Topical Sodium Thiosulfate and Fractional Carbon Dioxide Laser in Treating Dermatomyositis Associated Calcinosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by George Washington University
Sponsor:
Information provided by (Responsible Party):
George Washington University
ClinicalTrials.gov Identifier:
NCT01572844
First received: April 4, 2012
Last updated: April 25, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Dermatomyositis Associated Superficial Calcinosis
Device: Fractional Carbon Dioxide (FCO2) Laser (Candela QuadraLase (TM))
Drug: Sodium thiosulfate
Phase 2

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by George Washington University:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 5
Study Start Date: August 2012
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Laser
Drug: 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.

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Individuals of both sexes and of all ethnicities who wish to participate in the study and who have signed a written informed consent form to participate.
  • Subjects must be between the ages of 18-65 years.
  • Subjects must have a diagnosis of adult or juvenile dermatomyositis.
  • All patient must be on stable therapy for their condition. Overall disease activity must be considered mild or in remission.
  • Patients must have a history of failing at least one therapy for calcinosis associated with dermatomyositis.
  • Patients must have at least one localized area of superficial calcinosis with easily identifiable landmarks. Whenever possible, subjects will have a second localized area of superficial calcinosis that can serve as a control lesion for repeated assessment.
  • The calcinosis lesion being treated and the control calcinosis lesion must be stable (not increasing in size) based on the patient's history.
  • Patients must be able to attend all treatment sessions and assessment visits at our Washington, DC clinic over the 20 week period.

Exclusion Criteria:

  • Unstable dermatomyositis, or moderate or severely active juvenile dermatomyositis.
  • Serum creatine kinase greater than or equal to three times the upper limit of normal.
  • Inability to make study visits or anticipated poor compliance.
  • Active infections, including a history of recurrent superinfection or cellulitis at the sites of calcinosis (> 1 prior episode).
  • Pregnant females or nursing mothers.
  • Life threatening illness that would interfere with the patient's ability to complete the study.
  • Known or suspected history of drug or alcohol abuse within the past 6 months.
  • Participation in another clinical experimental therapeutic study within 30 days of screening visit.
  • History of severe illness or any other condition that would make the patient unsuitable for the study.
  • History of hepatitis B, hepatitis C, HIV, cancer-associated myositis, or an underlying malignancy.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01572844

Contacts
Contact: Sirlekar Bullocks 202-741-6141 sbullocks@mfa.gwu.edu

Locations
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         
Sponsors and Collaborators
George Washington University
Investigators
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 Mamyrova, MD George Washington University
Study Chair: Amir Zahir, MD George Washington University
Study Chair: Jack Short, MD George Washington University
Study Chair: Andrea Morris, MD George Washington University
  More Information

No publications provided

Responsible Party: George Washington University
ClinicalTrials.gov Identifier: NCT01572844     History of Changes
Other Study ID Numbers: 121131
Study First Received: April 4, 2012
Last Updated: April 25, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by George Washington University:
Dermatomyositis
Superficial calcinosis
Fractional carbon dioxide laser
Sodium thiosulfate

Additional relevant MeSH terms:
Polymyositis
Calcinosis
Dermatomyositis
Calcium Metabolism Disorders
Metabolic Diseases
Myositis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases
Sodium thiosulfate
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Chelating Agents
Antidotes

ClinicalTrials.gov processed this record on July 29, 2014