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
Showing 1 to 9 of 9 diseases
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 |
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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 acidin childhood on ... | 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 acidin childhood on ... | 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.. Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, inten ... | 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 acidin childhood on ... | 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 acidin childhood on ... | 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.. Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, inten ... | 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 acidin childhood on ... | 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 acidin childhood on ... | 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. Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, inten ... | Institute of Medical Genetics, Tokyo Women's Medical University | NULL | Completed | No Limit | <8 YEARS | BOTH | 13 | Phase 2A | Japan |
4 | NCT01671384 (ClinicalTrials.gov) | August 2013 | 13/8/2012 | Valproate and Levocarnitine in Children With Spinal Muscular Atrophy | Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years Randomized Placebo Controlled Trial of Valproateand Levocarnitine in Children With Spinal Muscular A ... | Spinal Muscular Atrophy | Drug: Valproate, Levocarnitine;Drug: Placebo | All India Institute of Medical Sciences, New Delhi | NULL | Recruiting | 2 Years | 15 Years | Both | 60 | Phase 3 | India |
5 | EUCTR2008-003915-11-DE (EUCTR) | 24/09/2009 | 25/06/2009 | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I Phase I/II Trial of Valproic Acidand Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI ... | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I Phase I/II Trial of Valproic Acidand Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI ... | Spinal Muscular Atrophy Type I in infants MedDRA version: 9.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital Spinal Muscular Atrophy Type I in infants MedDRA version: 9.1;Level: LLT;Classification code 1005120 ... | Trade Name: Orfiril Saft INN or Proposed INN: VALPROATE SODIUM Trade Name: Biocarn INN or Proposed INN: LEVOCARNITINE Trade Name: Orfiril Saft INN or Proposed INN: VALPROATESODIUM Trade Name: Biocarn INN or Proposed INN: ... | University of Utah | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 1/2 | Germany | ||
6 | NCT00661453 (ClinicalTrials.gov) | April 2008 | 14/4/2008 | CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I | Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I) Phase I/II Trial of Valproic Acidand Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI ... | Spinal Muscular Atrophy Type I | Drug: Valproic Acid and Levocarnitine | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc. | Completed | N/A | 12 Months | All | 40 | Phase 1/Phase 2 | United States;Canada;Germany |
7 | 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 Prospective Controlled Trial of Valproic Acidin Ambulant Adults With Spinal Muscular Atrophy (VALIAN ... | 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 |
8 | NCT00227266 (ClinicalTrials.gov) | September 2005 | 23/9/2005 | Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy | Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial) Multi-center Phase II Trial of Valproic Acidand Carnitine in Patients With Spinal Muscular Atrophy ( ... | Spinal Muscular Atrophy | Drug: Valproic Acid and Levocarnitine;Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;Abbott | Completed | 2 Years | 17 Years | All | 94 | Phase 2 | United States;Canada |
9 | 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 Study of Safety and Dosing Effect on SMN Levels of Valproic Acid(VPA) in Patients With Spinal Muscul ... | In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic A ... | 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 |