Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
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.
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).
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
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administrator@superficialsiderosis.org9035594123