An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 21
- 试验地点
- 5
- 主要终点
- Part A: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial
研究概览
简要总结
To determine the safety, tolerability and efficacy of a single administration of AAVB-039 via subretinal injection
研究设计
- 分配方式
- Na
- 主要目的
- Follow-up Period
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- •Aged ≥8 years to ≤55 years inclusive
- •Willingness to adhere to protocol per informed consent
- •No history of vitreoretinal or retinal macular surgery, No current history of cancer for 5 years, No history of myocardial infarct, stroke, or other heart conditions < 6 months
排除标准
- •Unwillingness to meet the requirements of the study
- •Participation in a clinical study with another Investigation Medicinal Product
- •Previous participation in another gene or cell therapy trial
- •Any condition that would preclude subretinal surgery
- •Complicating ocular and/or systemic diseases
研究组 & 干预措施
AAVB-039
干预措施: AAVB-039 (Drug)
结局指标
主要结局
Part A: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial
Part A: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial
Part B: Rate of change of area of ellipsoid zone (EZ) loss at 12 months
Part B: Rate of change of area of ellipsoid zone (EZ) loss at 12 months
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
次要结局
- Part A: Rate of change at 12, 24, and 60 months as measured by: Area of ellipsoid zone (EZ) loss, Area of atrophy as assessed via short wavelength fundus autofluorescence (SW-FAF), Macular sensitivity assessed via microperimetry, Best corrected visual acuity (BCVA) letter score, Low-luminance visual acuity (LLVA) letter score
- Part B: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial
- Part B: Rate of change at 12, 24, and 60 months as measured by: Area of ellipsoid zone (EZ) loss (24 and 60 months), Area of atrophy as assessed via short wavelength fundus autofluorescence (SW-FAF), Macular sensitivity assessed via microperimetry, Best corrected visual acuity (BCVA) letter score, Low-luminance visual acuity (LLVA) letter score
- Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039.
- Change in ellipsoid zone (EZ) loss following treatment with AAVB-039.
- Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
- Change in visual acuity following treatment with AAVB-039
研究者
Nicola Tuckwell
Scientific
Aavantgarde Bio S.r.l.