NCT05244304已完成3 期
Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 104
- 试验地点
- 20
- 主要终点
- To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
研究概览
简要总结
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
详细描述
Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.
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
- Double (Participant, Investigator)
盲法说明
Eligible subjects will be randomly assigned to begin treatment in 2:1 ratio to receive the study drug (either Tinlarebant 5 mg or matching placebo)
入排标准
- 年龄范围
- 12 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects 12 to 20 years old, inclusive.
- •Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
- •Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
- •Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
- •Subject agrees to comply with all protocol requirements.
排除标准
- •Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect.
- •History of ocular surgery in the study eye in the last 3 months.
- •Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
- •Any prior gene therapy.
- •Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).
研究组 & 干预措施
Tinlarebant
Experimental
5 mg tablet taken orally once a day
干预措施: Tinlarebant (Drug)
Placebo
Placebo Comparator
Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.
干预措施: Placebo (Drug)
结局指标
主要结局
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
时间窗: Baseline thru month 24
次要结局
- To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline(Baseline thru month 24)
- To measure change in plasma concentration of RBP4 levels (μM) from baseline(Baseline thru month 24)
- The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline(Baseline thru month 24)
- To assess the systemic and ocular safety and tolerability of tinlarebant(Baseline thru month 24)
- To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline(Baseline thru month 24)
- To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline(Baseline thru month 24)
研究者
研究点 (20)
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