Clinical trial NCT04890808: Therapeutic Antioxidant Supplementation
Official title: Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Superficial Siderosis
Keywords: Hemosiderin
Iron Toxicity
Superficial siderosis of the CNS
Infratentorial superficial siderosis
Neurodegenerative disorder
Subpial
Lead sponsor: Superficial Siderosis Research Alliance Inc.
Sponsor class: OTHER
First posted: 2021-05-18
Enrollment: 50
Sex: ALL
Minimum age (years): 16
Maximum age (years): 77
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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).

Eligibility criteria: 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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Primary outcomes:
measure: Slow the Rate of Clinical Decline
timeFrame: 36 Months
Secondary outcomes:
measure: Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability}
timeFrame: 36 Months
measure: Clinical Efficacy: Improved Quality of Life
timeFrame: 36 Months
measure: Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA)
timeFrame: 36 Months
measure: Clinical Efficacy: Rate of Change in Hearing
timeFrame: 36 Months
measure: Clinical Efficacy: Rate of Change in Mobility and Balance
timeFrame: 36 Months
measure: Clinical Efficacy: Hemosiderin Reduction (IP6 Arm)
timeFrame: 36 Months
Other outcomes:
Locations:
Study officials:
affiliation: Superfical Siderosis Research Alliance
name: Rori-Suzanne Daniel
role: STUDY_DIRECTOR
References: