3. 脊髄性筋萎縮症
［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］
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.
| 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 | 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 | 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 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 Utah | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 1;Phase 2 | Germany | ||
| 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 |
| 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) | 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 | 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) | 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 | 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 |