18. 脊髄小脳変性症（多系統萎縮症を除く。） Spinocerebellar degeneration Clinical trials / Disease details
臨床試験数 : 71 / 薬物数 : 99 - (DrugBank : 30) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 65
| 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 | 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 |