as a single-site, interventional, prospective, non-randomized, controlled study of 200 participants. Patients that participate in the study will be assigned to either valproic acid group or control in a 3:1 ratio.
Sponsor: Seoul National University Hospital

Current Primary Outcome: Mean change in visual field area from baseline to 48 weeks [ Time Frame: Baseline, week 24, and week 48 ]

Visual field area will be measured using kinetic perimetry (Goldmann perimetry) or static perimetry including the central 30 field.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Mean change in best corrected visual acuity (BCVA) [ Time Frame: Baseline, week 24, and week 48 ]
    BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
  • Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude [ Time Frame: Baseline and week 48 ]
  • Mean change in central macular thickness [ Time Frame: Baseline, week 24, and week 48 ]
    Central macular thickness as measured by Optical Coherence Tomography (OCT)
  • Mean change in fundus appearance [ Time Frame: Baseline and week 48 ]
    Fundus appearance as judged by color fundus photography
  • Mean change in total score on vision-related quality of life [ Time Frame: Baseline and week 48 ]
    Total score on vision-related quality of life as measured by the National Eye Institute Visual Function Questionnaire (NEI-VFQ25)
  • Occurrence of adverse effect related to Valproic acid [ Time Frame: Baseline through 48 weeks ]
  • Changes in clinical laboratory data [ Time Frame: Baseline through 48 weeks ]
    CBC, BUN, Creatinine, Liver panel (Cholesterol, Total protein, Albumin, Total bilirubin, Alkaline phosphatase, AST, ALT, GGT), Coagulation panel (PT INR, PT%, PT sec, aPTT, Fibrinogen), Electrolyte panel (Na, K, Cl, TCO2)
  • Mean change in central macular volume [ Time Frame: Baseline, week 24, and week 48 ]
    Central macular volume as measured by Optical Coherence Tomography (OCT)


Original Secondary Outcome: Same as current

Information By: Seoul National University Hospital

Dates:
Date Received: May 3, 2011
Date Started: March 2011
Date Completion:
Last Updated: April 12, 2016
Last Verified: April 2016