NCT07710196尚未招募3 期
A 24-Month, Randomized, Double-Masked, Placebo-Controlled Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis PigmentosaAssociated With Usher Syndrome
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 80
- 主要终点
- Ellipsoid Zone Area
研究概览
简要总结
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001. The main questions it aims to answer are:
Does NPI-001 slow down the loss of photoreceptors? What medical problems do participants have when taking NPI-001? Researchers will compare NPI-001 to a placebo (a look-alike substance that contains no drug) to see if NPI-001 works to preserve vision.
Participants will:
Take NPI-001 or a placebo twice a day, every day for 24 months and visit the clinic 9 times for checkups and tests.
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
- •Able to comprehend and willing to sign an informed consent form (OCF) and adhere to the study protocol.
- •Diagnosed with RP associated with Usher syndrome.
- •EZ width ≥ 500 microns, which includes the fovea in each eye at Visit
- •All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening).
- •Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits).
- •On stable dose of medications associated with other conditions for at least one month.
- •Willing to use contraception during study (for anyone of childbearing potential).
排除标准
- •Concurrent retinal pathologies that result in vision loss or inability to fixate.
- •Intraocular surgery within the last two months or capsulotomy within the last month.
- •Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents visualization of the posterior pole.
- •Likely to require cataract surgery within the next 12 months.
- •Unstable fixation during microperimetry in either eye at either Screening or Baseline visits.
- •Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment.
- •Use of N-acetylcysteine containing products in the previous 30 days prior to the baseline visit or unwilling to refrain from such supplements for the duration of the study.
- •Chronic liver or kidney disease, cystic fibrosis, severe asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia.
- •Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values > 1.5 X the upper limit of normal (ULN) at screening.
- •Platelet or hemoglobin values < 100 at screening.
- •History of known sensitivity to N-acetylcysteine or similar thiol compounds or any ingredients of NPI-
- •History of hypersensitivity to any medication or food resulting in systemic symptoms.
- •History of cancer (other than non-melanoma skin cancer) diagnosed or requiring treatment within the past 2 years.
- •Pregnant women or women planning to become pregnant in the next 25 months or men with partners planning to become pregnant in the next 25 months.
- •Lactating women who are breast-feeding.
- •Potential participant lives in the same household as a current participant in this study.
- •Subjects who are unwilling or unable to refrain from donation of blood from 30 days prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening until 56 days following the last study drug dose administered.
- •Inability to provide blood samples, including difficulty with venous access.
- •Any reason, in the opinion of the Principal Investigator, the participant should not participate.
结局指标
主要结局
Ellipsoid Zone Area
时间窗: 24 months
次要结局
未报告次要终点
研究者
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