Page last updated: 2024-10-26

valproic acid and Retinitis Pigmentosa

valproic acid has been researched along with Retinitis Pigmentosa in 16 studies

Valproic Acid: A fatty acid with anticonvulsant and anti-manic properties that is used in the treatment of EPILEPSY and BIPOLAR DISORDER. The mechanisms of its therapeutic actions are not well understood. It may act by increasing GAMMA-AMINOBUTYRIC ACID levels in the brain or by altering the properties of VOLTAGE-GATED SODIUM CHANNELS.
valproic acid : A branched-chain saturated fatty acid that comprises of a propyl substituent on a pentanoic acid stem.

Retinitis Pigmentosa: Hereditary, progressive degeneration of the retina due to death of ROD PHOTORECEPTORS initially and subsequent death of CONE PHOTORECEPTORS. It is characterized by deposition of pigment in the retina.

Research Excerpts

ExcerptRelevanceReference
"To evaluate the potential efficacy and assess the safety of orally administered valproic acid (VPA) in the treatment of autosomal dominant retinitis pigmentosa."9.27Effect of Oral Valproic Acid vs Placebo for Vision Loss in Patients With Autosomal Dominant Retinitis Pigmentosa: A Randomized Phase 2 Multicenter Placebo-Controlled Clinical Trial. ( Bernstein, PS; Birch, DG; Bramer, S; Chegarnov, E; Csaky, K; Erker, L; Fish, GE; Francis, PJ; Hartnett, ME; Heckenlively, J; Hughbanks-Wheaton, DK; Iannacone, A; Jayasundera, T; Lam, BL; Lindblad, R; McCormack, J; Pennesi, ME; Rose, S; Sklaver, NL; Smith, T; Teske, MP; VanVeldhuisen, PC; Wahle, A; Warner, J; Weleber, RG; Winthrop, KL; Yang, P; Zilliox, P, 2018)
"To evaluate the efficacy of valproic acid (VPA) on visual function in patients with retinitis pigmentosa (RP)."9.19Efficacy of oral valproic acid in patients with retinitis pigmentosa. ( Chohan, A; Gogia, V; Gupta, S; Kumar, A; Midha, N; Sehra, S, 2014)
"To evaluate the efficacy and safety of valproic acid (VPA) treatment in patients with retinitis pigmentosa (RP)."7.85The adverse effects of valproic acid on visual functions in the treatment of retinitis pigmentosa. ( Dervişogulları, MS; Güler, E; Totan, Y; Yüce, A, 2017)
"To examine the efficacy and safety of valproic acid (VPA) in patients with retinitis pigmentosa (RP)."7.77Therapeutic potential of valproic acid for retinitis pigmentosa. ( Bigelow, C; Checchi, JM; Clemson, CM; Kaushal, S; Krebs, M; Tzekov, R, 2011)
"VPA plays a complex role in patients with pigmentary retinal dystrophies and may be associated with VA and field decline as well as adverse side effects."5.39Long-term follow-up for efficacy and safety of treatment of retinitis pigmentosa with valproic acid. ( Bhalla, S; Bhullar, S; Joshi, D; Kasuga, D; Kay, CN; Park, Y, 2013)
"To evaluate the potential efficacy and assess the safety of orally administered valproic acid (VPA) in the treatment of autosomal dominant retinitis pigmentosa."5.27Effect of Oral Valproic Acid vs Placebo for Vision Loss in Patients With Autosomal Dominant Retinitis Pigmentosa: A Randomized Phase 2 Multicenter Placebo-Controlled Clinical Trial. ( Bernstein, PS; Birch, DG; Bramer, S; Chegarnov, E; Csaky, K; Erker, L; Fish, GE; Francis, PJ; Hartnett, ME; Heckenlively, J; Hughbanks-Wheaton, DK; Iannacone, A; Jayasundera, T; Lam, BL; Lindblad, R; McCormack, J; Pennesi, ME; Rose, S; Sklaver, NL; Smith, T; Teske, MP; VanVeldhuisen, PC; Wahle, A; Warner, J; Weleber, RG; Winthrop, KL; Yang, P; Zilliox, P, 2018)
"Data for this observational case study were collected at the Department of Ophthalmology, University of California, Los Angeles, from May 1 to July 30, 2015, and included SD-OCT images of a subset of patients from the Trial of Oral Valproic Acid for Retinitis Pigmentosa."5.22Quantification of Ellipsoid Zone Changes in Retinitis Pigmentosa Using en Face Spectral Domain-Optical Coherence Tomography. ( Birch, DG; Ferris, FL; Francis, P; Hariri, AH; Ho, A; Sadda, SR; Weleber, RG; Zhang, HY, 2016)
"To evaluate the efficacy of valproic acid (VPA) on visual function in patients with retinitis pigmentosa (RP)."5.19Efficacy of oral valproic acid in patients with retinitis pigmentosa. ( Chohan, A; Gogia, V; Gupta, S; Kumar, A; Midha, N; Sehra, S, 2014)
"To evaluate the efficacy and safety of valproic acid (VPA) treatment in patients with retinitis pigmentosa (RP)."3.85The adverse effects of valproic acid on visual functions in the treatment of retinitis pigmentosa. ( Dervişogulları, MS; Güler, E; Totan, Y; Yüce, A, 2017)
"To examine the efficacy and safety of valproic acid (VPA) in patients with retinitis pigmentosa (RP)."3.77Therapeutic potential of valproic acid for retinitis pigmentosa. ( Bigelow, C; Checchi, JM; Clemson, CM; Kaushal, S; Krebs, M; Tzekov, R, 2011)
"VPA plays a complex role in patients with pigmentary retinal dystrophies and may be associated with VA and field decline as well as adverse side effects."1.39Long-term follow-up for efficacy and safety of treatment of retinitis pigmentosa with valproic acid. ( Bhalla, S; Bhullar, S; Joshi, D; Kasuga, D; Kay, CN; Park, Y, 2013)

