Open Label, Interventional Single Arm Study Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 3
- 主要终点
- change in BVCA
研究概览
简要总结
Summary of the Study This clinical trial evaluates a novel peptide-based therapy for treating retinal dystrophies, age-related macular degeneration (AMD), and diabetic retinopathy (DR). The therapy consists of peptides derived from fetal tissues, mesenchymal stem cells (MSCs), and bioactive growth factors, administered sublingually for systemic absorption.
Study Objectives:
Primary Objectives: Assess safety and tolerability, and evaluate the therapy's effects on retinal function and structure.
Secondary Objectives: Explore improvements in visual acuity, retinal thickness, vascular health, and disease biomarkers.
Study Design:
Type: Open-label, single-arm interventional study. Duration: 12 months.
Participants: 150 adults, divided into three cohorts:
Retinal dystrophies. AMD (dry and wet forms). DR (moderate NPDR and PDR).
Intervention:
A sublingual solution containing peptides and growth factors, taken 4 times daily.
Outcome Measures:
Primary Outcomes: Safety (adverse events) and tolerability (treatment adherence).
Secondary Outcomes:
Functional: Visual acuity and field sensitivity improvements. Structural: Retinal thickness and vascular health. Biomarkers: Serum VEGF, oxidative stress, and inflammatory markers.
Study Procedures:
Monthly follow-ups for safety monitoring, vision tests, retinal imaging (OCT, FA), and blood biomarker analysis.
Comprehensive evaluations at baseline, 6 months, and 12 months. Significance: The study aims to provide an innovative, non-invasive treatment for debilitating retinal conditions, potentially improving vision and retinal health through systemic therapy.
详细描述
Protocol for Evaluating a Novel Peptide-Based Therapy in Retinal Dystrophies, Age-Related Macular Degeneration, and Diabetic Retinopathy
Authors :
Mohamed Yasser Sayed Saif Prof of ophthalmology , Beni-Suef University Sherin Amer Family medicine consultant, Alaa Abdelkarim Endocrinology consultant , ACE Cells Lab Ahmed Tamer Sayed Saif Assistant Professor of ophthalmology, Fayoum University Passant Sayed Saif Assistant Professor of Ophthalmology, Misr University for Science and Technology
- Background and Rationale Retinal diseases such as retinal dystrophies, age-related macular degeneration (AMD), and diabetic retinopathy (DR) are among the leading causes of blindness worldwide. These conditions result from different pathophysiological mechanisms but share common features like oxidative stress, chronic inflammation, and cellular degeneration.
The novel therapy under evaluation consists of low molecular weight peptides derived from fetal tissues and mesenchymal stem cells (MSCs). The formulation also incorporates bioactive growth factors and essential peptides to support retinal health and regeneration. Sublingual administration ensures systemic absorption and avoids invasive delivery methods. 2. Objectives
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •• Adults aged 18-65 years with a confirmed genetic or clinical diagnosis of RP.
- •Visual acuity of ≥1/60 in at least one eye.
- •Stable ocular and systemic condition over the past six months.
- •Ability to provide written informed consent
排除标准
- •• Significant ocular comorbidities (e.g., advanced glaucoma, diabetic retinopathy).
- •Recent ocular surgery (within six months) or current use of investigational drugs.
- •Systemic conditions affecting study outcomes (e.g., uncontrolled diabetes, autoimmune diseases).
- •Pregnancy or lactation.
结局指标
主要结局
change in BVCA
时间窗: 3 months
change in BCVA (best-corrected visual acuity). The calculation is based on detecting a clinically meaningful improvement in BCVA using a paired design (before and after treatment within the same subjects).
次要结局
- change in OCT(3 months)