NCT07158775招募中1 期
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAGChronosFP) in Patients With Geographic Atrophy Secondary to Dry AMD
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- [Phase 1] Number of the dose limiting toxicities (DLTs), incident and severity of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
研究概览
简要总结
This is a Phase 1/2 study, multi-center, dose-escalation interventional study of BS01 in subjects with GA secondary to dry AMD. Part 1 is an open label dose-escalation study; Part 2 is a dose-expansion study with dose(s) selected from Part 1 based on a benefit/risk assessment, and an untreated (sham injection) group to allow for a controlled comparison of efficacy and safety.
This is a seamless Phase 1/2 study in up to 10 patients for Phase 1 and 30 patients Phase 2 in patients with GA secondary to dry AMD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 50 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •General Inclusion Criteria
- •Signed informed consent obtained before screening.
- •Men or women between 50 and 85 years of age inclusive at the time of signing the informed consent.
- •Ophthalmic Inclusion Criteria
- •Geographic atrophy with some macula foveal involvement secondary to dry AMD.
- •Total GA area ≥ 5 and ≤ 17.5 mm2 (2 and 7 disk areas respectively), based on Heidelberg Region Finder or equivalent automated software.
- •If GA is multifocal, at least one focal lesion should measure ≥ 1.25 mm2 (0.5 disk area) to ensure measurable focal effects for efficacy evaluation.
- •Composite lesion encompassing confluent GA regions with total area meeting thresholds given in 2 above.
- •GA in part within 1200 microns from the foveal center.
- •The atrophic lesion must be able to be photographed in its entirety.
- •BCVA between 20/50 to 20/400, inclusive, using letter score per EDTRS chart.
- •Clear ocular media and adequate pupillary dilatation in both eyes to allow for all imaging procedures, including good quality stereoscopic fundus photography and fundus autofluorescence (FAF).
- •Central fixation.
排除标准
- •General Exclusion Criteria
- •Previous therapeutic radiation in the region of the SE.
- •Previous treatment with any ocular or systemic gene transfer product.
- •Any treatment with an investigational agent in the past 60 days for any condition.
- •Women who are pregnant or nursing.
- •Known hypersensitivity to topical ocular anesthetics or diagnostic drops to be used during the study.
- •Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.
- •Ophthalmic Exclusion Criteria
- •> 30% difference of BCVA using EDTRS in two baseline visual acuity assessments with at least 14 days apart during screening.
- •Any intraocular surgery or thermal laser within 3 months of study entry. Any prior thermal laser in the macular region, regardless of indication.
- •Any ocular or periocular infection (including blepharitis), or ocular surface inflammation in the past 12 weeks.
- •History of any of the following procedures: Posterior vitrectomy, filtering surgery (e.g., trabeculectomy), glaucoma drainage device, corneal transplant, or retinal detachment.
- •Any sign of diabetic retinopathy in either eye.
- •Intraocular pressure (IOP) > 25 mmHg in either eye.
- •Completely atrophic centrally located lesions based on ellipsoid zone (EZ) loss in spectraldomain optical coherence tomography (SD-OCT).
研究组 & 干预措施
BS01 (low dose)
Experimental
干预措施: BS01 (Biological)
Sham control
Sham Comparator
干预措施: Sham procedure control (Other)
BS01 (high dose)
Experimental
干预措施: BS01 (Biological)
结局指标
主要结局
[Phase 1] Number of the dose limiting toxicities (DLTs), incident and severity of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: 12 months
[Phase 2] Change from baseline in BCVA (Best Corrected Visual Acuity)
时间窗: 12 months
次要结局
未报告次要终点
研究者
研究点 (1)
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