NCT06852963进行中（未招募）1 期
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 Weeks
PYC Therapeutics12 个研究点 分布在 1 个国家目标入组 17 人开始时间: 2025年6月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 17
- 试验地点
- 12
- 主要终点
- To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.
研究概览
简要总结
This is a Phase 1/2 repeat-dose, open-label, two-arm, parallel group safety and efficacy study of two doses of VP-001 (30 μg and 75 μg) in participants with confirmed PRPF31 mutation-associated retinal dystrophy, including participants previously treated with VP001 in the PLATYPUS Study or WALLABY Study for a minimum of 8 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female sex; ≥18 years of age at Day 1/Baseline (Visit 2)
- •May have been previously enrolled in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol #VP001-CL102) study. At Screening Visit in this study, participants must have completed at least 8 weeks after last study agent administration in PLATYPUS Part B (Protocol #VP001-CL101) or WALLABY (Protocol # VP001-CL102) study
- •Have a confirmed clinical diagnosis of Retinitis Pigmentosa.
- •Have a confirmed genetic diagnosis of Retinitis Pigmentosa secondary to mutation in the PRPF31 gene.
- •For participants not previously enrolled in VP001-CL101 or VP001-CL102 studies: Meet all of the following for visual function in the study eye at the Screening Visit:
- •Mean microperimetry threshold: >5 decibel (dB) to <15 dB
- •Ellipsoid zone (EZ) length >1000 microns of which 500 microns is contiguous, by SD-OCT
- •In the opinion of the Investigator, rod function is observed in any direction >10 degrees per static perimetry at Screening Visit (Visit 1)
排除标准
- •Have any uncontrolled systemic disease that, in the opinion of the Investigator, would preclude participation in the study that include but are not limited to infection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemic control issues, or any other medical condition that may put the participant at risk due to study procedures.
- •Known mutations in genes that cause autosomal dominant RP, X-linked RP, or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PRPF31 mutations.
- •Have used anti-VEGF agents within 2 months or corticosteroid injections within the last 3 months.
- •Have had Ozurdex® implants placed within 3 months or Retisert® or Iluvien® implants placed within 3 years prior to Baseline (Visit 2).
- •Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinal surgery or any other ocular surgery
- •Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the investigator.
- •Have used any investigational drug or device within 90 days or 5 estimated half-lives (or within 60 days from last administration of VP-001 in the VP001-CL101 Part B or VP001-CL102 studies) of Baseline (Visit 2), whichever is longer,
- •Have a recent history (<6 months) or current excessive recreational drug or alcohol use, in the opinion of the investigator.
研究组 & 干预措施
Cohort 1: 30ug VP-001 every 8 weeks
Experimental
干预措施: VP-001 (Drug)
Cohort 2: 75ug of VP-001 every 12 weeks
Experimental
干预措施: VP-001 (Drug)
结局指标
主要结局
To determine the safety of two doses of repeatedly administered intravitreally VP-001 in participants with confirmed PRPF31 mutation-associated retinal dystrophy.
时间窗: 26 months
The incidence, severity, and relatedness of ocular TEAEs and TE-SAEs in the study eye over a 26-month time period for each of the repeat doses
次要结局
- To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ)(24 months)
- To determine the change from Baseline (Visit 2) through End of Study/Early Termination in BCVA letter score using ETDRS charts(24 months)
- To determine the change from baseline (Visit 2) through End of Study/Early Termination in Low Luminance Visual Acuity (LLVA) letter score(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in visual field sensitivity, Mean deviation (Mean Defect) as measured by standard static perimetry (Humphries)(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in mean retinal sensitivity(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in preserved EZ area on SD-OCT(24 months)
- Change from Baseline (Visit 2) through End of Study/Early Termination in participant reported outcome measures utilizing the Michigan Retinal Degeneration Questionnaire (MRDQ)(24 months)
研究者
研究点 (12)
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