90. 網膜色素変性症 Retinitis pigmentosa Clinical trials / Disease details
臨床試験数 : 147 / 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110
| 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 | EUCTR2017-003104-42-DK (EUCTR) | 08/02/2021 | 01/07/2020 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2- REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR INN or Proposed INN: Cotoretigene Toliparvovec Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | Netherlands;Germany;United Kingdom;France;United States;Canada;Finland;Brazil;Denmark | ||
| 2 | EUCTR2017-003104-42-FI (EUCTR) | 20/11/2018 | 16/10/2018 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR INN or Proposed INN: Cotoretigene Toliparvovec Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Brazil;Denmark;Netherlands;Germany;United Kingdom | ||
| 3 | EUCTR2017-003104-42-GB (EUCTR) | 26/06/2018 | 06/02/2018 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study - SOLSTICE | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 Product Code: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: AAV2-REP1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom | ||
| 4 | NCT03584165 (ClinicalTrials.gov) | June 4, 2018 | 29/6/2018 | Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia;X-Linked Retinitis Pigmentosa | Genetic: BIIB111;Genetic: BIIB112 | NightstaRx Ltd, a Biogen Company | NULL | Enrolling by invitation | 10 Years | N/A | Male | 440 | Phase 3 | United States;Brazil;Canada;Denmark;Finland;France;Germany;Netherlands;United Kingdom |
| 5 | EUCTR2017-003104-42-DE (EUCTR) | 05/01/2018 | 25/09/2017 | A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent study | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RetinalGene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study | Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP) MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853 MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV2-REP1 INN or Proposed INN: timrepigene emparvovec Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1 Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Ltd (A Biogen Company) | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 440 | Phase 3 | France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom | ||
| 6 | EUCTR2016-003852-60-GB (EUCTR) | 09/01/2017 | 09/11/2016 | A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa using AAV8 | A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) - XIRIUS Study | X-Linked retinitis pigmentosa (XLRP) MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: AAV8-RPGR Product Code: AAV8-RPGR Other descriptive name: AAV8-RPGR | NightstaRx Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 63 | Phase 1;Phase 2;Phase 3 | United States;United Kingdom |