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1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy3

3. Spinal muscular atrophy    [ 179 clinical trials,   102 drugs,   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways]
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.
3 / 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
1JPRN-JMA-IIA00259
16/08/201610/08/2016Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trialMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial spinal muscular atrophyIntervention 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 UniversityNULLRecruiting>=1 YEARS<8 YEARSBOTH28Phase 2BJapan
2JPRN-JMA-IIA00231
29/01/201601/12/2015Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory TrialMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial spinal muscular atrophyIntervention 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 UniversityNULLCompleted>=1 YEARS<7 YEARSBOTH28Phase 2BJapan
3JPRN-JMA-IIA00190
22/07/201401/08/2014Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophyMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy spinal muscular atrophyIntervention 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 UniversityNULLCompletedNo Limit<8 YEARSBOTH13Phase 2AJapan