Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

• Register: ClinicalTrials.gov
• Last refreshed on: 25 April 2016
• Main ID: NCT01399515
• Date of registration: 03/05/2011
• Prospective Registration: No
• Primary sponsor: Seoul National University Hospital
• Public title: Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa VPA_RP
• Date of first enrolment: March 2011
• Target sample size: 200
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01399515
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Korea, Republic of

Contacts

• Name: Hyeong Gon Yu, MD, PhD
• 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 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Eye Disease, Hereditary

Retinitis Pigmentosa

Eye Diseases

Retinal Diseases

Retinal Degeneration

Intervention(s)

Drug: Valproic Acid

Primary Outcome(s)

Mean change in visual field area from baseline to 48 weeks [Time Frame: Baseline, week 24, and week 48]

Secondary Outcome(s)

Changes in clinical laboratory data [Time Frame: Baseline through 48 weeks]

Mean change in central macular volume [Time Frame: Baseline, week 24, and week 48]

Mean change in best corrected visual acuity (BCVA) [Time Frame: Baseline, week 24, and week 48]

Mean change in central macular thickness [Time Frame: Baseline, week 24, and week 48]

Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude [Time Frame: Baseline and week 48]

Mean change in total score on vision-related quality of life [Time Frame: Baseline and week 48]

Mean change in fundus appearance [Time Frame: Baseline and week 48]

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

Secondary ID(s)

SNUH_OT_VPA

Source(s) of Monetary Support

Please refer to primary and secondary sponsors