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|+表6. 脊髄小脳変性症の治験 | |+表6. 脊髄小脳変性症の治験 | ||
! | !化合物!!ClinicalTrials.gov Identifier !! 年 !! 国 !! 対象疾患 !! Phase !! 結果 | ||
|- | |- | ||
| Taltirelin Hydrate || NCT04107740 || 2019- || 韓国 || SCD全般 || 4 || 現在進行中 | | Taltirelin Hydrate || [https://www.clinicaltrials.gov/ct2/show/NCT04107740 NCT04107740] || 2019- || 韓国 || SCD全般 || 4 || 現在進行中 | ||
|- | |- | ||
| KPS-0373(Rovatirelin) || NCT01970098 || 2013-2015 || 日本 || 軽度~中等度のSCD || 3 || 部分的に有効 | | KPS-0373(Rovatirelin) || [https://www.clinicaltrials.gov/ct2/show/NCT01970098 NCT01970098] || 2013-2015 || 日本 || 軽度~中等度のSCD || 3 || [https://jnnp.bmj.com/content/91/3/254 部分的に有効] | ||
|- | |- | ||
| Riluzole || NCT03347344 || 2018-2019 || フランス || SCA2 || 3 || 不明 | | Riluzole || [https://www.clinicaltrials.gov/ct2/show/NCT03347344?cond=Spinocerebellar+Degeneration&draw=3&rank=11 NCT03347344] || 2018-2019 || フランス || SCA2 || 3 || 不明 | ||
|- | |- | ||
| Transcranial Magnetic Stimulation || NCT01975909 || 2013-2016 || 米国 || genetically-confirmed SCA || Not Applicable || 有効性なし | | Transcranial Magnetic Stimulation || [https://www.clinicaltrials.gov/ct2/show/NCT03347344 NCT01975909] || 2013-2016 || 米国 || genetically-confirmed SCA || Not Applicable || 有効性なし | ||
|- | |- | ||
| Lithium Carbonate || NCT00683943 || 2008-2010 || 米国 || SCA1 || 1 || | | Lithium Carbonate || [https://www.clinicaltrials.gov/ct2/show/NCT00683943 NCT00683943] || 2008-2010 || 米国 || SCA1 || 1 || | ||
|- | |- | ||
| Intravenous Immune Globulin || NCT02287064 || 2015-2016 || 米国 || SCA types 1, 2, 3, 6, 10, or 11 || 1 || まだ発表なし | | Intravenous Immune Globulin || [https://www.clinicaltrials.gov/ct2/show/NCT02287064 NCT02287064] || 2015-2016 || 米国 || SCA types 1, 2, 3, 6, 10, or 11 || 1 || まだ発表なし | ||
|- | |- | ||
| Oral Trehalose || NCT04399265 || 2020ー- || マレーシア || Genetically confirmed SCA 3 || Not Applicable || 進行中 | | Oral Trehalose || [https://www.clinicaltrials.gov/ct2/show/NCT04399265 NCT04399265] || 2020ー- || マレーシア || Genetically confirmed SCA 3 || Not Applicable || 進行中 | ||
|- | |- | ||
| Varenicline (Chantix®) || NCT00992771 || 2009-2012 || 米国 || SCA3 || 2 || 有効 | | Varenicline (Chantix®) || [https://www.clinicaltrials.gov/ct2/show/NCT00992771 NCT00992771] || 2009-2012 || 米国 || SCA3 || 2 || [https://n.neurology.org/content/78/8/545.long 有効] | ||
|- | |- | ||
| Umbilical Cord Mesenchymal Stem Cells || NCT03378414 || 2017- || 中国 || SCA1,2,4,6 || 2 || 詳細不明 | | Umbilical Cord Mesenchymal Stem Cells || [https://www.clinicaltrials.gov/ct2/show/NCT03378414 NCT03378414] || 2017- || 中国 || SCA1,2,4,6 || 2 || 詳細不明 | ||
|- | |- | ||
| Troriluzole || NCT03701399 || 2018- || 米国 || SCA1,2,3,6,7,8,10 || 2,3 || 進行中 | | Troriluzole || [https://www.clinicaltrials.gov/ct2/show/NCT02960893 NCT03701399] || 2018- || 米国 || SCA1,2,3,6,7,8,10 || 2,3 || 進行中 | ||
