3. Spinal muscular atrophy Clinical trials / Disease details

Clinical trials : 217 /  Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80

  
9 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
4NCT01671384
(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
5EUCTR2008-003915-11-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 1/2Germany
6NCT00661453
(ClinicalTrials.gov)		April 200814/4/2008CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type IPhase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)Spinal Muscular Atrophy Type IDrug: Valproic Acid and LevocarnitineUniversity of UtahFamilies of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.CompletedN/A12 MonthsAll40Phase 1/Phase 2United States;Canada;Germany
7NCT00481013
(ClinicalTrials.gov)		July 200730/5/2007Valproic Acid in Ambulant Adults With Spinal Muscular AtrophyProspective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) StudySpinal Muscular AtrophyDrug: Valproic Acid (VPA);Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;AbbottCompleted18 Years60 YearsBoth33Phase 2United States
8NCT00227266
(ClinicalTrials.gov)		September 200523/9/2005Valproic Acid and Carnitine in Patients With Spinal Muscular AtrophyMulti-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)Spinal Muscular AtrophyDrug: Valproic Acid and Levocarnitine;Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;AbbottCompleted2 Years17 YearsAll94Phase 2United States;Canada
9NCT00374075
(ClinicalTrials.gov)		September 20036/9/2006Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular AtrophyIn Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Valproic AcidUniversity of UtahFamilies of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;AbbottCompleted2 YearsN/ABoth42Phase 1United States

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