Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa (VPA_RP)

April 12, 2016 updated by: Hyeong Gon Yu, Seoul National University Hospital

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

200

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Korea, Republic of
      • Seoul, Korea, Republic of, 110-744
        • Department of Ophthalmology, Seoul National University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosis of retinitis pigmentosa (RP) established by night blindness, visual field constriction, marked reduction of electroretinogram, and the clinical signs of RP in fundus examination
  • Best corrected visual acuity of 20/200 or more on a Snellen chart in at least one eye
  • Intact visual field of 5 or more as measured by the kinetic perimetry
  • Understand and sign the IRB-approved informed consent document for the study
  • Body weight: male (40 kg to 100 kg), female (40 kg to 80 kg)
  • Must be able to swallow tablets
  • Female subjects of childbearing potential must commit to practice acceptable methods of contraception

Exclusion Criteria:

  • Pregnant women
  • Lactating mothers
  • Medical problems that make consistent follow-up over the treatment period unlikely (e.g., stroke, myocardiac infarction, malignancy) or severe systemic disease
  • Other ocular disease: retinal disease other than RP or cystoid macular edema, glaucoma, cataract worse than +2PSC or infectious corneal disease
  • Coagulation disorder or bleeding-tendency
  • Liver dysfunction
  • Renal dysfunction
  • History of pancreatitis
  • History of neurological disorders including epilepsy, history of brain injury or any organic brain disorders
  • History of mental disorders including schizophrenia, bipolar disorder, or suicidality
  • Currently receiving valproic acid or other anti-convulsants
  • Has taken one of the following drugs at least 4 weeks prior to enrollment as these drugs are specifically known to affect the progression of RP: vitamin A, lutein, omega-3 fatty acid, or any antioxidant which affect the blood flow of retina or retinal function.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Control
Active Comparator: Valproic acid
Drug: Valproic Acid
One 500mg tablet by mouth daily
Other Names:
  • Valproate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline, week 24, and week 48

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Baseline, week 24, and week 48
Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude
Time Frame: Baseline and week 48
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)
Baseline, week 24, and week 48
Mean change in fundus appearance
Time Frame: Baseline and week 48
Fundus appearance as judged by color fundus photography
Baseline and week 48
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)
Baseline and week 48
Occurrence of adverse effect related to Valproic acid
Time Frame: Baseline through 48 weeks
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)
Baseline through 48 weeks
Mean change in central macular volume
Time Frame: Baseline, week 24, and week 48
Central macular volume as measured by Optical Coherence Tomography (OCT)
Baseline, week 24, and week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Seoul National University Hospital

Investigators

  • Principal Investigator: Hyeong Gon Yu, MD, PhD, Department of Ophthalmology, Seoul National University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2011

Primary Completion (Actual)

August 1, 2013

Study Completion (Actual)

November 1, 2015

Study Registration Dates

First Submitted

May 3, 2011

First Submitted That Met QC Criteria

July 20, 2011

First Posted (Estimate)

July 21, 2011

Study Record Updates

Last Update Posted (Estimate)

April 14, 2016

Last Update Submitted That Met QC Criteria

April 12, 2016

Last Verified

April 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNUH_OT_VPA

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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