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Valproate    (DrugBank: Valproate)

6 diseases
告示番号疾患名（ページ内リンク）臨床試験数
2筋萎縮性側索硬化症2
3脊髄性筋萎縮症2
90網膜色素変性症1
102ルビンシュタイン・テイビ症候群1
233ウォルフラム症候群2
256筋型糖原病3

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］
Searched query = "Amyotrophic lateral sclerosis", "ALS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 508 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03204500
(ClinicalTrials.gov)		May 201628/6/2017Dual Treatment With Lithium and Valproate in ALS.A Randomized Clinical Trial, Double Blind, Placebo-controlled of Lithium and Valproate in Amyotrophic Lateral Sclerosis.Amyotrophic Lateral Sclerosis;Amyotrophic Lateral Sclerosis, SporadicCombination Product: Active treatment with dual therapy;Drug: PlacebosEl Instituto Nacional de Neurologia y Neurocirugia Manuel Velasco SuarezNULLCompleted40 Years70 YearsAll43Phase 2Mexico
2NCT00136110
(ClinicalTrials.gov)		April 200524/8/2005Trial of Sodium Valproate in Amyotrophic Lateral SclerosisA Randomized, Double-Blind, Placebo-Controlled Sequential Clinical Trial of Sodium Valproate in ALSAmyotrophic Lateral SclerosisDrug: Sodium ValproateUMC UtrechtPrincess Beatrix Fund, The NetherlandsCompleted18 Years85 YearsBoth165Phase 3Netherlands

3. 脊髄性筋萎縮症　［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］
Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 179 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01671384
(ClinicalTrials.gov)		August 201313/8/2012Valproate and Levocarnitine in Children With Spinal Muscular AtrophyRandomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 YearsSpinal Muscular AtrophyDrug: Valproate, Levocarnitine;Drug: PlaceboAll India Institute of Medical Sciences, New DelhiNULLRecruiting2 Years15 YearsBoth60Phase 3India
2EUCTR2008-003915-11-DE
(EUCTR)		24/09/200925/06/2009Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type IPhase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I Spinal Muscular Atrophy Type I in infants
MedDRA version: 9.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital		Trade Name: Orfiril Saft
INN or Proposed INN: VALPROATE SODIUM
Trade Name: Biocarn
INN or Proposed INN: LEVOCARNITINE		University of UtahNULLNot RecruitingFemale: yes
Male: yes		36Phase 1;Phase 2Germany

90. 網膜色素変性症　［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］
Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 103 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1JPRN-JMA-IIA00053
24/12/201022/11/2010Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis PigmentosaClinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa Retinitis PigmentosaIntervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL.Yasuhiko HiramiMasayo Takahashi, Yasuo KurimotoCompleted>=20 YEARSNo LimitBOTH30NOT APPLICABLEJapan

102. ルビンシュタイン・テイビ症候群　［臨床試験数：3，薬物数：8（DrugBank：1），標的遺伝子数：7，標的パスウェイ数：17］
Searched query = "Rubinstein-Taybi syndrome", "RSTS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 3 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01619644
(ClinicalTrials.gov)		April 201212/6/2012Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic TrialRubinstein-Taybi Syndrome: Functional Imaging and Therapeutic TrialRubinstein-Taybi SyndromeDrug: sodium valproate;Drug: PlaceboUniversity Hospital, BordeauxFondation Syndrome de Rubinstein-TaybiCompleted6 Years21 YearsBoth41Phase 2France

233. ウォルフラム症候群　［臨床試験数：6，薬物数：12（DrugBank：7），標的遺伝子数：11，標的パスウェイ数：40］
Searched query = "Wolfram syndrome", "Diabetes Insipidus, Diabetes mellitus, optic atrophy, and deafness syndrome", "DIDMOAD syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 6 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2017-001215-37-PL
(EUCTR)		13/02/202010/10/2019Clinical Trial of a Treatment to Slow Disease Progression Compared to Usual Standard of Care in Children and Adults with WolframA pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome - Treat Wolfram Wolfram syndrome;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Trade Name: Sodium valproate 200 mg gastro-resistant tabletsUniversity of BirminghamNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		70Phase 2Poland;United Kingdom
2NCT03717909
(ClinicalTrials.gov)		December 28, 201811/10/2018Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram SyndromeA Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram SyndromeWolfram SyndromeDrug: Sodium Valproate 200Mg E/C Tablet;Drug: Sodium Valproate matched placeboUniversity of BirminghamNULLRecruiting5 YearsN/AAll70Phase 2France;Poland;Spain;United Kingdom

256. 筋型糖原病　［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 161 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03112889
(ClinicalTrials.gov)		January 201511/8/2015Sodium Valproate for GSDVA Phase II Pilot Study to Explore Treatment With Sodium Valproate in Adults With McArdle Disease (Glycogen Storage Disorder Type V, GSDV)Glycogen Storage Disease Type V;McArdle DiseaseDrug: Sodium ValproateUniversity College, LondonNULLCompleted18 Years64 YearsAll8Phase 2Denmark;United Kingdom
2EUCTR2014-001637-88-DK
(EUCTR)		12/08/201424/06/2014Effects on muscle function, in persons with McArdle disease, when treated with the drug Valproate.A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Valproate treatment in McArdle disease McArdle disease (Glycogen storage disorder type V)
MedDRA version: 17.0;Level: LLT;Classification code 10026969;Term: McArdle's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		Trade Name: Deprakine
INN or Proposed INN: SODIUM VALPROATE		John VissingNULLNot RecruitingFemale: yes
Male: yes		15Phase 2Denmark
3EUCTR2012-002933-12-GB
(EUCTR)		07/04/201404/04/2014A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle DiseaseA Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Sodium Valproate for GSDV Version 1.0 13th January 2014 McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdomyolysis which can cause acute renal failure and from middle age muscle wasting and weakness. Affected patients are unable to produce lactate during ischaemic exercise due to a congenital lack of the enzyme muscle glycogen phosphorylase which is essential for glycogen metabolism.
MedDRA version: 16.1;Level: LLT;Classification code 10026969;Term: McArdle's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		University College London,NULLNot Recruiting Female: yes
Male: yes		8Phase 2United States;Denmark;United Kingdom

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