Superficial SiderosisTAS-SS21NCT04890808
Therapeutic Antioxidant Supplementation
A study testing Inosine and IP6 in people with Superficial Siderosis, run by Superficial Siderosis Research Alliance Inc..
Trial 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… Read more about this study
- Who is it for?
- Ages 16 to 77 years
- Conditions the study is for
- Superficial Siderosis
- What is being tested?
- Inosine and IP6
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- May 2023to Sep 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 77 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
Show all 10Show fewer
- Known or suspected active bleed into the CNS
- Currently undergoing deferiprone chelation therapy
- Plan to begin deferiprone chelation therapy within three years
- -
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read the 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).
- Conditions
- Superficial Siderosis
- Being tested
- Inosine, IP6
- Keywords
- Hemosiderin, Iron Toxicity, Superficial siderosis of the CNS, Infratentorial superficial siderosis, Neurodegenerative disorder, Subpial
- Medical subject headings
- Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InosineDietary supplement
Patients will be dosed with two 500 mg capsules of Inosine twice daily for 36 months
IP6Dietary supplement
Patients will be dosed with two 500 mg capsules of IP6 twice daily for 36 months
| Study group | Role | What they receive |
|---|---|---|
| Over-the-Counter Dietary Supplement Inosine | Experimental | capsules containing 500 mg of inosine Two capsules Twice Daily Other Name: hypoxanthine 9-β-D-ribofuranoside 9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-hydroxymethyl) oxolan-2-yl]-1H-purin-6-one Receives: Dietary Supplement: Inosine |
| Over-the-Counter Dietary Supplement IP6 | Experimental | 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)] Receives: Dietary Supplement: IP6 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Slow the Rate of Clinical Decline
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.
Measured: 36 Months
Secondary outcomes (6)
Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability}
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.
Measured: 36 Months
Clinical Efficacy: Improved Quality of Life
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.
Measured: 36 Months
Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA)
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.
Measured: 36 Months
Clinical Efficacy: Rate of Change in Hearing
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.
Measured: 36 Months
Clinical Efficacy: Rate of Change in Mobility and Balance
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.
Measured: 36 Months
Clinical Efficacy: Hemosiderin Reduction (IP6 Arm)
Visible hemosiderin deposition reduction on MRI series from baseline visit to end-of-study. MRIs will be objectively analyzed for changes in iron deposition.
Measured: 36 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2021
- Study startMay 2023 (estimated)
- Primary completionJun 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Superficial SiderosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Superficial Siderosis Research Alliance Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Rori-Suzanne Daniel, Study director, Superfical Siderosis Research Alliance
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04890808
- Sponsor's protocol ID
- SSRA0921
- Acronym
- TAS-SS21
- Status last verified
- May 2021
- First submitted
- 12 May 2021
- Last updated
- 5 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Antioxidant Supplementation for Enhancement of Neural Protection From Free-iron Toxicity in Superficial Siderosis
- ClinicalTrials.gov ID
- NCT04890808
- Acronym / sponsor ID
- TAS-SS21 / SSRA0921
- Conditions
- Superficial Siderosis
- Keywords
- Hemosiderin, Iron Toxicity, Superficial siderosis of the CNS, Infratentorial superficial siderosis, Neurodegenerative disorder, Subpial
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Superficial Siderosis Research Alliance Inc.University, hospital or other
- Study start
- May 2023 (estimated)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 18 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Over-the-Counter Dietary Supplement Inosine | Experimental | Dietary Supplement: Inosinecapsules containing 500 mg of inosine Two capsules Twice Daily Other Name: hypoxanthine 9-β-D-ribofuranoside 9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-hydroxymethyl) oxolan-2-yl]-1H-purin-6-one |
| Over-the-Counter Dietary Supplement IP6 | Experimental | Dietary Supplement: IP6capsules 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)] |
Who may qualify
- Ages
- 16 to 77 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (10 listed)
- Diagnosed with urate urolithiasis, or recurrent urolithiasis, all unknown type
- History of Gout
- History of Kidney Stones
- P6 Arm Exclusion
- Long-term anticoagulant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InosinePatients will be dosed with two 500 mg capsules of Inosine twice daily for 36 months | Dietary supplement | Over-the-Counter Dietary Supplement Inosine |
| IP6Patients will be dosed with two 500 mg capsules of IP6 twice daily for 36 months | Dietary supplement | Over-the-Counter Dietary Supplement IP6 |
Primary outcome
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.
6 secondary outcomes
Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability}
Time frame: 36 Months
Clinical Efficacy: Improved Quality of Life
Time frame: 36 Months
Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA)
Time frame: 36 Months
Clinical Efficacy: Rate of Change in Hearing
Time frame: 36 Months
Clinical Efficacy: Rate of Change in Mobility and Balance
Time frame: 36 Months
Clinical Efficacy: Hemosiderin Reduction (IP6 Arm)
Time frame: 36 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2022.
- Superficial Siderosis Patient RegistryLink provided by the sponsor.
- Superficial Siderosis research AllianceLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Rori-Suzanne Daniel study director
Superfical Siderosis Research Alliance
Questions about this trial
What is this clinical trial studying?
A study testing Inosine and IP6 in people with Superficial Siderosis, run by Superficial Siderosis Research Alliance Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 5 May 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rori-Suzanne Daniel (9035594123, administrator@superficialsiderosis.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Slow the Rate of Clinical Decline (time frame: 36 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2022.