Drugs / Valproic Acid
last change Sep 2026 re-read 8 days ago

Valproic Acid

Small molecule targets GSK3B, UGT2B7 via inhibition

Developed for
myelodysplastic syndrome · acute myeloid leukemia · HIV infectious disease · bipolar disorder · cancer · epilepsy · glioblastoma · anaplastic astrocytoma
+106 more · chronic lymphocytic leukemia/small lymphocytic lymphoma · head and neck squamous cell carcinoma · sarcoma · acute kidney injury · brain neoplasm · breast cancer · central nervous system neoplasm · childhood malignant neoplasm · colorectal cancer · gliomatosis cerebri · ischemic disease · leukemia · lymphoma · non-small cell lung carcinoma · soft tissue sarcoma · spinal muscular atrophy · tropical spastic paraparesis · acute graft versus host disease · acute myeloid leukemia with 11q23 abnormalities · acute myeloid leukemia with abnormal bone marrow eosinophils inv(16)(p13q22) or t(16;16)(p13;q22) · acute myeloid leukemia with t(8;21)(q22;q22) translocation · acute promyelocytic leukemia · Alzheimer disease · anal squamous cell carcinoma · astrocytoma, IDH-mutant, grade 4 · autoimmune lymphoproliferative syndrome · B-cell chronic lymphocytic leukemia · baldness, male pattern · borderline epithelial tumor of ovary · brain cancer · breast carcinoma · breath-holding Spells · Burkitt lymphoma · cancer of unknown primary site · carcinoma of pharynx · Castleman disease · cervical squamous cell carcinoma · childhood medulloblastoma · chronic myeloid leukemia · delirium · Dengue hemorrhagic fever · diffuse intrinsic pontine glioma · diffuse large B-cell lymphoma · diffuse midline glioma, H3 K27M-mutant · endometrioid tumor · Epstein-Barr virus-related tumor · Erdheim-Chester disease · female breast carcinoma · female reproductive system neoplasm · focal epilepsy · focal segmental glomerulosclerosis · gliosarcoma · grade III glioma · head and neck cancer · hypersplenism · idiopathic nephrotic syndrome · infantile spasms · iris melanoma · ischemic stroke · lip and oral cavity carcinoma · lung cancer · lymph node disorder · lymphangioleiomyomatosis · male reproductive system neoplasm · malignant glioma · malignant pancreatic neoplasm · mantle cell lymphoma · medium/large size posterior uveal melanoma · melanoma · mesothelial neoplasm · metastatic malignant neoplasm · metastatic malignant neoplasm in the brain · minimal change disease · mood disorder · nasopharyngeal carcinoma · neoplasm · neuroblastoma · neuropathy, painful · NF2-related schwannomatosis · oral cavity cancer · oropharynx squamous cell carcinoma · ovarian carcinoma · ovarian clear cell tumor · ovarian serous tumor · pancreatic adenocarcinoma · photosensitive epilepsy · progressive supranuclear palsy · prostate cancer · respiratory tract neoplasm · small cell lung carcinoma · small size posterior uveal melanoma · specific phobia · spinal muscular atrophy, type 1 · squamous cell carcinoma of penis · thyroid cancer · thyroid gland carcinoma · traumatic brain injury · urinary bladder cancer · urothelial carcinoma · bipolar I disorder · childhood absence epilepsy · epilepsy with generalized tonic-clonic seizures · glioma · intracerebral hemorrhage · migraine disorder · retinitis pigmentosa
Investigated by
National Cancer Institute (NCI) · M.D. Anderson Cancer Center · Abbott · H. Lee Moffitt Cancer Center and Research Institute · National Cancer Institute, Naples · United States Department of Defense
+125 more · University of Utah · Celgene Corporation · Families of Spinal Muscular Atrophy · Leadiant Biosciences, Inc. · University of Bergen · Assistance Publique - Hôpitaux de Paris · Clinipace Worldwide · Dr. Hasan Alam · Eisai Inc. · Hannover Medical School · National Institute of Allergy and Infectious Diseases (NIAID) · New Mexico Cancer Research Alliance · Pfizer · Soroka University Medical Center · University of California, San Francisco · University of North Carolina, Chapel Hill · University of Sao Paulo · Westat · AHS Cancer Control Alberta · AIDS Malignancy Consortium · Academic Medical Center - University of Amsterdam · All India Institute of Medical Sciences · Amorepacific Corporation · Assiut University · Auxilio Mutuo Cancer Center · Barbara Ann Karmanos Cancer Institute · Barretos Cancer Hospital · Basque Health Service · Baylor College of Medicine · BioNumerik Pharmaceuticals, Inc. · BioPharma Services Inc. · Biogen · CIHR Canadian HIV Trials Network · Canadian Foundation for AIDS Research (CANFAR) · Cancer Institute of the State of São Paulo · CancerCare Manitoba · Canisius-Wilhelmina Hospital · Cerebral Therapeutics LLC · Children's Hospital of Orange County · Children's Oncology Group · Chonbuk National University · Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Columbia University · Cook Children's Medical Center · Desitin Arzneimittel GmbH · Deutsche Kinderkrebsstiftung · Dr Cipto Mangunkusumo General Hospital · Duke University · EMD Serono · Erasmus Medical Center · Estetra · European Lung Cancer Working Party · GIMEMA Foundation · Genentech, Inc. · Gia Dinh People Hospital · Gregory Jicha, 323-5550 · Heinrich-Heine University, Duesseldorf · IRCCS National Neurological Institute "C. Mondino" Foundation · Inflammatory Breast Cancer Network Foundation · Inova Health Care Services · Jean-Pierre Routy · Johns Hopkins University · Joliet Center for Clinical Research · Karuna Therapeutics, Inc. · Kentucky Alzheimer's Center · Koneti Rao · Maasstad Hospital · Maastricht University Medical Center · Medical University of South Carolina · Medisch Spectrum Twente · Merck Sharp & Dohme LLC · Miller Children's & Women's Hospital Long Beach · Ministry of Health, Italy · Mohammed M Milhem · all 131 on the full record