|- | |- | ||
| Sodium Phenylbutyrate || NCT01096095 || 2010-2012 || ブラジル || SCA3 || 2 || 中止 | | Sodium Phenylbutyrate || [https://www.clinicaltrials.gov/ct2/show/NCT01096095 NCT01096095] || 2010-2012 || ブラジル || SCA3 || 2 || 中止 | ||
|- | |- | ||
| Dalfampridine || NCT01811706 || 2013-2015 || 米国 || SCA1,2,3,6 || Not Applicable || 有効性なし | | Dalfampridine || [https://www.clinicaltrials.gov/ct2/show/NCT01811706 NCT01811706] || 2013-2015 || 米国 || SCA1,2,3,6 || Not Applicable || 有効性なし | ||
|- | |- | ||
| CAD-1883 || NCT04301284 || 2020- || 米国 || SCA1,2,3,6,7,10,17 || 2 || 延期(COVID-19のため | | CAD-1883 || [https://www.clinicaltrials.gov/ct2/show/NCT04301284 NCT04301284] || 2020- || 米国 || SCA1,2,3,6,7,10,17 || 2 || 延期(COVID-19のため | ||
|- | |- | ||
| Docosahexaenoic Acid (DHA) || NCT03109626 || 2015-2018 || イタリア || SCA38 || Not Applicable || 有効 | | Docosahexaenoic Acid (DHA) || [https://www.clinicaltrials.gov/ct2/show/NCT03109626 NCT03109626] || 2015-2018 || イタリア || SCA38 || Not Applicable || [https://onlinelibrary.wiley.com/doi/full/10.1002/ana.25059 有効] | ||
|- | |- | ||
| Stemchymal® || NCT02540655 || 2015- || 台湾 || SCA2,3 || 2 || 不明 | | Stemchymal® || [https://www.clinicaltrials.gov/ct2/show/NCT02540655 NCT02540655] || 2015- || 台湾 || SCA2,3 || 2 || 不明 | ||
|- | |- | ||
| Intravenous Cabaletta || NCT02147886 || 2014-2016 || イスラエル || SCA3 || 2 || 結果? | | Intravenous Cabaletta || [https://www.clinicaltrials.gov/ct2/show/NCT02147886 NCT02147886] || 2014-2016 || イスラエル || SCA3 || 2 || 結果? | ||
|- | |- | ||
| Coenzyme Q10 || NCT00957216 || 2009-2018 || 米国 || 弧発性SCD || 1 || 結果? | | Coenzyme Q10 || [https://www.clinicaltrials.gov/ct2/show/NCT00957216 NCT00957216] || 2009-2018 || 米国 || 弧発性SCD || 1 || 結果? | ||
|- | |- | ||
| BHV-4157 || NCT03408080 || 2018- || 米国 || SCA1,2,3,6 || 3 || エントリー中止中 | | BHV-4157 || [https://clinicaltrials.gov/ct2/show/NCT03408080 NCT03408080] || 2018- || 米国 || SCA1,2,3,6 || 3 || エントリー中止中 | ||
|- | |- | ||
| Vatiquinone || NCT04577352 || 2020- || 米国 || Friedreich Ataxia || 2,3 || 開始前 | | Vatiquinone || [https://www.clinicaltrials.gov/ct2/show/NCT04577352 NCT04577352] || 2020- || 米国 || Friedreich Ataxia || 2,3 || 開始前 | ||
|- | |- | ||
| CTI-1601 || NCT04519567 || 2020- || 米国 || Friedreich Ataxia || 1 || 開始前 | | CTI-1601 || [https://www.clinicaltrials.gov/ct2/show/NCT04519567 NCT04519567] || 2020- || 米国 || Friedreich Ataxia || 1 || 開始前 | ||
|- | |- | ||
| Etravirine || NCT04273165 || 2020- || イタリア || Friedreich Ataxia || 2 || 開始前 | | Etravirine || [https://clinicaltrials.gov/ct2/show/NCT04273165 NCT04273165] || 2020- || イタリア || Friedreich Ataxia || 2 || 開始前 | ||
|- | |- | ||
