3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details


臨床試験数 : 237 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

  
3 trials found
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1JPRN-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
2NCT00481013
(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
3NCT00374075
(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