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Trial of Oral Valproic Acid for Retinitis Pigmentosa (VPA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01233609
Recruitment Status : Completed
First Posted : November 3, 2010
Results First Posted : December 2, 2017
Last Update Posted : December 2, 2017
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
Foundation Fighting Blindness

Study Description
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Brief Summary:
The objectives of this study are to evaluate the efficacy of Valproic Acid (VPA) to both slow the progression of visual function loss and/or to restore visual function in patients with Autosomal Dominant Retinitis Pigmentosa (RP) and to collect safety and tolerability information.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Drug: Valproic Acid Drug: Placebo Phase 2

Detailed Description:
Retinitis Pigmentosa (RP) is an incurable and untreatable group of heterogeneous retinal degenerative diseases that cause severe visual loss. There is currently no therapeutic that substantially slows the progression of this disease, and certainly none that can restore vision in RP patients. The Valproic Acid (VPA) study is designed as a six-site, interventional, prospective, randomized, placebo controlled, double-blinded study of 90 participants to evaluate the efficacy of oral Valproic Acid to both slow the progression of visual function loss and/or to restore visual function in patients with an Autosomal Dominant RP genetic mutation and to collect safety and tolerability information. Patients that participate in the study will be randomized to either placebo or VPA in a 1:1 ratio.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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
 Drug: Placebo
Dosage per subject weight- same schedule as the active comparator


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. 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)

  3. 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.

  4. 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


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Understand/sign the IRB-approved study informed consent document.
  2. Age greater than or equal to 18 years, no upper age limit
  3. 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.
  4. Diagnosis of Retinitis Pigmentosa (RP).
  5. 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).
  6. Genotyped as autosomal dominant form of RP.
  7. 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.
  8. Female subjects of childbearing potential must have a negative urine pregnancy test at study entry and throughout the duration of the study.
  9. Willingness to comply with the protocol.

Exclusion Criteria:

  1. 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.
  2. Other retinal diseases: Glaucoma, retinal inflammatory disease (CME is allowable), cataract worse than +2 NS, or herpes simplex virus of the eye.
  3. Intact visual field of 5⁰ or less.
  4. Subject unable to provide reliable perimetry measurements in both eyes for both static and kinetic visual field, as determined by the Reading Center.
  5. Diabetes.
  6. History of cancer (other than non-melanoma skin cancer) diagnosed, or requiring treatment within the past 2 years.
  7. A hemoglobin concentration, a platelet count or an absolute neutrophil count below the lower limit of normal at study entry.
  8. Suspected liver dysfunction determined by having liver function values elevated above the upper limit of normal.
  9. History of pancreatitis by clinical features and/or laboratory abnormalities in the last 12 months.
  10. Renal dysfunction based on serum creatinine,(MDRD) equation.
  11. Urea cycle disorders.
  12. History of neurological conditions including epilepsy, history of brain injury, encephalitis, or any organic brain syndrome.
  13. History of schizophrenia, schizoaffective disorder, bipolar disorder, suicidality or organic mental disorders.
  14. Currently receiving valproic acid or other anti-convulsants.
  15. Sensitive to or have ever had an allergic reaction to valproic Acid.
  16. 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.
  17. Has taken one of the disallowed drugs at least 2 weeks prior to randomization.
  18. Pregnant women.
  19. Lactating mothers who are breast feeding their babies.
  20. RP patients involved in other clinical trials within the last 3 months.
  21. Require enrollment by consent of a legally authorized representative.
  22. Persons who are unable to read are not allowed to consent for themselves or others to participate in this study.
  23. The potential participant lives in the same household as a current participant in this protocol.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Foundation Fighting Blindness
United States Department of Defense
Investigators
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Study Director: Patricia Zilliox, PhD Foundation Fighting Blindness
More Information
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Additional Information:

Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Foundation Fighting Blindness
ClinicalTrials.gov Identifier: NCT01233609    
Other Study ID Numbers: H-13371
First Posted: November 3, 2010    Key Record Dates
Results First Posted: December 2, 2017
Last Update Posted: December 2, 2017
Last Verified: October 2017
Keywords provided by Foundation Fighting Blindness:
retinitis pigmentosa
valproic acid
visual field
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn
Valproic Acid
Anticonvulsants
 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