NCT04890808

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for not_applicable

Timeline
16mo left

Started May 2023

Longer than P75 for not_applicable

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress70%
May 2023Sep 2027

First Submitted

Initial submission to the registry

May 12, 2021

Completed
6 days until next milestone

First Posted

Study publicly available on registry

May 18, 2021

Completed
2 years until next milestone

Study Start

First participant enrolled

May 1, 2023

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2027

Last Updated

May 5, 2022

Status Verified

May 1, 2021

Enrollment Period

4.1 years

First QC Date

May 12, 2021

Last Update Submit

May 4, 2022

Conditions

Superficial Siderosis

Keywords

HemosiderinIron ToxicitySuperficial siderosis of the CNSInfratentorial superficial siderosisNeurodegenerative disorderSubpial

Outcome Measures

Primary Outcomes (1)

  • 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.

    36 Months

Secondary Outcomes (6)

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

    36 Months

  • Clinical Efficacy: Improved Quality of Life

    36 Months

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

    36 Months

  • Clinical Efficacy: Rate of Change in Hearing

    36 Months

  • Clinical Efficacy: Rate of Change in Mobility and Balance

    36 Months

  • +1 more secondary outcomes

Study Arms (2)

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

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\[dihydrogen (phosphate)\]

Dietary Supplement: IP6

Interventions

InosineDIETARY_SUPPLEMENT

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

Over-the-Counter Dietary Supplement Inosine
IP6DIETARY_SUPPLEMENT

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

Over-the-Counter Dietary Supplement IP6

Eligibility Criteria

Age16 Years - 77 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Diagnosed with urate urolithiasis, or recurrent urolithiasis, all unknown type
  • History of Gout
  • History of Kidney Stones
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Links

MeSH Terms

Conditions

Neurodegenerative Diseases

Interventions

Inosine

Condition Hierarchy (Ancestors)

Nervous System Diseases

Intervention Hierarchy (Ancestors)

Purine NucleosidesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Rori-Suzanne Daniel

    Superfical Siderosis Research Alliance

    STUDY DIRECTOR

Central Study Contacts

Rori-Suzanne Daniel

CONTACT

9035594123administrator@superficialsiderosis.org

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
SUPPORTIVE CARE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 12, 2021

First Posted

May 18, 2021

Study Start

May 1, 2023

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

September 1, 2027

Last Updated

May 5, 2022

Record last verified: 2021-05

Data Sharing

IPD Sharing
Will not share