Research

Studies (16)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's15 (93.75)24.3611
2020's1 (6.25)2.80

Authors

AuthorsStudies
Razzaghi, N1
Fernandez-Gonzalez, P1
Mas-Sanchez, A1
Vila-Julià, G1
Perez, JJ1
Garriga, P1
Vent-Schmidt, RYJ1
Wen, RH1
Zong, Z1
Chiu, CN1
Tam, BM1
May, CG1
Moritz, OL1
Todd, L1
Zelinka, C1
Totan, Y1
Güler, E1
Yüce, A1
Dervişogulları, MS1
Huang, XF1
Birch, DG2
Bernstein, PS1
Iannacone, A1
Pennesi, ME1
Lam, BL1
Heckenlively, J1
Csaky, K1
Hartnett, ME1
Winthrop, KL1
Jayasundera, T1
Hughbanks-Wheaton, DK1
Warner, J1
Yang, P1
Fish, GE1
Teske, MP1
Sklaver, NL1
Erker, L1
Chegarnov, E1
Smith, T1
Wahle, A1
VanVeldhuisen, PC1
McCormack, J1
Lindblad, R1
Bramer, S1
Rose, S2
Zilliox, P2
Francis, PJ2
Weleber, RG2
Brooks, BP1
Jeffrey, B1
Bhalla, S1
Joshi, D1
Bhullar, S1
Kasuga, D1
Park, Y1
Kay, CN1
Kumar, A1
Midha, N1
Gogia, V1
Gupta, S1
Sehra, S1
Chohan, A1
Hariri, AH1
Zhang, HY1
Ho, A1
Francis, P1
Ferris, FL1
Sadda, SR1
Clemson, CM1
Tzekov, R2
Krebs, M2
Checchi, JM1
Bigelow, C2
Kaushal, S2
van Schooneveld, MJ1
van den Born, LI1
van Genderen, M1
Bollemeijer, JG1
Sandberg, MA1
Rosner, B1
Weigel-DiFranco, C1
Berson, EL1
Clemson, C1
Checchi, J1
Sisk, RA1

Clinical Trials (2)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa[NCT01233609]Phase 290 participants (Actual)Interventional2010-11-30Completed
NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa[NCT05537220]Phase 3438 participants (Anticipated)Interventional2023-10-11Recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

Mean Change From Baseline in Best Corrected Visual Acuity

Mean change in best corrected visual acuity as assessed by ETDRS (Early Treatment Diabetic Retinopathy Study) method from baseline to week 52 (NCT01233609)
Timeframe: baseline to week 52

Interventionletters read correctly (Mean)
Valproic Acid -- Right Eye-1.4
Valproic Acid--Left Eye0.0
Placebo --Right Eye0.2
Placebo --Left Eye1.3

Mean Change in Visual Field Area From Baseline to 52 Weeks--I4e Isopter

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 (NCT01233609)
Timeframe: baseline to week 52

InterventionVisual field area (degrees squared) (Mean)
Placebo--Right Eye80.9
Placebo--Left Eye115.7
Valproic Acid--Right Eye5.3
Valproic Acid--Left Eye19.5

Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter

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 (NCT01233609)
Timeframe: baseline to week 52

InterventionVisual field area (degrees squared) (Mean)
Placebo--Right Eye-122.9
Placebo--Left Eye-112.0
Valproic Acid--Right Eye-293.7
Valproic Acid--Left Eye-237.1

Static Perimetry by Treatment Arm--Full Field Hill of Vision

Mean change from baseline at week 52 for Full field Hill of Vision (Static perimetry) (NCT01233609)
Timeframe: baseline to week 52

Interventiondb-steridians (Mean)
Placebo--Right Eye-0.3
Placebo--Left Eye-1.4
Valproic Acid--Right Eye-0.2
Valproic Acid--Left Eye-0.6

Static Perimetry Volume--30 Degree Hill of Vision

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. (NCT01233609)
Timeframe: baseline to week 52

Interventiondb-steridans (Mean)
Placebo--Right Eye-0.3
Placebo--Left Eye-0.3
Valproic Acid--Right Eye-0.2
Valproic Acid--Left Eye-0.2

Reviews

1 review available for valproic acid and Retinitis Pigmentosa

ArticleYear
Current Pharmacological Concepts in the Treatment of the Retinitis Pigmentosa.
    Advances in experimental medicine and biology, 2018, Volume: 1074

