303. アッシャー症候群 Usher syndrome Clinical trials / Disease details
臨床試験数 : 9 / 薬物数 : 13 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
| 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 | EUCTR2012-002574-31-FR (EUCTR) | 25/10/2013 | 15/05/2014 | Study of SAR421869 in Patients With Retinitis Pigmentosa associated with Usher Syndrome Type 1B | A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B | Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B. MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10063396;Term: Usher's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: UshStat (SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed INN: Lentiviral vector containing the human MY07A gene Other descriptive name: UshStat | sanofi-aventis recherche & développement | NULL | Not Recruiting | Female: yes Male: yes | 27 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France | ||
| 2 | NCT02065011 (ClinicalTrials.gov) | September 12, 2013 | 14/2/2014 | A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B | An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B | Usher's Syndrome | Drug: Blood draw for the laboratory assessment | Sanofi | NULL | Active, not recruiting | 18 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;France |
| 3 | NCT01505062 (ClinicalTrials.gov) | March 26, 2012 | 4/1/2012 | Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | Usher Syndrome;Retinitis Pigmentosa | Drug: SAR421869 | Sanofi | NULL | Terminated | 6 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;France |