Intralesional Injection of STS in Treatment of Calcinosis

Official title

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

Overview

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.

Detailed description

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.

Interventions

• Drug 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.

Primary outcome measures

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

Secondary outcome measures (5)

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

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• Pregnant women will be excluded

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 1 center

• UPMC Arthritis and Autoimmunity Center — Pittsburgh

Publications

• 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

Identifiers

NCT: NCT06672822 · STUDY24040146 · 5P50AR080612

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