Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Sponsor
- Enrollment
- 50
- Primary Endpoint
- Slow the Rate of Clinical Decline
Study Overview
Brief Summary
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.
Detailed Description
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).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Supportive Care
- Masking
- None
Eligibility Criteria
- Ages
- 16 Years to 77 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Outcomes
Primary Outcomes
Slow the Rate of Clinical Decline
Time Frame: 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.
Secondary Outcomes
- 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)