| RT001 || NCT04102501 || 2019- || 米国 || Friedreich Ataxia || 3 || 進行中 | | RT001 || [https://www.clinicaltrials.gov/ct2/show/NCT04102501 NCT04102501] || 2019- || 米国 || Friedreich Ataxia || 3 || 進行中 | ||
|- | |- | ||
| Resveratrol || NCT03933163 || 2019- || 米国 || Friedreich Ataxia || 2 || 進行中 | | Resveratrol || [https://clinicaltrials.gov/ct2/show/NCT03933163 NCT03933163] || 2019- || 米国 || Friedreich Ataxia || 2 || 進行中 | ||
|- | |- | ||
| MIN-102 || NCT03917225 || 2019- || EU || Friedreich Ataxia || 2 || リクルート終了 | | MIN-102 || [https://clinicaltrials.gov/ct2/show/NCT03917225 NCT03917225] || 2019- || EU || Friedreich Ataxia || 2 || リクルート終了 | ||
|- | |- | ||
| gamma interferon || NCT03888664<br>NCT02797080<br>NCT02593773<br> NCT01965327 || || || Friedreich Ataxia || 2、3 || | | gamma interferon || [https://clinicaltrials.gov/ct2/show/NCT03888664 NCT03888664]<br>[https://clinicaltrials.gov/ct2/show/NCT02797080 NCT02797080]<br>[https://clinicaltrials.gov/ct2/show/NCT02593773 NCT02593773]<br>[https://www.clinicaltrials.gov/ct2/show/NCT01965327 NCT01965327]|| || || Friedreich Ataxia || 2、3 || | ||
|- | |- | ||
| Nicotinamide || NCT03761511 || 2018- || EU || Friedreich Ataxia || 2 || 進行中 | | Nicotinamide || [https://clinicaltrials.gov/ct2/show/NCT03761511 NCT03761511] || 2018- || EU || Friedreich Ataxia || 2 || 進行中 | ||
|- | |- | ||
| TAK-831 || NCT03214588 || 2017-2019 || 米国 || Friedreich Ataxia || 2 || 有効性なし | | TAK-831 || [https://www.clinicaltrials.gov/ct2/show/NCT03214588 NCT03214588] || 2017-2019 || 米国 || Friedreich Ataxia || 2 || 有効性なし | ||
|- | |- | ||
| Rosuvastatin || NCT02705547 || 2016-2018 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | | Rosuvastatin || [https://www.clinicaltrials.gov/ct2/show/NCT02705547 NCT02705547] || 2016-2018 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | ||
|- | |- | ||
| (+)-Epicatechin || NCT02660112 || 2016-2019 || 米国 || Friedreich Ataxia || 2 || 有効性なし | | (+)-Epicatechin || [https://clinicaltrials.gov/ct2/show/NCT02660112 NCT02660112] || 2016-2019 || 米国 || Friedreich Ataxia || 2 || 有効性なし | ||
|- | |- | ||
| Methylprednisolone || NCT02424435 || 2015-2020 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | | Methylprednisolone || [https://www.clinicaltrials.gov/ct2/show/NCT02424435 NCT02424435] || 2015-2020 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | ||
|- | |- | ||
| EPI-743(alpha-tocotrienol quinone) || NCT01962363 || 2013-2016 || 米国 || Friedreich's Ataxia with Point Mutations || 2 || 効果あり | | EPI-743(alpha-tocotrienol quinone) || [https://clinicaltrials.gov/ct2/show/NCT01962363 NCT01962363] || 2013-2016 || 米国 || Friedreich's Ataxia with Point Mutations || 2 || [https://n.neurology.org/content/86/16_Supplement/P5.388 効果あり] | ||
|- | |- | ||