    Topics: Animals; Clinical Trials as Topic; Docosahexaenoic Acids; Drug Discovery; Drug Evaluation, Preclinic

2018

Trials

3 trials available for valproic acid and Retinitis Pigmentosa

ArticleYear
Effect of Oral Valproic Acid vs Placebo for Vision Loss in Patients With Autosomal Dominant Retinitis Pigmentosa: A Randomized Phase 2 Multicenter Placebo-Controlled Clinical Trial.
    JAMA ophthalmology, 2018, 08-01, Volume: 136, Issue:8

    Topics: Administration, Oral; Adult; Aged; Anticonvulsants; Double-Blind Method; Electroretinography; Female

2018
Efficacy of oral valproic acid in patients with retinitis pigmentosa.
    Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics, 2014, Volume: 30, Issue:7

    Topics: Administration, Oral; Adolescent; Adult; Cohort Studies; Female; Humans; Male; Middle Aged; Pilot Pr

2014
Quantification of Ellipsoid Zone Changes in Retinitis Pigmentosa Using en Face Spectral Domain-Optical Coherence Tomography.
    JAMA ophthalmology, 2016, Jun-01, Volume: 134, Issue:6

    Topics: Aged; Electroretinography; Female; GABA Agents; Humans; Male; Middle Aged; Observer Variation; Repro

2016

Other Studies

12 other studies available for valproic acid and Retinitis Pigmentosa

ArticleYear
Effect of Sodium Valproate on the Conformational Stability of the Visual G Protein-Coupled Receptor Rhodopsin.
    Molecules (Basel, Switzerland), 2021, May-19, Volume: 26, Issue:10

    Topics: Animals; Humans; Mutation; Protein Conformation; Receptors, G-Protein-Coupled; Retinitis Pigmentosa;

2021
Opposing Effects of Valproic Acid Treatment Mediated by Histone Deacetylase Inhibitor Activity in Four Transgenic
    The Journal of neuroscience : the official journal of the Society for Neuroscience, 2017, 01-25, Volume: 37, Issue:4

    Topics: Animals; Autophagosomes; Histone Deacetylase Inhibitors; Histones; Humans; Photoreceptor Cells; Reti

2017
Valproic Acid for a Treatment of Retinitis Pigmentosa: Reasons for Optimism and Caution.
    The Journal of neuroscience : the official journal of the Society for Neuroscience, 2017, 05-24, Volume: 37, Issue:21

    Topics: Animals; Animals, Genetically Modified; Histone Deacetylase Inhibitors; Retinitis Pigmentosa; Valpro

2017
The adverse effects of valproic acid on visual functions in the treatment of retinitis pigmentosa.
    Indian journal of ophthalmology, 2017, Volume: 65, Issue:10

    Topics: Adolescent; Adult; Dose-Response Relationship, Drug; Electroretinography; Enzyme Inhibitors; Female;

2017
Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa: Lessons Learned From a Trial of Valproic Acid.
    JAMA ophthalmology, 2018, 08-01, Volume: 136, Issue:8

    Topics: Blindness; Humans; Randomized Controlled Trials as Topic; Retinitis Pigmentosa; Valproic Acid; Visio

2018
Errors in Potential Conflicts of Interest Disclosures, Author Affiliation, and Role of Funder.
    JAMA ophthalmology, 2018, 12-01, Volume: 136, Issue:12

    Topics: Blindness; Conflict of Interest; Disclosure; Humans; Retinitis Pigmentosa; Valproic Acid

2018
Long-term follow-up for efficacy and safety of treatment of retinitis pigmentosa with valproic acid.
    The British journal of ophthalmology, 2013, Volume: 97, Issue:7

    Topics: Adolescent; Adult; Follow-Up Studies; GABA Agents; Humans; Liver Function Tests; Middle Aged; Retina

2013
Therapeutic potential of valproic acid for retinitis pigmentosa.
    The British journal of ophthalmology, 2011, Volume: 95, Issue:1

    Topics: Adolescent; Adult; Female; Humans; Male; Middle Aged; Pilot Projects; Retinitis Pigmentosa; Retrospe

2011
The conclusions of Clemson et al concerning valproic acid are premature.
    The British journal of ophthalmology, 2011, Volume: 95, Issue:1

    Topics: Humans; Retinitis Pigmentosa; Valproic Acid; Visual Acuity

2011
Lack of scientific rationale for use of valproic acid for retinitis pigmentosa.
    The British journal of ophthalmology, 2011, Volume: 95, Issue:5

    Topics: Humans; Retinitis Pigmentosa; Treatment Outcome; Valproic Acid; Visual Acuity

2011
Authors’ response.
    The British journal of ophthalmology, 2011, Volume: 95, Issue:8

    Topics: Humans; Retinitis Pigmentosa; Valproic Acid; Visual Acuity

2011
Valproic acid treatment may be harmful in non-dominant forms of retinitis pigmentosa.
    The British journal of ophthalmology, 2012, Volume: 96, Issue:8

    Topics: Female; Humans; Male; Retinitis Pigmentosa; Valproic Acid

2012