Trial of Oral Valproic Acid for Retinitis Pigmentosa (VPA)
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|ClinicalTrials.gov Identifier: NCT01233609|
Recruitment Status : Completed
First Posted : November 3, 2010
Results First Posted : December 2, 2017
Last Update Posted : December 2, 2017
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|Condition or disease||Intervention/treatment||Phase|
|Retinitis Pigmentosa||Drug: Valproic Acid Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||90 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa|
|Study Start Date :||November 2010|
|Actual Primary Completion Date :||December 2015|
|Actual Study Completion Date :||December 2015|
Active Comparator: Valproic Acid
Subjects who receive valproic acid
Drug: Valproic Acid
One to four 250mg softgels by mouth daily (dose determined by body weight)
Other Name: Valproate
Placebo Comparator: Placebo
Subjects who receive placebo
Dosage per subject weight- same schedule as the active comparator
- Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter [ Time Frame: baseline to week 52 ]Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the III4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
- Mean Change in Visual Field Area From Baseline to 52 Weeks--I4e Isopter [ Time Frame: baseline to week 52 ]Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the I4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
- Static Perimetry by Treatment Arm--Full Field Hill of Vision [ Time Frame: baseline to week 52 ]Mean change from baseline at week 52 for Full field Hill of Vision (Static perimetry)
- Static Perimetry Volume--30 Degree Hill of Vision [ Time Frame: baseline to week 52 ]Mean Change from baseline to week 52 for Static Perimetry Volume --30 Degree Hill of Vision. Full field static perimetry protocol was followed using the Octopus 900 (Haag-Streit) for a single session for each eye.
- Mean Change From Baseline in Best Corrected Visual Acuity [ Time Frame: baseline to week 52 ]Mean change in best corrected visual acuity as assessed by ETDRS (Early Treatment Diabetic Retinopathy Study) method from baseline to week 52
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Understand/sign the IRB-approved study informed consent document.
- Age greater than or equal to 18 years, no upper age limit
- Males and non-child bearing females must weigh ≥40 Kg and ≤158.9 Kg; Females of child bearing potential must weigh ≥40 Kg and ≤74.9 Kg.
- Diagnosis of Retinitis Pigmentosa (RP).
- Visual acuity of greater than or equal to 35 letters in at least one eye as measured by the EVA-ETDRS (equivalent to 20/200 on a Snellen chart).
- Genotyped as autosomal dominant form of RP.
- Female subjects of childbearing potential and male subjects able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse or must commit to practice at least two acceptable methods of contraception to minimize the chance of pregnancy during the study and for the 13 week period after stopping the study drug.
- Female subjects of childbearing potential must have a negative urine pregnancy test at study entry and throughout the duration of the study.
- Willingness to comply with the protocol.
- Medical problems that make consistent follow-up over the treatment period unlikely (e.g. stroke, severe MI, end stage malignancy), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections.
- Other retinal diseases: Glaucoma, retinal inflammatory disease (CME is allowable), cataract worse than +2 NS, or herpes simplex virus of the eye.
- Intact visual field of 5⁰ or less.
- Subject unable to provide reliable perimetry measurements in both eyes for both static and kinetic visual field, as determined by the Reading Center.
- History of cancer (other than non-melanoma skin cancer) diagnosed, or requiring treatment within the past 2 years.
- A hemoglobin concentration, a platelet count or an absolute neutrophil count below the lower limit of normal at study entry.
- Suspected liver dysfunction determined by having liver function values elevated above the upper limit of normal.
- History of pancreatitis by clinical features and/or laboratory abnormalities in the last 12 months.
- Renal dysfunction based on serum creatinine,(MDRD) equation.
- Urea cycle disorders.
- History of neurological conditions including epilepsy, history of brain injury, encephalitis, or any organic brain syndrome.
- History of schizophrenia, schizoaffective disorder, bipolar disorder, suicidality or organic mental disorders.
- Currently receiving valproic acid or other anti-convulsants.
- Sensitive to or have ever had an allergic reaction to valproic Acid.
- Sensitive to or have ever had an allergic reaction to peanuts as peanut oil is an inactive ingredient in valproic acid capsules and the placebo.
- Has taken one of the disallowed drugs at least 2 weeks prior to randomization.
- Pregnant women.
- Lactating mothers who are breast feeding their babies.
- RP patients involved in other clinical trials within the last 3 months.
- Require enrollment by consent of a legally authorized representative.
- Persons who are unable to read are not allowed to consent for themselves or others to participate in this study.
- The potential participant lives in the same household as a current participant in this protocol.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01233609
|United States, Florida|
|University of Miami, Bascom Palmer Eye Institute|
|Miami, Florida, United States, 33136|
|United States, Michigan|
|University of Michigan, Ann Arbor|
|Ann Arbor, Michigan, United States, 48105|
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|United States, Tennessee|
|University of Tennessee, Hamilton Eye Institute|
|Memphis, Tennessee, United States, 38163|
|United States, Texas|
|Retina Foundation of the Southwest|
|Dallas, Texas, United States, 75231|
|United States, Utah|
|University of Utah School of Medicine, Moran Eye Center|
|Salt Lake City, Utah, United States, 84132|
|Study Director:||Patricia Zilliox, PhD||Foundation Fighting Blindness|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Foundation Fighting Blindness|
|Other Study ID Numbers:||
|First Posted:||November 3, 2010 Key Record Dates|
|Results First Posted:||December 2, 2017|
|Last Update Posted:||December 2, 2017|
|Last Verified:||October 2017|
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Central Nervous System Depressants