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) DEPAKENE — 1978-02-28 fda.gov ↗

Trials 110 · started per year

today
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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 04 trials · 1 missed
Phase 0 NCT02608736 Dec 2015 → Dec 2016 head and neck squamous cell carcinoma Barretos Cancer Hospital Completed No outcome recorded
Phase 0 NCT02446431 Jul 2014 → Jul 2024 overdue cancer Miller Children's & Women's Hospital Long Beach Recruiting No outcome recorded
Phase 0 NCT01729598 Apr 2012 → Oct 2014 Alzheimer disease Gregory Jicha, 323-5550 Completed Missed primary
Phase 0 NCT01738815 Dec 2011 → May 2013 urinary bladder cancer State University of New York - Upstate Medical University Completed No outcome recorded
Phase 130 trials
Phase 1 NCT06729970 Dec 2024 → Jan 2026 — Karuna Therapeutics, Inc. Completed No outcome recorded
Phase 1 NCT06214130 Sep → Nov 2023 — Desitin Arzneimittel GmbH Completed No outcome recorded
Phase 1 NCT03512860 Apr → Oct 2018 — Estetra Completed No outcome recorded
Phase 1 NCT03385525 Sep → Oct 2017 — Biogen Completed No outcome recorded
Phase 1 NCT04482764 Jan 2017 → Jan 2018 breath-holding Spells Assiut University Completed No outcome recorded
Phase 1 NCT02872428 Nov 2016 → Sep 2017 Dengue hemorrhagic fever Dr. Hasan Alam Terminated No outcome recorded Stop: Enrollment
Phase 1 NCT02520115 Aug 2015 → Sep 2018 borderline epithelial tumor of ovary, endometrioid tumor, ovarian carcinoma, ovarian clear cell tumor +1 Barbara Ann Karmanos Cancer Institute Completed No outcome recorded
Phase 1 NCT01951560 Sep 2013 → May 2016 — Dr. Hasan Alam Completed No outcome recorded
Phase 1 NCT01861990 May → Dec 2013 anaplastic astrocytoma, brain cancer, childhood medulloblastoma, glioblastoma Medical University of South Carolina Withdrawn No outcome recorded
Phase 1 NCT01552434 Oct 2012 → May 2026 Castleman disease, Erdheim-Chester disease, NF2-related schwannomatosis, breast carcinoma +11 M.D. Anderson Cancer Center Terminated No outcome recorded
Phase 1/211 trials
Phase 1/2 NCT04529954 Jan 2021 → Mar 2024 focal epilepsy Cerebral Therapeutics LLC Terminated No outcome recorded
Phase 1/2 NCT03525730 Apr 2018 → Jul 2019 HIV infectious disease Erasmus Medical Center Completed No outcome recorded
Phase 1/2 NCT01369368 Aug 2013 → Oct 2017 acute myeloid leukemia University of Bergen Unknown No outcome recorded
Phase 1/2 NCT01203735 Feb 2011 → Feb 2013 non-small cell lung carcinoma Soroka University Medical Center Unknown No outcome recorded
Phase 1/2 NCT01295593 Dec 2010 → Apr 2013 B-cell chronic lymphocytic leukemia Cliniques universitaires Saint-Luc- Université Catholique de Louvain Unknown No outcome recorded
Phase 1/2 NCT00995332 Sep 2009 → Feb 2013 acute myeloid leukemia University of Bergen Completed No outcome recorded
