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Therapeutic Antioxidant Supplementation

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 16 Years - 77 Years
Gender All
More Inclusion & Exclusion 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. -

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04890808
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

N/A
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Superficial Siderosis Research Alliance Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Rori-Suzanne Daniel
Principal Investigator Affiliation Superfical Siderosis Research Alliance
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Not yet recruiting
Countries
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Superficial Siderosis
Study Website: View Trial Website
Additional Details

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).

Arms & Interventions

Arms

Experimental: Over-the-Counter Dietary Supplement Inosine

capsules containing 500 mg of inosine Two capsules Twice Daily Other Name: hypoxanthine 9-╬▓-D-ribofuranoside 9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-hydroxymethyl) oxolan-2-yl]-1H-purin-6-one

Experimental: Over-the-Counter Dietary Supplement IP6

capsules containing 500mg of IP6 Two capsules Twice Daily Other Name: Inositol hexaphosphate (1R,2S,3r,4R,5S,6s)-cyclohexane-1,2,3,4,5,6-hexayl hexakis[dihydrogen (phosphate)]

Interventions

Dietary Supplement: - Inosine

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

Dietary Supplement: - IP6

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

Contact Information

This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:

Rori-Suzanne Daniel

administrator@superficialsiderosis.org

9035594123

For additional contact information, you can also visit the trial on clinicaltrials.gov.