| Omaveloxolone || NCT02255435 || 2014- || 米国 || Friedreich Ataxia || 2 || リクルート停止 | | Omaveloxolone || [https://clinicaltrials.gov/ct2/show/NCT02255435 NCT02255435] || 2014- || 米国 || Friedreich Ataxia || 2 || リクルート停止 | ||
|- | |- | ||
| Acetyl-L-Carnitine || NCT01921868 || 2013-2017 || 米国 || Friedreich Ataxia || Not Applicable || 結果未報告 | | Acetyl-L-Carnitine || [https://www.clinicaltrials.gov/ct2/show/NCT01921868 NCT01921868] || 2013-2017 || 米国 || Friedreich Ataxia || Not Applicable || 結果未報告 | ||
|- | |- | ||
| VP 20629 || NCT01898884 || 2013-2016 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | | VP 20629 || [https://clinicaltrials.gov/ct2/show/NCT01898884 NCT01898884] || 2013-2016 || 米国 || Friedreich Ataxia || 1 || リクルート終了 | ||
|- | |- | ||
| EPI-743 || NCT01728064 || 2012-2015 || 米国 || Friedreich Ataxia || 2 || 有効性なし | | EPI-743 || [https://clinicaltrials.gov/ct2/show/NCT01728064 NCT01728064] || 2012-2015 || 米国 || Friedreich Ataxia || 2 || 有効性なし | ||
|- | |- | ||
| Bupropion and Citalopram || NCT01716221 || 2012-2016 || 米国 || Friedreich Ataxia || 4 || 終了 | | Bupropion and Citalopram || [https://clinicaltrials.gov/ct2/show/NCT01716221 NCT01716221] || 2012-2016 || 米国 || Friedreich Ataxia || 4 || 終了 | ||
|- | |- | ||
| Resveratrol || NCT01339884 || 2011-2014 || オーストラリア || Friedreich Ataxia || 1,2 || 終了 | | Resveratrol || [https://clinicaltrials.gov/ct2/show/NCT01339884 NCT01339884] || 2011-2014 || オーストラリア || Friedreich Ataxia || 1,2 || 終了 | ||
|- | |- | ||
| Idebenone || NCT01303406 || 2011-2016 || EU || Friedreich Ataxia || 3 || 有効性なし | | Idebenone || [https://clinicaltrials.gov/ct2/show/NCT01303406 NCT01303406] || 2011-2016 || EU || Friedreich Ataxia || 3 || 有効性なし | ||
|- | |- | ||
| A0001(alpha-tocopherolquinone) || NCT01035671 || 2009-2011 || 米国 || Friedreich Ataxia || 2 || 容量依存性にFRDA評価スケール改善 | | A0001(alpha-tocopherolquinone) || [https://clinicaltrials.gov/ct2/show/NCT01035671 NCT01035671] || 2009-2011 || 米国 || Friedreich Ataxia || 2 || [https://onlinelibrary.wiley.com/doi/full/10.1002/mds.25058 容量依存性にFRDA評価スケール改善] | ||
|- | |- | ||
| Erythropoietin || NCT01016366 || 2009-2016 || EU || Friedreich Ataxia || 2 || 有効性なし | | Erythropoietin || [https://clinicaltrials.gov/ct2/show/NCT01016366 NCT01016366] || 2009-2016 || EU || Friedreich Ataxia || 2 || 有効性なし | ||
|- | |- | ||
| idebenone || NCT00993967 || 2009-2016 || EU || Friedreich Ataxia || 3 || 認容性を副作用の確認 | | idebenone || [https://clinicaltrials.gov/ct2/show/NCT00993967 NCT00993967] || 2009-2016 || EU || Friedreich Ataxia || 3 || 認容性を副作用の確認 | ||
|- | |- | ||
| deferiprone || NCT00530127 || 2007-2009 || EU || Friedreich Ataxia || 1,2 || 非重症例で進行抑制の可能性(+) | | deferiprone || [https://clinicaltrials.gov/ct2/show/NCT00530127 NCT00530127] || 2007-2009 || EU || Friedreich Ataxia || 1,2 || [https://onlinelibrary.wiley.com/doi/full/10.1002/ana.24248 非重症例で進行抑制の可能性(+)] | ||
