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NCT Number: NCT04890808

Therapeutic Antioxidant Supplementation

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.

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Key information

Conditions

Superficial Siderosis Nervous System Diseases Neurodegenerative Diseases

Age range

16 year–77 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

-

Treatment and study plan

Inosine

Dietary Supplement

Patients will be dosed with two 500 mg capsules of Inosine twice daily for 36 months

IP6

Dietary Supplement

Patients will be dosed with two 500 mg capsules of IP6 twice daily for 36 months

Primary outcomes

  1. 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

  1. Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability}

    Time frame: 36 Months

    Therapeutic use of antioxidants through a safe, effective dose range and schedule to the extent that the risk-benefit relationship is acceptable for the patient. Evaluation will include the overall adverse event (AE) and serious AE (SAE) rate.

  2. Clinical Efficacy: Improved Quality of Life

    Time frame: 36 Months

    The rate of change in points on the iSS-QoL(custom) from baseline assessment to end-of-study will be scored based on 16 questions with a point range of 1-5. Higher end-of-study scores indicate greater satisfaction.

  3. Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA)

    Time frame: 36 Months

    The rate of change in points on the Montreal Cognitive Assessment (MoCA) scale from baseline assessment to end-of-study will be assessed for patients in each treatment group. The MoCA assesses attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Points (range 0-30) are awarded for the correct completion of MoCA tasks. Cognition decline will be defined as ≥ 3pt decline of total MoCA score from baseline assessment to final 36mo testing assessment.

  4. Clinical Efficacy: Rate of Change in Hearing

    Time frame: 36 Months

    The baseline audiogram will be compared to the post-study audiogram for each patient. The hearing levels in decibels against the frequency in hertz will be compared pre-study, baseline, and end-of-study for average rate-of-decline. A paired t-test will be used to determine statistical significance in the average difference in the threshold hearing level at any particular frequency.

  5. Clinical Efficacy: Rate of Change in Mobility and Balance

    Time frame: 36 Months

    The Timed Up & Go (TUG), 2-Minute Walk Test (2MWT), and Activities-specific Balance Confidence (ABC) Scale will be scored individually. The baseline score will be compared to the annual and end-of-study scores for each patient. A paired t-test will be used to determine statistical significance in the average difference.

  6. Clinical Efficacy: Hemosiderin Reduction (IP6 Arm)

    Time frame: 36 Months

    Visible hemosiderin deposition reduction on MRI series from baseline visit to end-of-study. MRIs will be objectively analyzed for changes in iron deposition.

Study contacts

Contact information is provided by the study sponsor or research team.

Rori-Suzanne Daniel

CONTACT

[email protected]

9035594123

Sponsors and collaborators

Lead sponsor

Superficial Siderosis Research Alliance Inc.

Other

Registry information

Official study title

Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis

Acronym: TAS-SS21

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
May 18, 2021
Registry last updated
May 5, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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