RAAV2tYF-GRK1-RPGR ( DrugBank: - )

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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	2

90. 網膜色素変性症

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臨床試験数 : 130 /  薬物数 : 180 - (DrugBank : 41) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 109

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	NCT04850118 (ClinicalTrials.gov)	August 2021	5/4/2021	A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations	A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene	X-Linked Retinitis Pigmentosa	Biological: rAAV2tYF-GRK1-hRPGRco	Applied Genetic Technologies Corp	NULL	Not yet recruiting	8 Years	50 Years	Male	63	Phase 2/Phase 3	Israel;Netherlands;United States
2	NCT03316560 (ClinicalTrials.gov)	April 16, 2018	10/10/2017	Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations	A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene	X-Linked Retinitis Pigmentosa	Biological: rAAV2tYF-GRK1-RPGR	Applied Genetic Technologies Corp	NULL	Recruiting	6 Years	50 Years	Male	42	Phase 1/Phase 2	United States