Case-control study for the evaluation of the effect of valproic acid for retinitis pigmentosa

• Register: KCT
• Last refreshed on: 27 April 2013
• Main ID: KCT0000310
• Date of registration: 04/01/2012
• Primary sponsor: SEOUL NATIONAL UNIVERSITY HOSPITAL
• Public title: Case-control study for the evaluation of the effect of valproic acid for retinitis pigmentosa
• Scientific title: Evaluation of the effect of valproic acid for retinitis Pigmentosa
• Date of first enrolment: 2011-02-17
• Target sample size: 196
• Recruitment status: Recruiting
• URL: http://cris.nih.go.kr/cris/en/search/search_result_st01.jsp?seq=1732
• Study type: Interventional Study
• Study design: Primary Purpose : Treatment, Intervention Model : Parallel, Blinding/Masking : Open, Allocation : Non-RCT

Countries of recruitment

Korea, Republic of

Contacts

• Name: Cinoo Kim
• Affiliation: Department of Ophthalmology, Seoul National University Hospital

• Name: Hyeong Gon Yu
• Affiliation: Department of Ophthalmology, Seoul National University Hospital

Key inclusion & exclusion criteria

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.

Age minimum: 18(Year)
Age maximum: N/A(No Limit)
Gender: Both

Health Condition(s) or Problem(s) studied

Diseases of the eye and adnexa

Intervention(s)

Drug : Patients that participate in the study will be assigned to either valproic acid group or control in a 3:1 ratio. Enrolled subjects in valproic acid group will be treated with oral valproic acid 500mg daily for 48 weeks, and safety will be monitored every 12 week. Enrolled subjects in control group will be observed without any medication.

Primary Outcome(s)

Mean change in visual field area

Secondary Outcome(s)

Changes in clinical laboratory data: CBC, BUN, Creatinine, Liver panel (Cholesterol, Total protein, Albumin, Total bilirubin, Alkaline phosphatase, AST, ALT, GGT), Coagulation panel (PT INR, PT%, PT

Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude

Mean change in best corrected visual acuity (BCVA)

Mean change in central macular thickness

Mean change in central macular volume

Mean change in fundus appearance

Mean change in total score on vision-related quality of life

Occurrence of adverse effect related to Valproic acid

Secondary ID(s)

NCT01399515

Secondary Sponsor(s)

SEOUL NATIONAL UNIVERSITY HOSPITAL