Phase 1/2 NCT00661453 Apr 2008 → May 2012 spinal muscular atrophy, type 1 University of Utah Completed No outcome recorded
Phase 1/2 NCT00605657 Jan 2008 → Sep 2011 autoimmune lymphoproliferative syndrome, hypersplenism, lymph node disorder Koneti Rao Completed No outcome recorded
Phase 1/2 NCT00358319 Mar 2005 → Apr 2007 melanoma H. Lee Moffitt Cancer Center and Research Institute Terminated No outcome recorded
Phase 1/2 NCT00175812 Nov 2004 → May 2008 acute myeloid leukemia University of Bergen Completed No outcome recorded
Phase 242 trials · 6 missed
Phase 2 NCT07831369 Sep 2026 → Sep 2027 expected astrocytoma, IDH-mutant, grade 4, glioblastoma Inova Health Care Services Recruiting No outcome recorded
Phase 2 NCT06714357 Mar 2025 → Dec 2026 expected colorectal cancer National Cancer Institute, Naples Recruiting No outcome recorded
Phase 2 NCT06020898 Sep 2023 → Apr 2024 ischemic stroke RenJi Hospital Unknown No outcome recorded
Phase 2 NCT05821556 Jun 2023 → Aug 2026 overdue pancreatic adenocarcinoma National Cancer Institute, Naples Recruiting No outcome recorded
Phase 2 NCT04531579 Jan 2022 → Jun 2023 acute kidney injury, ischemic disease Westat Withdrawn No outcome recorded
Phase 2 NCT04531592 Jan 2022 → Jun 2023 acute kidney injury, ischemic disease Westat Withdrawn No outcome recorded
Phase 2 NCT04310176 May 2019 → Dec 2023 colorectal cancer National Cancer Institute, Naples Unknown No outcome recorded
Phase 2 NCT03357757 Feb 2018 → Mar 2022 Epstein-Barr virus-related tumor, anal squamous cell carcinoma, cervical squamous cell carcinoma, head and neck squamous cell carcinoma +1 AHS Cancer Control Alberta Completed No outcome recorded
Phase 2 NCT02789813 Jul 2016 → Jun 2018 specific phobia University of Basel Completed No outcome recorded
Phase 2 NCT02848105 Jul 2016 → Jul 2019 acute graft versus host disease Shanghai Jiao Tong University School of Medicine Unknown No outcome recorded
Phase 2/32 trials
Phase 2/3 NCT07166393 May 2026 → Feb 2030 expected traumatic brain injury Northwestern University Not yet recruiting No outcome recorded
Phase 2/3 NCT02896270 Oct 2016 → Oct 2019 focal segmental glomerulosclerosis, idiopathic nephrotic syndrome, minimal change disease University Hospital Brussels Unknown No outcome recorded
Phase 34 trials
Phase 3 NCT03243461 Jul 2018 → Dec 2023 anaplastic astrocytoma, diffuse midline glioma, H3 K27M-mutant, glioblastoma, gliomatosis cerebri University of Göttingen Unknown No outcome recorded
Phase 3 NCT02374567 Jan 2015 → Jun 2017 anxiety disorder, dementia, depressive disorder, schizophrenia +1 Hannover Medical School Terminated No outcome recorded
Phase 3 NCT00681980 Feb 2008 → Sep 2009 tropical spastic paraparesis University of Sao Paulo Completed No outcome recorded
Phase 3 NCT02456454 Jan 2006 → Jun 2010 bipolar disorder Nationwide Children's Hospital Completed No outcome recorded
