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

Seoul National University

Status and phase

Completed

Phase 2

Conditions

Retinal Degeneration

Eye Disease, Hereditary

Retinitis Pigmentosa

Eye Diseases

Retinal Diseases

Treatments

Drug: Valproic Acid

Study type

Interventional

Funder types

Other

Identifiers

NCT01399515

SNUH_OT_VPA

Details and patient eligibility

About

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.

Full 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.

Enrollment

200 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.