18. 脊髄小脳変性症（多系統萎縮症を除く。）
［臨床試験数：59，薬物数：78（DrugBank：28），標的遺伝子数：44，標的パスウェイ数：59］
Searched query = "Spinocerebellar degeneration", "SCD", "Spinocerebellar ataxia type I", "SCA1", "Spinocerebellar ataxia type II", "SCA2", "Spinocerebellar ataxia type III", "SCA3", "Machado-Joseph disease", "Spinocerebellar ataxia type VI", "SCA6", "Spinocerebellar ataxia type VII", "SCA7", "Spinocerebellar ataxia type X", "SCA10", "Spinocerebellar ataxia type XII", "SCA12", "Dentatorubural pallidoluysian atrophy", "Dentatorubropallidoluysial atrophy", "DRPLA", "Naito-Koyanagi disease", "Friedreich ataxia", "FRDA", "Ataxia with vitamin E deficiency", "AVED", "Early-onset ataxia with ocular motor ataxia and hypoalbuminemia", "EOAH"
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 | NCT01493973 (ClinicalTrials.gov) | January 2013 | 15/12/2011 | Efficacy Study of Epoetin Alfa in Friedreich Ataxia | A Double-blind, Randomized, Placebo-controlled, Clinical Trial to Test the Efficacy of Epoetin Alfa on Physical Performance of Friedreich Ataxia Patients. | Friedreich Ataxia | Drug: Epoetin alfa;Drug: Placebo | Federico II University | Friedreich's Ataxia Research Alliance;Associazione Italiana per la lotta alle Sindromi Atassiche (AISA) | Completed | 12 Years | N/A | Both | 56 | Phase 2 | Italy |
| 2 | EUCTR2011-006156-37-IT (EUCTR) | 10/07/2012 | 26/07/2012 | Erythropoieitn in Friedreich Ataxia | A double-blind, randomized, placebo-controlled, clinical trial to test the efficacy of Epoetin alfa on physical performance of Friedreich Ataxia patients. - FRIEMAX | FRIEDREICH ATAXIA MedDRA version: 14.1;Level: PT;Classification code 10008025;Term: Cerebellar ataxia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: EPREX*1SIR 40000UI/ML 1ML INN or Proposed INN: EPOETIN ALFA Trade Name: EPREX*1SIR 10000UI 1ML INN or Proposed INN: EPOETIN ALFA Trade Name: BINOCRIT*1SIR 40000UI/1ML INN or Proposed INN: EPOETIN ALFA | UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy | |||
| 3 | EUCTR2008-000040-13-AT (EUCTR) | 11/02/2009 | 27/01/2009 | Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia | Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia | Friedreich's ataxia (FRDA) is the most common autosomal recessive neurodegenerativ disease (1:50 000) affecting the central and peripheral nervous system. Extraneural organs are also affected during the course of the disease as a significant proportian of patients develop cardiomyopathy or diabetes. FRDA is caused by a GAA triplet expansion in the FRDA gene on chromosome 9q13 resulting in a loss of function of the gene product Frataxin. | Trade Name: Neorecormon Product Name: Neorecormon Product Code: EU1/97/031-032 INN or Proposed INN: H-116PI-DE.pdf Other descriptive name: Epoeitin beta | Medizinische Universität Innsbruck, Univ.-Klinik für Neurologie | NULL | Not Recruiting | Female: yes Male: yes | 7 | Phase 2 | Austria | ||
| 4 | EUCTR2007-003357-85-IT (EUCTR) | 10/10/2007 | 09/11/2007 | Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND | Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND | Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation in the FXN gene, which encodes a protein named frataxin. As a result of the mutation, frataxin is quantitatively reduced but qualitatively normal. Thus, any pharmacological agent able to increase frataxin intracellular levels would have a great therapeutic relevance. MedDRA version: 6.1;Level: PT;Classification code 10003591 | Trade Name: EPREX*1FL 40000UI/ML 1ML INN or Proposed INN: Erythropoietin Other descriptive name: Erythropoietin | ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Not Recruiting | Female: yes Male: yes | Italy |