VPA ( DrugBank: - )

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1 disease

ID	Disease name (Link within this page)	Number of trials
3	Spinal muscular atrophy	5

3. Spinal muscular atrophy

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Clinical trials : 237 /  Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 75

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	JPRN-JMA-IIA00259	16/08/2016	10/08/2016	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial	spinal muscular atrophy	Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper..	Institute of Medical Genetics, Tokyo Women's Medical University	NULL	Recruiting	>=1 YEARS	<8 YEARS	BOTH	28	Phase 2B	Japan
2	JPRN-JMA-IIA00231	29/01/2016	01/12/2015	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial	spinal muscular atrophy	Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. Control intervention1:Placebo, Dose form:GRANULES, Route of administration:ORAL, Intended dose regimen:Placebo is to be taken once a day after supper..	Institute of Medical Genetics, Tokyo Women's Medical University	NULL	Completed	>=1 YEARS	<7 YEARS	BOTH	28	Phase 2B	Japan
3	JPRN-JMA-IIA00190	22/07/2014	01/08/2014	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy	spinal muscular atrophy	Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA is to be taken once a day after supper. Administration of VPA starts with an initial dosage (standard dose: 12.5 mg / kg) for 4 weeks. The dosage then increases to a maintenance dosage (standard dose: 25 mg/kg) from the 5th week to the 12th week. Administration, however, could be increased to an additional 50 mg/day in case of a blood concentration of VPA below 50 mcg/mL From the 13th week, administered doses are to be decreased (standard dose: 12.5 mg/kg) and continued to the 14th week. . Control intervention1:No.	Institute of Medical Genetics, Tokyo Women's Medical University	NULL	Completed	No Limit	<8 YEARS	BOTH	13	Phase 2A	Japan
4	NCT00481013 (ClinicalTrials.gov)	July 2007	30/5/2007	Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy	Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study	Spinal Muscular Atrophy	Drug: Valproic Acid (VPA);Drug: Placebo	University of Utah	Families of Spinal Muscular Atrophy;Abbott	Completed	18 Years	60 Years	Both	33	Phase 2	United States
5	NCT00374075 (ClinicalTrials.gov)	September 2003	6/9/2006	Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy	In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy	Spinal Muscular Atrophy	Drug: Valproic Acid	University of Utah	Families of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;Abbott	Completed	2 Years	N/A	Both	42	Phase 1	United States

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