Retinitis PigmentosaPhase 2VPA_RPNCT01399515
Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
A Phase 2 study testing Valproic Acid in people with Retinitis Pigmentosa and Retinal Diseases, run by Seoul National University Hospital at 1 site in South Korea.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Retinal Diseases, Eye Diseases
- What is being tested?
- Valproic Acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteSouth Korea
- Study dates
- Mar 2011to Nov 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Department of Ophthalmology, Seoul National University Hospital
Seoul, 110-744
About this study
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.
Read the 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.
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Eye Diseases, Eye Disease, Hereditary, Retinal Degeneration
- Being tested
- Valproic Acid
- Keywords
- Retinitis pigmentosa, Valproic acid
- Medical subject headings
- Retinitis Pigmentosa, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valproic AcidDrug
Also called Valproate
One 500mg tablet by mouth daily
| Study group | Role | What they receive |
|---|---|---|
| Valproic acid | Active comparator | Not described Receives: Drug: Valproic Acid |
| Control | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, week 24, and week 48
Secondary outcomes (8)
Mean change in best corrected visual acuity (BCVA)
BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
Measured: Baseline, week 24, and week 48
Mean change in 30-Hz flicker Electroretinogram (ERG) amplitude
Measured: Baseline and week 48
Mean change in central macular thickness
Central macular thickness as measured by Optical Coherence Tomography (OCT)
Measured: Baseline, week 24, and week 48
Mean change in fundus appearance
Fundus appearance as judged by color fundus photography
Measured: Baseline and week 48
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)
Measured: Baseline and week 48
Occurrence of adverse effect related to Valproic acid
Measured: Baseline through 48 weeks
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)
Measured: Baseline through 48 weeks
Mean change in central macular volume
Central macular volume as measured by Optical Coherence Tomography (OCT)
Measured: Baseline, week 24, and week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2011 (estimated)
- Study startMar 2011
- Primary completionAug 2013
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by Seoul National University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seoul National University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hyeong Gon Yu, Seoul National University Hospital
- Investigators
- Hyeong Gon Yu, MD, PhD, Principal investigator, Department of Ophthalmology, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01399515
- Sponsor's protocol ID
- SNUH_OT_VPA
- Acronym
- VPA_RP
- Status last verified
- Apr 2016
- First submitted
- 3 May 2011
- Last updated
- 14 Apr 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT01399515
- Acronym / sponsor ID
- VPA_RP / SNUH_OT_VPA
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Eye Diseases, Eye Disease, Hereditary, Retinal Degeneration
- Keywords
- Retinitis pigmentosa, Valproic acid
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Seoul National University HospitalUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Aug 2013 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 21 Jul 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Valproic acid | Active comparator | Drug: Valproic Acid |
| Control | No intervention | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Valproic AcidAlso: ValproateOne 500mg tablet by mouth daily | Drug | Valproic acid |
Primary outcome
Mean change in visual field area from baseline to 48 weeks
Time frame: Baseline, week 24, and week 48
Visual field area will be measured using kinetic perimetry (Goldmann perimetry) or static perimetry including the central 30 field.
8 secondary outcomes
Mean change in best corrected visual acuity (BCVA)
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 central macular thickness
Time frame: Baseline, week 24, and week 48
Mean change in fundus appearance
Time frame: Baseline and week 48
Mean change in total score on vision-related quality of life
Time frame: Baseline and week 48
Occurrence of adverse effect related to Valproic acid
Time frame: Baseline through 48 weeks
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hyeong Gon Yu, MD, PhD principal investigator
Department of Ophthalmology, Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Valproic Acid in people with Retinitis Pigmentosa and Retinal Diseases, run by Seoul National University Hospital at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change in visual field area from baseline to 48 weeks (time frame: Baseline, week 24, and week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2016.