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

Primary Purpose

Retinitis Pigmentosa, Retinal Diseases, Eye Diseases

Status
Completed
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Valproic Acid
Sponsored by
Seoul National University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring Retinitis pigmentosa, Valproic acid

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Department of Ophthalmology, Seoul National University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

No Intervention

Arm Label

Valproic acid

Control

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Mean change in best corrected visual acuity (BCVA)
BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude
Mean change in central macular thickness
Central macular thickness as measured by Optical Coherence Tomography (OCT)
Mean change in fundus appearance
Fundus appearance as judged by color fundus photography
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)
Occurrence of adverse effect related to Valproic acid
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)
Mean change in central macular volume
Central macular volume as measured by Optical Coherence Tomography (OCT)

Full Information

First Posted
May 3, 2011
Last Updated
April 12, 2016
Sponsor
Seoul National University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT01399515
Brief Title
Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
Acronym
VPA_RP
Study Type
Interventional

2. Study Status

Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
March 2011 (undefined)
Primary Completion Date
August 2013 (Actual)
Study Completion Date
November 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Seoul National University Hospital

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa, Retinal Diseases, Eye Diseases, Eye Disease, Hereditary, Retinal Degeneration
Keywords
Retinitis pigmentosa, Valproic acid

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
200 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Valproic acid
Arm Type
Active Comparator
Arm Title
Control
Arm Type
No Intervention
Intervention Type
Drug
Intervention Name(s)
Valproic Acid
Other Intervention Name(s)
Valproate
Intervention Description
One 500mg tablet by mouth daily
Primary Outcome Measure Information:
Title
Mean change in visual field area from baseline to 48 weeks
Description
Visual field area will be measured using kinetic perimetry (Goldmann perimetry) or static perimetry including the central 30 field.
Time Frame
Baseline, week 24, and week 48
Secondary Outcome Measure Information:
Title
Mean change in best corrected visual acuity (BCVA)
Description
BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
Time Frame
Baseline, week 24, and week 48
Title
Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude
Time Frame
Baseline and week 48
Title
Mean change in central macular thickness
Description
Central macular thickness as measured by Optical Coherence Tomography (OCT)
Time Frame
Baseline, week 24, and week 48
Title
Mean change in fundus appearance
Description
Fundus appearance as judged by color fundus photography
Time Frame
Baseline and week 48
Title
Mean change in total score on vision-related quality of life
Description
Total score on vision-related quality of life as measured by the National Eye Institute Visual Function Questionnaire (NEI-VFQ25)
Time Frame
Baseline and week 48
Title
Occurrence of adverse effect related to Valproic acid
Time Frame
Baseline through 48 weeks
Title
Changes in clinical laboratory data
Description
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)
Time Frame
Baseline through 48 weeks
Title
Mean change in central macular volume
Description
Central macular volume as measured by Optical Coherence Tomography (OCT)
Time Frame
Baseline, week 24, and week 48

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyeong Gon Yu, MD, PhD
Organizational Affiliation
Department of Ophthalmology, Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Ophthalmology, Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of

12. IPD Sharing Statement

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Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

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