AAV2-REP1 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
90網膜色素変性症5

90. 網膜色素変性症


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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
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
4NCT03584165
(ClinicalTrials.gov)
June 4, 201829/6/2018Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis PigmentosaA 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 StudyChoroideremia;X-Linked Retinitis PigmentosaGenetic: BIIB111;Genetic: BIIB112NightstaRx Ltd, a Biogen CompanyNULLEnrolling by invitation10 YearsN/AMale440Phase 3United States;Brazil;Canada;Denmark;Finland;France;Germany;Netherlands;United Kingdom
5EUCTR2017-003104-42-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