Topical Sodium Thiosulfate and Fractional Carbon Dioxide Laser in Treating Dermatomyositis Associated Calcinosis
Dermatomyositis is an inflammatory muscle disorder that is often associated with many skin findings. One of the skin findings seen in up to 50% of individuals with juvenile dermatomyositis, an early onset form of this condition, and up to 20-30% of adult dermatomyositis patients who have not responded to treatment, is calcinosis, or deposits of calcium within the skin and muscle tissue. In addition to being cosmetically unappealing, involvement of deeper tissues with calcinosis may lead to contractures, or shortening of muscles, which may have a significant impact on functioning and quality of life. Unfortunately, there is no known effective treatment of dermatomyositis associated calcinosis. However, recent reports have shown that a medication called sodium thiosulfate has been effective in treating individuals with calciphylaxis, a condition where calcium is deposited within blood vessels, and with tumoral calcinosis, a genetic form of calcification, when receiving this medication by vein. In addition, recent advances in laser technology have led to the development of methods that may allow topical medications to penetrate deeper layers of the skin. The investigators have designed a pilot study to evaluate the use of topical sodium thiosulfate solution in treating superficial calcinosis in individuals with juvenile and adult dermatomyositis. The investigators will use laser to assist in the delivery of this medication to areas of calcinosis.
Dermatomyositis Associated Superficial Calcinosis
Device: Fractional Carbon Dioxide (FCO2) Laser (Candela QuadraLase (TM))
Drug: Sodium thiosulfate
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Novel Drug Delivery of Sodium Thiosulfate for Calcinosis Associated With Adult and Juvenile Dermatomyositis|
- Improvement in dermatomyositis-associated calcinosis of a single lesion by assessing its severity as measured by a difference in Physician Calcinosis Visual Analog Scales. [ Time Frame: Weeks 0,4,8,12,16,20 ] [ Designated as safety issue: No ]
- Improvement in dermatomyositis-associated calcinosis of a single lesion by assessing its hardness as measured by a difference in durometer (Rex durometer Model 1600, Type OO) measurements. [ Time Frame: Weeks 0,4,8,12,16,20 ] [ Designated as safety issue: No ]
- Improvement in dermatomyositis-associated calcinosis of a single lesion by assessing its severity as measured by a difference in Patient Calcinosis Visual Analog Scales. [ Time Frame: Weeks 0,4,8,12,16,20 ] [ Designated as safety issue: No ]
- Improvement in dermatomyositis-associated calcinosis as measured by a difference in plain film (x-ray) studies. [ Time Frame: At baseline and study completion (week 20). ] [ Designated as safety issue: No ]
- Improvement in patient functionality and/or quality of life. [ Time Frame: At baseline, week 8, and week 20. ] [ Designated as safety issue: No ]This will be assessed through the use of the Dermatology Life Quality Index (DLQI), Health Assessment Questionnaire (HAQ), Childhood Health Assessment Questionnaire (CHAQ), Manual Muscle Testing (MMT8), and range of motion evaluations.
|Study Start Date:||August 2012|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||August 2015 (Final data collection date for primary outcome measure)|
Experimental: Dermatomyositis Associated Calcinosis
Individuals with a diagnosis of dermatomyositis associated superficial calcinosis and who meet the inclusion and exclusion criteria. This group will consist of 5 individuals.
Device: Fractional Carbon Dioxide (FCO2) Laser (Candela QuadraLase (TM))
At each treatment visit, a 2 X 2 cm (+/- 1 cm) area of the target calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. Patients will receive a total of 8-10 treatments over a 6 month period.
Other Name: Candela QuadraLase (TM) Fractional Carbon Dioxide LaserDrug: Sodium thiosulfate
Following treatment with FCO2 laser, 4 ml of 5% STS solution will be applied to the treatment site only. Subjects will receive a total of 8-10 treatments over a 6 month period.
Five individuals who meet the eligibility criteria will take part in this study. They will have a variety of assessments performed throughout the treatment period in order to evaluate both dermatomyositis and calcinosis severity and their potential response to fractional carbon dioxide and sodium thiosulfate treatment. A medical history will be taken and baseline assessments will be performed during the screening period. Serum creatinine kinase levels will be determined on this visit and repeated at the end of the study (week 20); these levels will be one measure of monitoring disease activity during the study. One calcinosis lesion will be treated, assessed, and followed. If a second calcinosis lesion is present, it will act as a control (not treated). Two weeks prior to the first treatment session, an optional (not required) skin biopsy of the target (treated) calcinosis lesion will be offered to the the first 3 patients ≥ 18 years of age to determine optimal fractional carbon dioxide laser settings that will be used for treatment. Area and durometer (a device that measures hardness) measurements and photographs of the calcinosis lesions will be performed at weeks 0,4,8,12,16,and 20. One x-ray of the control and one x-ray of the target calcinosis lesion will also be performed during the screening period and at week 20. Assessment of muscle strength, physical functioning, endurance, and range of motion, as well as myositis activity outside of the muscles will be performed during the screening period and at weeks 8 and 20. Myositis damage assessment will be performed at the screening period and at week 20. Questionnaires to assess physical functioning pertaining to activities of daily living and quality of life, as well as the quality of life related to skin disease and the calcinosis lesions will be completed during the screening period and at weeks 8 and 20. Treatment of the target calcinosis lesion with fractional carbon dioxide laser and topical sodium thiosulfate will occur on weeks 0,2,4,6,8,10,12,14,16,and 18. Each patient will receive a total of 8-10 treatments over a 6 month period. Assessments for any side effects from the treatment will be performed prior to each treatment session on weeks 0,2,4,6,8,10,12,14,16,18, and 20.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01572844
|Contact: Laura Roosafirstname.lastname@example.org|
|Contact: Nicole Swansonemail@example.com|
|United States, District of Columbia|
|The George Washington University Medical Faculty Associates Departments of Dermatology and Rheumatology||Recruiting|
|Washington, District of Columbia, United States, 20037|
|Contact: Alison Ehrlich, MD 202-741-2600|
|Principal Investigator: Alison Ehrlich, MD|
|Principal Investigator: Gary Simon, MD|
|Sub-Investigator: James Katz, MD|
|Sub-Investigator: Sabrina Newman, MD|
|Sub-Investigator: Gulnara Mamyrova, MD|
|Sub-Investigator: Amir Zahir, MD|
|Principal Investigator:||Alison Ehrlich, MD||George Washington University|
|Principal Investigator:||Gary Simon, MD||George Washington University|
|Study Chair:||James Katz, MD||George Washington University|
|Study Chair:||Gulnara Mamayrova, MD||George Washington University|
|Study Chair:||Laura Roosa||George Washington University|
|Study Chair:||Andrea Morris, MD||George Washington University|