Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 50
- 主要终点
- Slow the Rate of Clinical Decline
研究概览
简要总结
A remotely administered study, non-randomized, non-blinded, controlled parallel assignment phase 2 trial to determine if oral inosine or inositol hexaphosphate will provide an effective long-term therapy to combat or slow neural damage progression either concurrently with existing iron chelation therapy or during the natural course of the disorder. Clinical changes in hearing, balance, and mobility, and cognition will be assessed for 36 months through patient-reported outcomes of study assigned assessments.
详细描述
Capsules containing over-the-counter dietary supplement formulas of inosine 500 mg or 500 mg of inositol hexaphosphate(IP6) will be taken orally, two capsules two times per day for 36 months. Initial screening will determine patient arm assignment.
Before dosing begins, patients will submit copies of previous audiogram results (3yrs), radiology reports (3yrs), and MRI series (3yrs) if available and complete baseline assessment activities: Montreal Cognitive Assessment (MoCA) administered remotely, Timed Up, and Go (TUG), 2-Minute Walk Test (2-MWT), The Activities-specific Balance Confidence (ABC) Scale, iSS-QoL (custom) patient outcome reported
Assessment will take place four times: baseline, end-of-year 1 (12mo), end-of-year 2 (24mo), end-of-study (36mo).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 77 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of iSS (superficial siderosis of the CNS)
- •No illegal drug use
- •No history of myocardial infarction or stroke
- •No history of severe chronic obstructive pulmonary disease
- •Can safely swallow large capsules
- •Exhibits at least one confirmed iSS related symptom: Hearing Loss, Balance, Memory/Cognitive problems
- •Does not have a known hypersensitivity or intolerance to any study antioxidant
- •Has not taken part in another treatment study for any condition within the last 30 days (about four and a half weeks)
- •Not currently pregnant or breastfeeding
排除标准
- •Inosine Arm Exclusion :
- •Diagnosed with urate urolithiasis, or recurrent urolithiasis, all unknown type
- •History of Gout
- •History of Kidney Stones
- •P6 Arm Exclusion
- •Long-term anticoagulant
- •Known or suspected active bleed into the CNS
- •Currently undergoing deferiprone chelation therapy
- •Plan to begin deferiprone chelation therapy within three years
结局指标
主要结局
Slow the Rate of Clinical Decline
时间窗: 36 Months
Deceleration of clinical decline will be quantified by evaluating three primary areas- hearing, balance and mobility, and cognition. End-of-study quantifiable slowing of the historical and year-1 mean rate of decline across all three study areas with a measurable increase on Year-3 iSS-QOL from Year-1 iSS-QOL scoring.
次要结局
- Clinical Efficacy: Improved Quality of Life(36 Months)
- Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA)(36 Months)
- Clinical Efficacy: Rate of Change in Hearing(36 Months)
- Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability}(36 Months)
- Clinical Efficacy: Rate of Change in Mobility and Balance(36 Months)
- Clinical Efficacy: Hemosiderin Reduction (IP6 Arm)(36 Months)