90. 網膜色素変性症
［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］
Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
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 | EUCTR2013-005393-22-NL (EUCTR) | 04/01/2019 | 20/09/2016 | A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision. | A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | QLT Inc. | NULL | NA | Female: yes Male: yes | 48 | Phase 3 | France;United States;Canada;Brazil;Denmark;Germany;Netherlands;United Kingdom;Switzerland | |||
| 2 | EUCTR2013-005393-22-DK (EUCTR) | 03/11/2016 | 06/09/2016 | A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision. | A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QLT091001 Product Code: QLT091001 INN or Proposed INN: Zuretinol Acetate Other descriptive name: QLT091001 | QLT Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | France;United States;Canada;Brazil;Denmark;Netherlands;Germany;United Kingdom;Switzerland | ||
| 3 | EUCTR2011-004214-42-NL (EUCTR) | 04/10/2012 | 23/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital AmaurosisRetinitis pigmentosa MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | United States;Canada;Germany;Netherlands;United Kingdom | |||
| 4 | EUCTR2011-004214-42-GB (EUCTR) | 03/09/2012 | 02/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital Amaurosis Retinitis pigmentosa MedDRA version: 20.0;Level: PT;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | Phase 1 | United States;Canada;Netherlands;Germany;United Kingdom | |||
| 5 | EUCTR2011-004214-42-DE (EUCTR) | 06/08/2012 | 03/05/2012 | A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | Leber Congenital AmaurosisRetinitis pigmentosa MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Code: QLT091001 | QLT Inc. | NULL | Not Recruiting | Female: yes Male: yes | 31 | United States;Canada;Netherlands;Germany;United Kingdom | |||
| 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 | NCT01543906 (ClinicalTrials.gov) | February 2012 | 17/2/2012 | Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) | An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65) | Retinitis Pigmentosa (RP) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 18 Years | 70 Years | Both | 5 | Phase 1 | Canada;Ireland |
| 7 | NCT01521793 (ClinicalTrials.gov) | January 2012 | 6/1/2012 | Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) | An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) | LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 5 Years | 65 Years | Both | 27 | Phase 1 | United States;Canada;Germany;Netherlands;United Kingdom |
| 8 | NCT01014052 (ClinicalTrials.gov) | November 2009 | 12/11/2009 | Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations | Phase 1b Study to Evaluate QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) | LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa) | Drug: QLT091001 | QLT Inc. | NULL | Completed | 5 Years | 65 Years | Both | 32 | Phase 1 | United States;Canada;Germany;Netherlands;United Kingdom |