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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01399515
Recruitment Status : Completed
First Posted : July 21, 2011
Last Update Posted : April 14, 2016
Information provided by (Responsible Party):
Hyeong Gon Yu, Seoul National University Hospital

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.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Retinal Diseases Eye Diseases Eye Disease, Hereditary Retinal Degeneration Drug: Valproic Acid Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 200 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : March 2011
Actual Primary Completion Date : August 2013
Actual Study Completion Date : November 2015

Arm Intervention/treatment
Active Comparator: Valproic acid Drug: Valproic Acid
One 500mg tablet by mouth daily
Other Name: Valproate

No Intervention: Control

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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)

  2. Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude [ Time Frame: Baseline and week 48 ]
  3. Mean change in central macular thickness [ Time Frame: Baseline, week 24, and week 48 ]
    Central macular thickness as measured by Optical Coherence Tomography (OCT)

  4. Mean change in fundus appearance [ Time Frame: Baseline and week 48 ]
    Fundus appearance as judged by color fundus photography

  5. 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)

  6. Occurrence of adverse effect related to Valproic acid [ Time Frame: Baseline through 48 weeks ]
  7. 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)

  8. Mean change in central macular volume [ Time Frame: Baseline, week 24, and week 48 ]
    Central macular volume as measured by Optical Coherence Tomography (OCT)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01399515

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Korea, Republic of
Department of Ophthalmology, Seoul National University Hospital
Seoul, Korea, Republic of, 110-744
Sponsors and Collaborators
Seoul National University Hospital
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Principal Investigator: Hyeong Gon Yu, MD, PhD Department of Ophthalmology, Seoul National University Hospital

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Responsible Party: Hyeong Gon Yu, Professor, Seoul National University Hospital Identifier: NCT01399515     History of Changes
Other Study ID Numbers: SNUH_OT_VPA
First Posted: July 21, 2011    Key Record Dates
Last Update Posted: April 14, 2016
Last Verified: April 2016

Keywords provided by Hyeong Gon Yu, Seoul National University Hospital:
Retinitis pigmentosa
Valproic acid

Additional relevant MeSH terms:
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Eye Diseases
Retinal Diseases
Genetic Diseases, Inborn
Eye Diseases, Hereditary
Retinitis Pigmentosa
Retinal Degeneration
Retinal Dystrophies
Valproic Acid
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs