2024-519535-42-00招募中2 期
A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (ASTRA).
Splicebio S.L.2 个研究点 分布在 1 个国家目标入组 10 人开始时间: 2025年7月14日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- The primary endpoint is safety and tolerability through Week 96 assessed by incidence and/or clinically significant changes in ocular and non-ocular adverse events (AEs).
研究概览
简要总结
The objective of this study is to evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt disease type 1 (STGD1) confirmed genotypically to have bi-allelic ABCA4 gene mutations.
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Provide written consent.
- •Are male or female subjects ≥18 to ≤65 years (inclusive).
- •Are able to understand and comply with the study procedures.
- •Have a diagnosis of Stargardt disease type 1 (STGD1).
- •Clinical evidence consistent with Stargardt Disease type
- •For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day
- •For both WOCBP and male subjects (or their female partners who are of child-bearing potential), agree to either strict abstinence or, if sexually active, use an acceptable contraception measure for 4 months from Day
- •Must have clear ocular media and adequate pupillary dilation in the study eye, including no allergy to dilating eyedrops, to permit good quality retinal imaging.
排除标准
- •Have had any intraocular surgery (including cataract surgery) or thermal laser within 90 days of the Screening Visit or planned intraocular surgery (including cataract surgery) or thermal laser during the period of the study, in the study eye.
- •Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.
- •Have had any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the period of the study.
- •Have two pathogenic or likely pathogenic variants in inherited retinal dystrophy (IRD) genes (other than ABCA4) or a single pathogenic or likely pathogenic variant in autosomal dominant or X-linked IRD genes.
- •Have a history of amblyopia in the study eye.
- •Are unwilling to stop taking the following products at Screening and throughout the study: a. Supplements containing vitamin A or beta-carotene, liver-based products. b. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary.
- •Have any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal or supra-choroidal injections.
- •Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.
- •Have known serious allergies to the fluorescein dye.
- •Have any significant ocular or non-ocular disease/disorder.
结局指标
主要结局
The primary endpoint is safety and tolerability through Week 96 assessed by incidence and/or clinically significant changes in ocular and non-ocular adverse events (AEs).
The primary endpoint is safety and tolerability through Week 96 assessed by incidence and/or clinically significant changes in ocular and non-ocular adverse events (AEs).
次要结局
- 3.Change from baseline in total lesion size growth (as measured by DDAF and well demarcated questionably decreased autofluorescence (QDAF)) on FAF imaging.
- 4. Change from baseline in ellipsoid zone area as measured by spectral-domain optical coherence tomography (SD-OCT).
- 5. Change from baseline in retinal sensitivity based on microperimetry.
- 6. Change from baseline in best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS).
- 7. Change from baseline in low luminance visual acuity (LLVA) using ETDRS.
- 8. Change from baseline in contrast sensitivity scores.
- 1. Change from baseline in lesion size growth (as measured by definitely decreased autofluorescence [DDAF]) on fundus autofluorescence (FAF) imaging compared between SB-007 and untreated control.
- 2. Change from baseline in lesion size growth (as measured by DDAF) on FAF imaging.
研究者
Leigh Shaw
Scientific
Splicebio S.L.
研究点 (2)
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