A 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
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 277
- 试验地点
- 49
- 主要终点
- Percentage of Participants with Adverse Events (AEs)
研究概览
简要总结
The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.
详细描述
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •CHM Participants
- •a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.
- •XLRP Participants
- •a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.
排除标准
- •Participants are not eligible for study participation if they meet the following exclusion criterion.
- •a. In the opinion of the Investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Untreated
Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.
BIIB111
Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.
干预措施: BIIB111 (Genetic)
BIIB112
Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.
干预措施: BIIB112 (Genetic)
结局指标
主要结局
Percentage of Participants with Adverse Events (AEs)
时间窗: Up to 5 years
Ophthalmic Examination Assessment: Lens Opacity Grading
时间窗: Up to 5 years
Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
时间窗: Up to 5 years
Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
时间窗: Up to 5 years
Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
时间窗: Up to 5 years
Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
时间窗: Up to 5 years
次要结局
- Change from Baseline in Best-Corrected Visual Acuity (BCVA)(Up to 5 years)
- Percentage of Participants with no Decrease from Baseline in BCVA or a Decrease from Baseline in BCVA of <5 ETDRS Letters in Choroideremia (CHM) Participants(Up to 5 years)
- Assessment of Microperimetry at Each Visit(Up to 5 years)
- Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)(Up to 5 years)
- Assessment of Fundus Photography at Each Visit(Up to 5 years)
- Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit(Up to 5 years)
- Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants(18 Months to 60 Months, Post-Day 0 Visits)
- Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants(Up to 5 years)
- Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants(Up to 5 years)
- Assessment of Fundus Autofluorescence (AF) at Each Visit(Up to 5 years)
- Change from Baseline in Visual Field(Up to 5 years)
- Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants(18 Months to 60 Months, Post-Day 0 Visits)