Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

Retinitis Pigmentosa Clinical Trial

— VPA_RP  

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01399515
• Other study ID #: SNUH_OT_VPA
• Status: Completed
• Phase: Phase 2
• First received: May 3, 2011
• Last updated: April 12, 2016
• Start date: March 2011
• Est. completion date: November 2015

Study information

• Verified date: April 2016
• Source: Seoul National University Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 200
• Est. completion date: November 2015
• Est. primary completion date: August 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

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

Intervention

Drug:
• Valproic Acid
One 500mg tablet by mouth daily

Locations

Country	Name	City	State
Korea, Republic of	Department of Ophthalmology, Seoul National University Hospital	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Seoul National University Hospital

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change in visual field area from baseline to 48 weeks	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	No
Secondary	Mean change in best corrected visual acuity (BCVA)	BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)	Baseline, week 24, and week 48	No
Secondary	Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude		Baseline and week 48	No
Secondary	Mean change in central macular thickness	Central macular thickness as measured by Optical Coherence Tomography (OCT)	Baseline, week 24, and week 48	No
Secondary	Mean change in fundus appearance	Fundus appearance as judged by color fundus photography	Baseline and week 48	No
Secondary	Mean change in total score on vision-related quality of life	Total score on vision-related quality of life as measured by the National Eye Institute Visual Function Questionnaire (NEI-VFQ25)	Baseline and week 48	No
Secondary	Occurrence of adverse effect related to Valproic acid		Baseline through 48 weeks	Yes
Secondary	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 sec, aPTT, Fibrinogen), Electrolyte panel (Na, K, Cl, TCO2)	Baseline through 48 weeks	Yes
Secondary	Mean change in central macular volume	Central macular volume as measured by Optical Coherence Tomography (OCT)	Baseline, week 24, and week 48	No