A Randomized, Double-Masked, Vehicle-Controlled, Adequate and Well-Controlled Pivotal Study With Co-Primary Endpoints to Evaluate the Safety and Efficacy of d-MAPPS™ Ophthalmic Solution in Subjects With Chronic Ocular Graft-Versus-Host Disease (oGVHD)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 6
- 主要终点
- Change From Baseline to Day 90 in Modified Oxford Corneal Staining Score
研究概览
简要总结
This is a Phase III, multicenter, randomized, double-masked, vehicle-controlled, parallel-group clinical trial designed to evaluate the efficacy, safety, and tolerability of d-MAPPS™ Ophthalmic Solution in adult subjects with chronic ocular graft-versus-host disease (oGVHD). Eligible subjects will receive masked study treatment for 90 days, with the primary efficacy assessment performed at the Day 90 study visit.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Age 18 years or older.
- •Documented chronic ocular graft-versus-host disease (oGVHD) following allogeneic hematopoietic stem cell transplantation.
- •Investigator determines the participant is medically stable and appropriate for study participation.
- •Ocular Surface Disease Index (OSDI) Total Score of at least 33 at Baseline.
- •UNC Dry Eye Management Scale Visual Analog Scale (UNC DEMS VAS) score of at least 3 at Baseline.
- •Modified Oxford Corneal Staining Score of at least 1 in at least one eye.
- •Snellen Corrected Distance Visual Acuity sufficient to complete protocol-defined assessments.
- •Written informed consent obtained before study procedures.
- •Willing and able to comply with study visits, study treatment, and protocol requirements.
- •Background therapies stable for at least 30 days before Baseline.
排除标准
- •Acute ocular graft-versus-host disease or ocular disease that may interfere with efficacy assessment.
- •Active ocular or periocular infection, ocular malignancy, or active herpetic keratitis.
- •Intraocular surgery, refractive surgery, or ocular laser procedure within 6 weeks before Baseline or planned during the study.
- •Change in systemic immunosuppressive therapy, topical glaucoma therapy, or oral tetracycline therapy within 30 days before Baseline.
- •Initiation within 30 days before Baseline or during the trial of topical cyclosporine, lifitegrast, tacrolimus, autologous serum eye drops, platelet-rich plasma eye drops, amniotic membrane products, or another prescription therapy intended to treat ocular surface disease.
- •Participation in another investigational study within 30 days before Screening or concurrent enrollment.
- •Pregnancy or breastfeeding.
研究组 & 干预措施
Vehicle Controlled
干预措施: Matching vehicle control (Biological)
Active Study Drug
干预措施: d-MAPPS™ Ophthalmic Solution (Biological)
结局指标
主要结局
Change From Baseline to Day 90 in Modified Oxford Corneal Staining Score
时间窗: Baseline to Day 90 (±5 days)
Corneal fluorescein staining will be assessed in the prespecified study eye using the Modified Oxford grading scale. Scores range from 0 to 5, where 0 represents no corneal staining and higher scores represent progressively greater staining. A decrease from Baseline indicates improvement.
Change From Baseline to Day 90 in Ocular Surface Disease Index (OSDI) Total Score
时间窗: Baseline to Day 90 (±5 days)
The Ocular Surface Disease Index (OSDI) will assess ocular symptoms and their effect on vision-related functioning. Total scores range from 0 to 100, with higher scores representing greater symptom burden. A decrease from Baseline indicates improvement.
次要结局
- Percentage of Participants With a ≥1-Grade Decrease in Modified Oxford Corneal Staining Score(Baseline to Day 90 (±5 days))
- Percentage of Participants With a ≥13-Point Decrease in OSDI Total Score(Baseline to Day 90 (±5 days))
- Change From Baseline to Day 90 in Tear Break-Up Time (TBUT)(Baseline to Day 90 (±5 days))
- Percentage of Participants Receiving Protocol-Defined Rescue Therapy(Randomization through Day 90)
- Time to First Administration of Protocol-Defined Rescue Therapy(Randomization through Day 90)
- Percentage of Participants With Protocol-Defined Treatment Failure(Randomization through Day 90)
- Change From Baseline to Day 90 in Supportive Efficacy Measures in the Fellow Eye(Baseline to Day 90 (±5 days))
- Percentage of Participants With Modified Oxford Corneal Staining Score Grade 0 at Day 90(Day 90 (±5 days))
- Change From Baseline to Day 90 in Superficial Punctate Keratitis (SPK) Severity(Baseline to Day 90 (±5 days))
- Percentage of Participants With a ≥1-Grade Decrease in Superficial Punctate Keratitis (SPK) Severity(Baseline to Day 90 (±5 days))
- Percentage of Participants With Superficial Punctate Keratitis (SPK) Severity of 0 at Day 90(Day 90 (±5 days))
- Change From Baseline to Day 90 in NIH Eye Score(Baseline to Day 90 (±5 days))
- Percentage of Participants With a ≥1-Grade Decrease in NIH Eye Score(Baseline to Day 90 (±5 days))
- Percentage of Participants With No Worsening in NIH Eye Score at Day 90(Baseline to Day 90 (±5 days))
- Change From Baseline to Day 90 in UNC Dry Eye Management Scale Visual Analog Scale Score(Baseline to Day 90 (±5 days))