A Phase 1 Study of Intranasal Reduced Glutathione in Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Determination of Safety
研究概览
简要总结
Excessive free radical formation and depletion of the brain's primary antioxidant, glutathione, are established components of Parkinson's disease (PD) pathophysiology. While there is rationale for the therapeutic use of reduced glutathione (GSH) in PD, and even some preliminary evidence to suggest the use of GSH can lead to symptomatic improvement, obstacles surrounding currently employed delivery methods have hindered the clinical utility of this therapy. Intranasal GSH, (in)GSH, is a novel method of delivery for this popular CAM therapy in patients with PD, and bypasses the obstacles associated with other delivery methods. It has been used in clinical practice since 2005. The aim of this study is to evaluate safety, tolerability, and preliminary absorption data of (in)GSH in volunteers with PD in a Phase I single ascending dose escalation study.
详细描述
Individuals will be randomized to one of three treatment (100 mg GSH/ ml, 200 mg GSH/ ml, or placebo) arms in a double-blind fashion. All study medication will be administered 1 ml three times daily for three months, with a one-month wash out.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 21 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Parkinson's Disease made by neurologist within previous 10 years
- •Modified Hoehn and Yahr Stage <3
- •Subjects must be able to attend study visits at screening, baseline, weeks 4, 8, 12, 16
- •Subjects must be able to demonstrate self-administration of study medication or have active caregiver who can administer daily.
- •Dose and frequency of all pharmaceutical medications must be stable for one month prior to enrollment.
- •Diet, exercise and supplementation must be kept constant throughout participation in study
- •Ability to read and speak English
排除标准
- •Dementia as evidenced by Montreal Cognitive Assessment (MoCA) <24
- •Diseases with features common to Parkinson's Disease (eg. essential tremor, multiple system atrophy, progressive supranuclear palsy)
- •History of stroke, CVA
- •Elevated levels of ALT, AST, BUN or creatinine
- •Chronic sinusitis as defined by SNOT-20 score >1.0 on items 1-
- •Presence of other serious illness
- •History of brain surgery
- •History of structural brain damage
- •History of intranasal telangiectasia
- •Supplementation with glutathione and agents shown to increase glutathione will not be permitted and will require a 90 day washout period.
- •Pregnant or at risk of becoming pregnant.
研究组 & 干预措施
Intranasal GSH 100mg/ml
Study participant will be provided with monthly supply of study medication and will be asked to intake 100mg/ml of intranasal glutathione (n=15) An amount of 1ml with a frequency 3x per days and duration of 12 weeks with a dosage of 2100mg
干预措施: Intranasal glutathione - (in)GSH (Drug)
Intranasal glutathione 200mg/ml
Study participant will be provided with monthly supply of study medication and will be asked to intake 200/ml of intranasal glutathione (n=15) An amount of 1ml with a frequency 3x per days and duration of 12 weeks with a dosage of 4200mg
干预措施: Intranasal glutathione - (in)GSH (Drug)
Saline intranasal delivery
Study participant will be provided with monthly supply of study medication and will be asked to intake Intranasal saline delivery (n=15) An amount of 1ml with a frequency 3x per day with a duration of 12 weeks
干预措施: Saline Intranasal Delivery (Drug)
结局指标
主要结局
Determination of Safety
时间窗: 12 weeks
1a. Laboratory monitoring for adverse events will include CBC, ALT, AST, BUN, creatinine, uric acid, and urinalysis. Data will be collected throughout the 12-week intervention and at 1-mo following cessation of the study medication. 1b. Clinical adverse events will be measured using a daily patient diary and record score cards specifically screening for sinus irritation. Monitoring of Side Effects System (MOSES) will be used to screen for systemic and generalized adverse events. 1c. Effect on PD symptoms will be measured by the UPDRS to screen for accelerated disease activity.
Determination of Tolerability
时间窗: 12 weeks
Participants will be asked to keep a daily log and unused study medication will be measured at each clinical visit. Tolerability will be measured by frequency and severity of reported adverse events and withdrawal from study. The goal will be to identify the maximum tolerated dose (MTD) which will be defined as the highest dose achieving adherence, as defined as 80% of the group taking the prescribed dose 80% of the time.
次要结局
- Description of systemic absorption characteristics(12 weeks)