Aav8-rpgr    (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3

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.
3 / 103 trials found
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PhaseCountries
1EUCTR2017-003104-42-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
2NCT03116113
(ClinicalTrials.gov)
March 16, 201729/3/2017A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis PigmentosaA 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)X-Linked Retinitis PigmentosaBiological: AAV8-RPGRNightstaRx Ltd, a Biogen CompanyNULLRecruiting10 YearsN/AMale63Phase 2;Phase 3United States;United Kingdom
3EUCTR2016-003852-60-GB
(EUCTR)
09/01/201709/11/2016A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa using AAV8A 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 LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
63Phase 1;Phase 2;Phase 3United States;United Kingdom