NCT01399515

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Mar 2011

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2011

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

May 3, 2011

Completed
3 months until next milestone

First Posted

Study publicly available on registry

July 21, 2011

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2013

Completed
2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2015

Completed
Last Updated

April 14, 2016

Status Verified

April 1, 2016

Enrollment Period

2.4 years

First QC Date

May 3, 2011

Last Update Submit

April 12, 2016

Conditions

Retinitis PigmentosaRetinal DiseasesEye DiseasesEye Disease, HereditaryRetinal Degeneration

Keywords

Retinitis pigmentosaValproic acid

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (8)

  • Mean change in best corrected visual acuity (BCVA)

    Baseline, week 24, and week 48

  • Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude

    Baseline and week 48

  • Mean change in central macular thickness

    Baseline, week 24, and week 48

  • Mean change in fundus appearance

    Baseline and week 48

  • Mean change in total score on vision-related quality of life

    Baseline and week 48

  • +3 more secondary outcomes

Study Arms (2)

Valproic acid

ACTIVE COMPARATOR
Drug: Valproic Acid

Control

NO INTERVENTION

Interventions

Valproic AcidDRUG

One 500mg tablet by mouth daily

Also known as: Valproate
Valproic acid

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Ophthalmology, Seoul National University Hospital

Seoul, 110-744, South Korea

Location

MeSH Terms

Conditions

Retinitis PigmentosaRetinal DiseasesEye DiseasesEye Diseases, HereditaryRetinal Degeneration

Interventions

Valproic Acid

Condition Hierarchy (Ancestors)

Retinal DystrophiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pentanoic AcidsValeratesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsFatty Acids, VolatileFatty AcidsLipids

Study Officials

  • Hyeong Gon Yu, MD, PhD

    Department of Ophthalmology, Seoul National University Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

May 3, 2011

First Posted

July 21, 2011

Study Start

March 1, 2011

Primary Completion

August 1, 2013

Study Completion

November 1, 2015

Last Updated

April 14, 2016

Record last verified: 2016-04

Locations

KR(1)