Therapeutic Antioxidant Supplementation

Официальное название

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

Обзор

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.

Подробное описание

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).

Вмешательства

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

Первичные конечные точки

• Slow the Rate of Clinical Decline [Срок оценки: 36 Months]

Вторичные конечные точки (6)

• Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability} [Срок оценки: 36 Months]
• Clinical Efficacy: Improved Quality of Life [Срок оценки: 36 Months]
• Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA) [Срок оценки: 36 Months]
• Clinical Efficacy: Rate of Change in Hearing [Срок оценки: 36 Months]
• Clinical Efficacy: Rate of Change in Mobility and Balance [Срок оценки: 36 Months]
• Clinical Efficacy: Hemosiderin Reduction (IP6 Arm) [Срок оценки: 36 Months]

Критерии участия

Критерии включения

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

Критерии исключения

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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Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Поддерживающая терапия

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT04890808 · SSRA0921

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