Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

NCT ID: NCT01399515

Last Updated: 2016-04-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 200 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-03-31
• Study Completion Date: 2015-11-30

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 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.

Conditions

Retinitis Pigmentosa; Retinal Diseases; Eye Diseases; Eye Disease, Hereditary; Retinal Degeneration

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Valproic acid

Group Type: ACTIVE_COMPARATOR

Valproic Acid

Intervention Type: DRUG

One 500mg tablet by mouth daily

Control

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Valproic Acid

One 500mg tablet by mouth daily

Intervention Type: DRUG

Other Intervention Names

Valproate

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Seoul National University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Hyeong Gon Yu

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Hyeong Gon Yu, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Department of Ophthalmology, Seoul National University Hospital

Locations

Department of Ophthalmology, Seoul National University Hospital

Seoul, , South Korea

Countries

South Korea

Other Identifiers

SNUH_OT_VPA

Identifier Type: -

Identifier Source: org_study_id