Phase 410 trials · 2 met primary
Phase 4 NCT05697614 Mar → May 2023 epilepsy Dr Cipto Mangunkusumo General Hospital Unknown No outcome recorded
Phase 4 NCT03161509 Jul 2017 → Dec 2018 — Rambam Health Care Campus Terminated No outcome recorded
Phase 4 NCT04967326 Jan 2016 → Dec 2018 epilepsy Gia Dinh People Hospital Completed Met primary
Phase 4 NCT02343575 Jan 2015 → Jan 2018 delirium Stanford University Terminated No outcome recorded Stop: Enrollment
Phase 4 NCT02429596 May 2012 → May 2015 epilepsy IRCCS National Neurological Institute "C. Mondino" Foundation Unknown No outcome recorded
Phase 4 NCT00609245 Dec 2007 → Dec 2008 photosensitive epilepsy Vanderbilt University Medical Center Completed Met primary
Phase 4 NCT00854581 Nov 2007 → Jun 2011 lymphoma, precancerous condition University of Miami Terminated No outcome recorded
Phase 4 NCT00298857 Mar 2006 → Mar 2007 obesity disorder Northwell Health Terminated No outcome recorded Stop: Business
Phase 4 NCT00211250 Jul 2005 → Jul 2006 mood disorder Joliet Center for Clinical Research Completed No outcome recorded
Phase 4 NCT00431522 Dec 2004 → Oct 2006 bipolar disorder Sanofi Completed No outcome recorded
Phase not applicable7 trials · 1 missed
— NCT07052136 Nov 2024 → Apr 2025 — RESnTEC, Institute of Research Completed No outcome recorded
— NCT05236166 Apr 2017 → May 2021 epilepsy University Magna Graecia Completed No outcome recorded
— NCT04486092 Feb 2015 → Jul 2021 bipolar disorder National Cheng-Kung University Hospital Unknown No outcome recorded
— NCT02056236 Apr 2014 → Apr 2021 cardiac arrest University of Twente Completed Missed primary
— NCT00810680 Sep 2008 → Mar 2009 chronic lymphocytic leukemia/small lymphocytic lymphoma All India Institute of Medical Sciences Unknown No outcome recorded
— NCT00519181 Mar 2006 → Jun 2007 tropical spastic paraparesis University Hospital Pierre Zobda-Quitman Terminated No outcome recorded
— NCT00075777 Feb 2005 → Jul 2007 sarcoma AIDS Malignancy Consortium Completed No outcome recorded
Comparator or background therapy22 trials
Phase 1 NCT03885947 Background Feb 2019 → Mar 2021 Hodgkins lymphoma, acute lymphoblastic leukemia, myelodysplastic syndrome Alla Keyzner Completed No outcome recorded
Phase 1 NCT02761291 Background May 2016 → Jun 2017 nasopharyngeal carcinoma Chang Gung Memorial Hospital Unknown No outcome recorded
Phase 1 NCT00530907 Background Jun 2007 → Nov 2013 — M.D. Anderson Cancer Center Completed No outcome recorded
Phase 1/2 NCT06199557 Comparator May 2024 → Nov 2025 overdue acute myeloid leukemia, myelodysplastic syndrome Haukeland University Hospital Recruiting No outcome recorded
Phase 1/2 NCT04729543 Background Oct 2020 → Dec 2024 overdue uveal melanoma Erasmus Medical Center Recruiting No outcome recorded

