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Sodium valproate    (DrugBank: Valproate)

4 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1
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
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.
1 / 508 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT00136110
(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

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

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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