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.
Who can participate
Age range16 Years – 77 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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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What they're measuring
1
Slow the Rate of Clinical Decline
Timeframe: 36 Months
Trial details
NCT IDNCT04890808
SponsorSuperficial Siderosis Research Alliance Inc.