2023-507994-16-00尚未招募3 期
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 (SOLSTICE)
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 54
- 试验地点
- 3
- 主要终点
- Safety assessments will be performed as per protocol
研究概览
简要总结
To evaluate the long-term safety and efficacy of a sub retinal injection of: • AAV2-REP1 in participants with CHM who have been previously treated with AAV2-REP1 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR study. • AAV8-RPGR in participants with XLRP who have been previously treated with AAV8-RPGR and who have exited an antecedent study.
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •CHM Participants: a. Are willing and able to give informed consent for participation in the study, and
- •CHM Participants: b. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of AAV2- REP1 for CHM
- •XLRP Participants: a. Are willing and able to give informed consent for participation in the study
- •XLRP Participants: b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited an antecedent study
排除标准
- •In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
结局指标
主要结局
Safety assessments will be performed as per protocol
Safety assessments will be performed as per protocol
次要结局
- Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart
- Proportion of participants with no decrease from Baseline in BCVA or a decrease from Baseline in BCVA of <5 ETDRS letters (in CHM subjects only)
- Proportion of participants with an increase from Baseline in BCVA of ≥ 10 ETDRS letters (in CHM subjects only)
- Proportion of participants with an increase from Baseline in BCVA of ≥ 15 ETDRS letters (in CHM subjects only)
- Available assessments of fundus autofluorescence at each visit
- Available assessments of fundus photography at each visit
- Available assessments of spectral-domain optical coherence tomography (SD-OCT) at each visit
- Available assessments of microperimetry at each visit
- Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25)
- Change from Baseline in visual field (in AAV8-RPGR-treated participants only)
- Proportion of participants with an increase from Baseline in low luminance visual acuity (LLVA) of ≥10 ETDRS letters (in AAV8-RPGRtreated participants only)
- Proportion of participants with an increase from Baseline in LLVA of ≥ 15 ETDRS letters (in AAV8-RPGR-treated participants only)
- Datasets from the 2 disease populations will be analyzed separately.
研究者
CTA Lead Department
Scientific
Biogen Idec Research Limited
研究点 (3)
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