Evidence & citations 24 cited values

Every value below carries the sentence it was read from. 146 sources stand behind the page.

FieldValueCited text
Known as VALPROIC acid “In recent years, a number of preclinical studies have found that valproic acid has a potential neuroprotective effect in acute ischemic brain injury, which can decrease infarct...” NCT06020898 ↗
132

“Valproic acid: Starting at 500mg daily, dose weekly adjusted according to blood serum level (50 and 125µg/ml), efficacy and tolerability.” NCT00976794 ↗

“Combination of Valproic Acid and Lenalidomide in the Treatment of Patients With Myelodysplastic Syndrome With Favorable Risk Profile” NCT00977132 ↗

“Drugs: carbamazepine, lamotrigine, levetiracetam, oxcarbazepine, phenobarbital, phenytoin, topiramate, valproic acid, zonisamide” NCT05236166 ↗

“Valproic acid with 1000mg loading dose with 500mg q12 4 hours later to maintain a trough level above 75ug/ml” NCT02848105 ↗

“5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®)” NCT00339196 ↗

“Option 2: valproic acid bolus 10-20 mg/kg in 30 min, followed by15 mg/kg/day in 2 dosages intravenously” NCT02056236 ↗

“carbamazepine, valproic acid, topiramate, oxcarbazepine, levetiracetam or lamotrigine” NCT02429596 ↗

“as well as the plasma levels of hydralazine and valproic acid.” NCT00395655 ↗

“brain tumors (lamotrigine, levetiracetam, valproic acid)” NCT02670161 ↗

“Valproic Acid (VPA) expanded cord blood stem cells” NCT03885947 ↗

Show 122 more sources

Known as 2-propylpentanoic acid “Valproic acid (VPA, 2-propylpentanoic acid) is an established drug in the long-term therapy of epilepsy.” NCT00439673 ↗
Known as Depakine “5-azacytidine (Vidaza®) + Valproic Acid (Depakine ®) Before Administration of Retinoic Acid (Vesanoid®)” NCT00339196 ↗
Known as Depakine-Chrono “Valproic Acid (Depakine-Chrono®) (VPA) will be administered concomitantly for a minimum of 6 cycles.” NCT01342692 ↗
Known as Depakote “The purpose of this research study is to test the safety, tolerability, and effectiveness of the combination of three drugs, sorafenib (Nexavar®), valproic acid (Depakote®),...” NCT01817751 ↗
1

“Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)” NCT00605657 ↗

Known as Depakote (Valproic Acid) “Depakote (Valproic Acid)” NCT00996060 ↗
Known as Depakote ER “Patients with plasma HIV RNA<50 c/mL added sustained-release VPA (Depakote ER) twice daily, RAL 400 mg twice daily, or ENF 90 mcg twice daily.” PMID 20186346 ↗ Feb 2010
1

“Valproic Acid (Depakote ER) in Patients With Advanced Thyroid Cancer” NCT00525135 ↗

