Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa (VPA_RP)
This study is ongoing, but not recruiting participants.
Sponsor:
Seoul National University Hospital
Information provided by (Responsible Party):
Hyeong Gon Yu, Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01399515
First received: May 3, 2011
Last updated: April 17, 2013
Last verified: April 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Retinitis Pigmentosa Retinal Diseases Eye Diseases Eye Disease, Hereditary Retinal Degeneration |
Drug: Valproic Acid |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
Resource links provided by NLM:
Genetics Home Reference related topics:
Lenz microphthalmia syndrome
oculofaciocardiodental syndrome
Peters plus syndrome
retinitis pigmentosa
X-linked juvenile retinoschisis
U.S. FDA Resources
Further study details as provided by Seoul National University Hospital:
Primary Outcome Measures:
- Mean change in visual field area from baseline to 48 weeks [ Time Frame: Baseline, week 24, and week 48 ] [ Designated as safety issue: No ]Visual field area will be measured using kinetic perimetry (Goldmann perimetry) or static perimetry including the central 30 field.
Secondary Outcome Measures:
- Mean change in best corrected visual acuity (BCVA) [ Time Frame: Baseline, week 24, and week 48 ] [ Designated as safety issue: No ]BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
- Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude [ Time Frame: Baseline and week 48 ] [ Designated as safety issue: No ]
- Mean change in central macular thickness [ Time Frame: Baseline, week 24, and week 48 ] [ Designated as safety issue: No ]Central macular thickness as measured by Optical Coherence Tomography (OCT)
- Mean change in fundus appearance [ Time Frame: Baseline and week 48 ] [ Designated as safety issue: No ]Fundus appearance as judged by color fundus photography
- Mean change in total score on vision-related quality of life [ Time Frame: Baseline and week 48 ] [ Designated as safety issue: No ]Total score on vision-related quality of life as measured by the National Eye Institute Visual Function Questionnaire (NEI-VFQ25)
- Occurrence of adverse effect related to Valproic acid [ Time Frame: Baseline through 48 weeks ] [ Designated as safety issue: Yes ]
- Changes in clinical laboratory data [ Time Frame: Baseline through 48 weeks ] [ Designated as safety issue: Yes ]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)
- Mean change in central macular volume [ Time Frame: Baseline, week 24, and week 48 ] [ Designated as safety issue: No ]Central macular volume as measured by Optical Coherence Tomography (OCT)
| Estimated Enrollment: | 200 |
| Study Start Date: | March 2011 |
| Estimated Study Completion Date: | August 2013 |
| Estimated Primary Completion Date: | August 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: Valproic acid |
Drug: Valproic Acid
One 500mg tablet by mouth daily
Other Name: Valproate
|
| No Intervention: Control |
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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01399515
Locations
| Korea, Republic of | |
| Department of Ophthalmology, Seoul National University Hospital | |
| Seoul, Korea, Republic of, 110-744 | |
Sponsors and Collaborators
Seoul National University Hospital
Investigators
| Principal Investigator: | Hyeong Gon Yu, MD, PhD | Department of Ophthalmology, Seoul National University Hospital |
More Information
No publications provided
| Responsible Party: | Hyeong Gon Yu, Professor, Seoul National University Hospital |
| ClinicalTrials.gov Identifier: | NCT01399515 History of Changes |
| Other Study ID Numbers: | SNUH_OT_VPA |
| Study First Received: | May 3, 2011 |
| Last Updated: | April 17, 2013 |
| Health Authority: | Korea: Institutional Review Board |
Keywords provided by Seoul National University Hospital:
|
Retinitis pigmentosa Valproic acid |
Additional relevant MeSH terms:
|
Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Retinal Degeneration Retinal Diseases Retinitis Retinitis Pigmentosa Retinal Dystrophies Valproic Acid Anticonvulsants Central Nervous System Agents |
Therapeutic Uses Pharmacologic Actions Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action GABA Agents Neurotransmitter Agents Physiological Effects of Drugs Antimanic Agents Tranquilizing Agents Central Nervous System Depressants Psychotropic Drugs |
ClinicalTrials.gov processed this record on May 16, 2013