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

  
4 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2017-003104-42-DK
(EUCTR)
08/02/202101/07/2020A 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 studyA 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)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3Netherlands;Germany;United Kingdom;France;United States;Canada;Finland;Brazil;Denmark
2EUCTR2017-003104-42-FI
(EUCTR)
20/11/201816/10/2018A 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 studyA 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)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Brazil;Denmark;Netherlands;Germany;United Kingdom
3EUCTR2017-003104-42-GB
(EUCTR)
26/06/201806/02/2018A 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 studyA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom
4EUCTR2017-003104-42-DE
(EUCTR)
05/01/201825/09/2017A 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 studyA 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)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom