Clinical trial NCT06672822: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Systemic Sclerosis (SSc)
Dermatomyositis
Mixed Connective Tissue Disease (MCTD)
Calcinosis
Keywords: calcinosis
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
Collaborators: name: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
First posted: 2024-11-04
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label, single-arm, single center US study
Primary purpose: TREATMENT

Brief summary: 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.

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

Primary outcomes:
measure: Primary endpoint: Change in size of calcinosis lesion from baseline to 12 weeks
timeFrame: 12 weeks
Secondary outcomes:
measure: Change in pain (interference)
timeFrame: up to 12 weeks
measure: Change in Pain Intensity
timeFrame: up to 12 weeks
measure: Health-related Quality of Life using the EQ-5D-5L
timeFrame: up to 12 weeks
measure: Scleroderma Health Assessment Questionnaire (SHAQ)
timeFrame: up to 12 weeks
measure: Change in calcinosis related symptom severity
timeFrame: up to 12 weeks
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: UPMC Arthritis and Autoimmunity Center
state: Pennsylvania
Study officials:
affiliation: University of Pittsburgh
name: Robyn T Domsis, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.