A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients With Leber Congenital Amaurosis Type 2

The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene.
Funding Source - FDA OOPD

开始日期2009-06-17

申办/合作机构

Beacon Therapeutics (USA), Inc.

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100 项与 Leber congenital amaurosis gene therapy - Applied Genetic Technologies Corporation 相关的临床结果

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100 项与 Leber congenital amaurosis gene therapy - Applied Genetic Technologies Corporation 相关的专利（医药）

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项与 Leber congenital amaurosis gene therapy - Applied Genetic Technologies Corporation 相关的文献（医药）

2018-12-01·Human Gene Therapy

Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

Article

作者: Thean, Beverly ; Pennesi, Mark E. ; Chegarnov, Elvira ; Flotte, Terence R. ; Whitebirch, Chris ; Humphries, Margaret ; Stout, J. Timothy ; Chulay, Jeffrey D. ; Weleber, Richard G. ; Yang, Paul ; Beasley, Kathleen N.

Previously, results at 2 years after subretinal injection of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in eight adults and four children with retinal degeneration caused by RPE65 mutations were reported. Now, results at 5 years after treatment in 11 of these subjects are reported. Subjects received a subretinal injection of rAAV2-CB-hRPE65 in the poorer-seeing eye, at either of two dose levels, and were followed for 5 years after treatment. The primary safety outcomes were ocular and non-ocular adverse events. Efficacy outcomes included changes in best corrected visual acuity, static perimetry hill of vision measurements for the central 30° (V₃₀), and total (V_TOT) visual field and kinetic perimetry visual field area. The only adverse events reported during years 3, 4, and 5 were minor intercurrent illnesses. Pediatric subjects had improvement in visual acuity and static perimetry in the treated eye, sometimes with a smaller improvement in the untreated eye, during the first 2 years of the study that persisted during years 3-5, with no consistent changes in kinetic perimetry during the study. Most adult subjects had no consistent changes in visual acuity or static perimetry during the study. Three adult subjects with markedly abnormal baseline kinetic visual field area had improvement in the treated eye during the first 1-2 years after treatment, but the absolute magnitude of the improvement was small and was not sustained at subsequent visits. There were no clinically significant adverse events. Visual acuity and static perimetry testing results suggest that treating patients at a younger age is associated with better visual function outcomes during 5 years after treatment.

2016-07-01·Ophthalmology1区 · 医学

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

1区 · 医学

Article

作者: Humphries, Margaret ; Hauswirth, William W ; Kaushal, Shalesh ; Jensen, Lauren ; Calcedo, Roberto ; Flotte, Terence R ; McBride, Maureen T ; Erker, Laura R ; Stout, J Timothy ; Chulay, Jeffrey D ; Pennesi, Mark E ; Wilson, David J ; Weleber, Richard G

PURPOSETo provide an initial assessment of the safety of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in adults and children with retinal degeneration caused by RPE65 mutations.DESIGNNonrandomized, multicenter clinical trial.PARTICIPANTSEight adults and 4 children, 6 to 39 years of age, with Leber congenital amaurosis (LCA) or severe early-childhood-onset retinal degeneration (SECORD).METHODSPatients received a subretinal injection of rAAV2-CB-hRPE65 in the poorer-seeing eye, at either of 2 dose levels, and were followed up for 2 years after treatment.MAIN OUTCOME MEASURESThe primary safety measures were ocular and nonocular adverse events. Exploratory efficacy measures included changes in best-corrected visual acuity (BCVA), static perimetry central 30° visual field hill of vision (V30) and total visual field hill of vision (VTOT), kinetic perimetry visual field area, and responses to a quality-of-life questionnaire.RESULTSAll patients tolerated subretinal injections and there were no treatment-related serious adverse events. Common adverse events were those associated with the surgical procedure and included subconjunctival hemorrhage in 8 patients and ocular hyperemia in 5 patients. In the treated eye, BCVA increased in 5 patients, V30 increased in 6 patients, VTOT increased in 5 patients, and kinetic visual field area improved in 3 patients. One subject showed a decrease in BCVA and 2 patients showed a decrease in kinetic visual field area.CONCLUSIONSTreatment with rAAV2-CB-hRPE65 was not associated with serious adverse events, and improvement in 1 or more measures of visual function was observed in 9 of 12 patients. The greatest improvements in visual acuity were observed in younger patients with better baseline visual acuity. Evaluation of more patients and a longer duration of follow-up will be needed to determine the rate of uncommon or rare side effects or safety concerns.

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
莱伯先天性黑蒙	临床2期	-	UMass Chan Medical School	-

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT00749957 (CTgov)	临床1/2期	2型利伯先天性黑朦症	12	rAAV2-CB-hRPE65 (Lower Dose of rAAV2-CB-hRPE65)	鹹淵艱構蓋膚顧醖範膚(遞蓋顧獵壓選醖窪鹹築) = 壓簾積窪獵餘遞獵築醖淵鹽壓積簾獵艱鹹憲鹹 (鹽糧獵壓製艱窪襯遞願, 壓鹽窪簾憲壓餘鹽淵範 ~ 鹹鬱鬱淵壓齋顧範積構) 更多	-	2016-04-08
				rAAV2-CB-hRPE65 (Higher Dose of rAAV2-CB-hRPE65)	鹹淵艱構蓋膚顧醖範膚(遞蓋顧獵壓選醖窪鹹築) = 遞構鑰鹹壓壓壓鏇願廠淵鹽壓積簾獵艱鹹憲鹹 (鹽糧獵壓製艱窪襯遞願, 餘廠憲簾鹽糧衊齋鹹衊 ~ 蓋鬱鬱淵選顧範齋選鹽) 更多