Intralesional Injection of STS in Treatment of Calcinosis

Официальное название

An Open Label Evaluation of Intralesional Injection of Sodium Thiosulfate in the Treatment of Cutaneous or Tendon Calcinosis in Connective Tissue Disease

Обзор

The specific objective of this study is to perform a small, open-label study to assess the safety and efficacy of intralesional, subcutaneous injection of STS on calcinosis symptoms and lesion size in systemic sclerosis (SSc), mixed connective tissue disease (MCTD) and dermatomyositis (DM) patients. Injection will be guided by ultrasound, lesion size assessed by ultrasound, and symptom burden by patient-reported outcome measures.

Подробное описание

Calcinosis is a condition in which calcium builds up in the skin and tissue under the skin. It develops in ∼30% of adult dermatomyositis,18%-49% of systemic sclerosis patients, 25-40% of patients with limited systemic sclerosis. There is no standard treatment recommended by physicians currently. Many treatments have been tested, all with limited benefits and without good evidence that any are effective. They are, based only on single cases or small numbers of patients in studies called "case series." But in some cases, these agents may be effective.

There are several case series that have shown the effectiveness of injecting a chemical compound called sodium thiosulfate (or "STS") into calcinosis lesions. There was some improvement in terms of pain control, and a decrease in size and resolution of the calcinosis. The aim of our study is to test intralesional sodium thiosulfate (STS) injection for symptoms relief of calcinosis. STS has been approved by the U.S. Food and Drug Administration for certain conditions related to childhood cancers. It has not been approved by the FDA to treat calcinosis. The specific aims of this study are to assess change in calcinosis size and quality-of-life measures.

Вмешательства

• Препарат Sodium Thiosulfate (STS)
  Depending on the size of the calcinosis lesion, 1-5ml of STS (250mg/ml) will be used for injection under ultrasound guidance.

Первичные конечные точки

• Primary endpoint: Change in size of calcinosis lesion from baseline to 12 weeks [Срок оценки: 12 weeks]

Вторичные конечные точки (5)

• Change in pain (interference) [Срок оценки: up to 12 weeks]
• Change in Pain Intensity [Срок оценки: up to 12 weeks]
• Health-related Quality of Life using the EQ-5D-5L [Срок оценки: up to 12 weeks]
• Scleroderma Health Assessment Questionnaire (SHAQ) [Срок оценки: up to 12 weeks]
• Change in calcinosis related symptom severity [Срок оценки: up to 12 weeks]

Критерии участия

Критерии включения

• Clinical diagnosis of systemic sclerosis, mixed connective tissue disease or inflammatory myopathy
• Must be over 18 years of age
• Participants must be competent to give informed consent
• Participants must have radiographic evidence (xray or ultrasound) of calcinosis.
• Participants must need symptomatic relief

Критерии исключения

• Pregnant women will be excluded

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 1 центр

• UPMC Arthritis and Autoimmunity Center — Pittsburgh

Публикации

• Boulman N, Slobodin G, Rozenbaum M, Rosner I. Calcinosis in rheumatic diseases. Semin Arthritis Rheum. 2005 Jun;34(6):805-12. doi: 10.1016/j.semarthrit.2005.01.016. PMID 15942915
• Chander S, Gordon P. Soft tissue and subcutaneous calcification in connective tissue diseases. Curr Opin Rheumatol. 2012 Mar;24(2):158-64. doi: 10.1097/BOR.0b013e32834ff5cd. PMID 22227955

Идентификаторы

NCT: NCT06672822 · STUDY24040146 · 5P50AR080612

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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