Known as Epival “The purpose of this study is to examine whether the co-administration of valproic acid (Epival®), with highly active antiretroviral therapy (HAART) can reduce the size of HIV...” NCT00289952 ↗
Known as Ergenyl “E.g. Valproat-neuraxpharm®, Valproat-neuraxpharm® Lösung, Ergenyl®, Ergenyl®-Lösung oder Orfiril® Saft (Valproic acid);” NCT03243461 ↗
Known as Ergenyl-Lösung “E.g. Valproat-neuraxpharm®, Valproat-neuraxpharm® Lösung, Ergenyl®, Ergenyl®-Lösung oder Orfiril® Saft (Valproic acid);” NCT03243461 ↗
Known as Orfiril Saft “E.g. Valproat-neuraxpharm®, Valproat-neuraxpharm® Lösung, Ergenyl®, Ergenyl®-Lösung oder Orfiril® Saft (Valproic acid);” NCT03243461 ↗
Known as sodium valproate “The aim of the present study was therefore to determine the effects of sodium valproate (VPA), a GSK-3 inhibitor, on disease progression in progressive supranuclear palsy (PSP).” PMID 27136096 ↗ Apr 2016
Known as Sodium valproate or valproic acid (Depakote) “Sodium valproate or valproic acid (Depakote)” NCT00563992 ↗
Known as VAL “valproic acid (VAL, a UGT2B7 inhibiting drug)” NCT03512860 ↗
Known as Valproat-neuraxpharm “E.g. Valproat-neuraxpharm®, Valproat-neuraxpharm® Lösung, Ergenyl®, Ergenyl®-Lösung oder Orfiril® Saft (Valproic acid);” NCT03243461 ↗
Known as Valproat-neuraxpharm Lösung “E.g. Valproat-neuraxpharm®, Valproat-neuraxpharm® Lösung, Ergenyl®, Ergenyl®-Lösung oder Orfiril® Saft (Valproic acid);” NCT03243461 ↗
Known as valproate “We used valproate (VPA), a histone deacetylase inhibitor that has been used for decades as a chronic, safe treatment for epileptic disorders.” PMID 17717136 ↗ Aug 2007
Known as Valproic acid (VP) “Valproic acid (VP)” NCT04729543 ↗
Known as Valproic acid (VPA) (depakote) “Valproic acid (VPA) (depakote)” NCT00614458 ↗
Known as VPA “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...” NCT01233609 ↗
38

“Owing to its HDACi activity and its safe use for epileptic disorders, valproic acid (VPA) is a good candidate for anticancer therapy that we have largely explored preclinically...” PMID 32849914 ↗ Aug 2020

“Valproic acid (VPA), a branched short-chain fatty acid, is widely used in clinical as an antiepileptic drug and a mood stabilizer, primarily in the treatment of epilepsy,...” NCT02761291 ↗

“PURPOSE: To study the efficacy and tolerability of valproic acid (VPA) and radiation, followed by VPA and bevacizumab in children with newly diagnosed diffuse intrinsic pontine...” PMID 32285998 ↗ Apr 2020

“We conducted a phase 1/2 study of the combination of 5-aza-2'-deoxycytidine (decitabine) and the histone deacetylase inhibitor valproic acid (VPA) in patients with advanced...” PMID 16882711 ↗ Aug 2006

“histone deacetylase (HDAC) inhibitor valproic acid (VPA; Barker et al. 2013; Wolff et al. 2008, 2011; Felix et al. 2011; Su et al. 2011; Rokes et al. 2010; Masoudi et al. 2008;...” NCT03243461 ↗

“Due to its histone deacetylase (HDAC) inhibiting activity and its safe use as a chronic therapy for epileptic disorders, valproic acid (VPA) has been considered a good...” PMID 27884140 ↗ Nov 2016

“Conflicting results have been reported regarding the ability of valproic acid (VPA) to reduce the size of HIV reservoirs in patients receiving suppressive highly active...” PMID 22276680 ↗ Jan 2012

“The trial investigates the use of VPA (Valproic Acid) for the treatment of adult patients with biopsy proven idiopathic focal segmentel glomerulosclerosis (FSGS) or minimal...” NCT02896270 ↗

“The aim of the present study was therefore to determine the effects of sodium valproate (VPA), a GSK-3 inhibitor, on disease progression in progressive supranuclear palsy (PSP).” PMID 27136096 ↗ Apr 2016

“Valproic acid (VPA) is a generic low-cost anticonvulsant and mood stabilizer with HDAC inhibitory activity, and anticancer properties also demonstrated in PDAC models.” PMID 39300376 ↗ Sep 2024

Show 28 more sources

Action Inhibit “the histone deacetylase inhibitor valproic acid (VPA)” PMID 16882711 ↗ Aug 2006
Modality Small molecule “Valproic acid is a short chained fatty acid” NCT00339196 ↗
Route Oral “valproic acid (lowest dose in the first 5 patients and a higher dose in patients 6 and 7) orally every 8 hours” NCT00000629 ↗
Target GSK3B “valproic acid was able to inhibit the activity of GSK3b” NCT00385710 ↗