|- | |- | ||
| EGb 761 || NCT00824512 || 209-2013 || フランス || Friedreich Ataxia || 2 || 有効性なし | | EGb 761 || [https://clinicaltrials.gov/ct2/show/NCT00824512 NCT00824512] || 209-2013 || フランス || Friedreich Ataxia || 2 || 有効性なし | ||
|- | |- | ||
| pioglitazone || NCT00811681 || 2008-2013 || フランス || Friedreich Ataxia || 3 || 終了 | | pioglitazone || [https://clinicaltrials.gov/ct2/show/NCT00811681 NCT00811681] || 2008-2013 || フランス || Friedreich Ataxia || 3 || 終了 | ||
|- | |- | ||
| varenicline || NCT00803868 || 2008-2010 || 米国 || Friedreich Ataxia || 2,3 || 途中で中止 | | varenicline || [https://www.clinicaltrials.gov/ct2/show/NCT00803868 NCT00803868] || 2008-2010 || 米国 || Friedreich Ataxia || 2,3 || 途中で中止 | ||
|- | |- | ||
| Triheptanoin || NCT04513002 || 2020- || オーストラリア || Ataxia Telangiectasia || 2 || リクルート開始前 | | Triheptanoin || [https://clinicaltrials.gov/ct2/show/NCT04513002 NCT04513002] || 2020- || オーストラリア || Ataxia Telangiectasia || 2 || リクルート開始前 | ||
|- | |- | ||
| vitamin B3 || NCT03962114 || 2019- || オランダ || Ataxia Telangiectasia || 2 || リクルート中 | | vitamin B3 || [https://clinicaltrials.gov/ct2/show/NCT03962114 NCT03962114] || 2019- || オランダ || Ataxia Telangiectasia || 2 || リクルート中 | ||
|- | |- | ||
| N-Acetyl-L-Leucine || NCT03759678 || 2018- || 米,英,独 || Ataxia Telangiectasia || 2 || リクルート中 | | N-Acetyl-L-Leucine || [https://clinicaltrials.gov/ct2/show/NCT03759678 NCT03759678] || 2018- || 米,英,独 || Ataxia Telangiectasia || 2 || リクルート中 | ||
|- | |- | ||
| Metformin,Pioglitazone || NCT02733679 || 2016-2019 || 英国 || Ataxia Telangiectasia || 4 || 終了 | | Metformin,Pioglitazone || [https://clinicaltrials.gov/ct2/show/NCT02733679 NCT02733679] || 2016-2019 || 英国 || Ataxia Telangiectasia || 4 || 終了 | ||
|- | |- | ||
| Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate || NCT01052623 || 2010-2011 || ドイツ || Ataxia Telangiectasia || 4 || 結果? | | Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate || [https://clinicaltrials.gov/ct2/show/NCT01052623 NCT01052623] || 2010-2011 || ドイツ || Ataxia Telangiectasia || 4 || 結果? | ||
|- | |- | ||
| amantadine sulphate || NCT00950196 || 2009-2011 || イスラエル || Ataxia Telangiectasia || 4 || 運動症状を改善 | | amantadine sulphate || [https://clinicaltrials.gov/ct2/show/NCT00950196 NCT00950196] || 2009-2011 || イスラエル || Ataxia Telangiectasia || 4 || [https://journals.sagepub.com/doi/10.1177/0883073812441999 運動症状を改善] | ||
|- | |- | ||
| Baclofen || NCT00640003 || 2008-2011 || 米国 || Ataxia Telangiectasia || 1 || 結果? | | Baclofen || [https://clinicaltrials.gov/ct2/show/NCT00640003 NCT00640003] || 2008-2011 || 米国 || Ataxia Telangiectasia || 1 || 結果? | ||
|} | |} | ||