Calcinosis, Dermatomyositis, Mixed Connective Tissue Disease (MCTD), Systemic Sclerosis (SSc)
Conditions
Keywords
calcinosis
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.
Interventions
Depending on the size of the calcinosis lesion, 1-5ml of STS (250mg/ml) will be used for injection under ultrasound guidance.
Sponsors
Robyn T. Domsic, MD, MPH
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open label, single-arm, single center US study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary endpoint: Change in size of calcinosis lesion from baseline to 12 weeks | 12 weeks | Change in size of calcinosis lesions will be measured by x-ray at baseline and then again at 12 weeks. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in pain (interference) | up to 12 weeks | We will evaluate pain impact on quality-of-life using the PROMIS® (Patient-Reported Outcomes Measurement Information System) Pain Interference Short Form 4a. This is a 4-question patient-reported outcome measure asking patients about their experience in the last week. Responses are in a likert scale format from 'not at all' to 'very much' Higher scores indicate higher degrees of interference with important aspects of life. The raw score is converted to a T-score with a mean of 50 and a standard deviation (SD) of 10. |
| Change in Pain Intensity | up to 12 weeks | We will measure pain intensity using the PROMIS® Pain Intensity 3a scale. This is a 30-item questionnaire inquiring about pain over the last week. Higher scores indicate higher pain intensity. Raw scores are converted to a T-score for analysis. The t-score is set to a mean of 50 and a standard deviation (SD) of 10. |
| Health-related Quality of Life using the EQ-5D-5L | up to 12 weeks | EuroQol(EQ)-5D-5L is a validated self-reported patient questionnaire assessing five domains of health quality: pain/discomfort, mobility, self-care, activity and anxiety/depression plus a patient global assessment visual analog scale (VAS). Patients can report no, some problems or extreme problems (scored 1-3); the VAS is scored 0-100. Iull health is a score of 1 and values below zero are regarded as a state worse than death. |
| Scleroderma Health Assessment Questionnaire (SHAQ) | up to 12 weeks | The SHAQ is composed of the standard Health Assessment Questionnaire (HAQ-DI)) plus additional visual analog scales to measure symptoms important in scleroderma: vascular, digital ulcers, lung, gastrointestinal, pain and patient global assessment. The HAQ-DI questions are scored 0-3. Higher scores indicate greater disability. |
| Change in calcinosis related symptom severity | up to 12 weeks | The Mawdsley calcinosis scale consists of Part A and Part B. Part A asks about presence of calcinosis or ulcers overlying calcinosis. If the response is present, then Part B is completed. Part B consists of 17 questions inquiring over the last 2 weeks of symptoms. Responses are 0-10 from no limitation/not all to maximum/worst possible. Higher scores indicate higher symptom severity from calcinosis. |
Countries
United States
Contacts
CONTACTMaureen M Laffoon, BS
PRINCIPAL_INVESTIGATORRobyn T Domsis, MD
University of Pittsburgh
Outcome results
None listed