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

NCT01572844 | Completed Phase 2 Results DMC

• 1 other identifier: 121131
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Calcinosis

Keywords

Dermatomyositis; Superficial calcinosis; Fractional carbon dioxide laser; Sodium thiosulfate

Outcome Measures

Primary Outcomes (1)

• Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Physician Calcinosis Visual Analog Scales.

  Change in dermatomyositis-associated calcinosis of a single lesion by assessing its severity as measured by a difference in Physician Calcinosis Visual Analog Scales. The Visual Analog Scale range is from zero (0) to ten (10). Zero (0) being no evidence of disease activity and ten (10) being extremely active or severe disease activity. A negative percent change indicates improvement in the lesion.

  Change from Visit 2 to Visit 12 (week 20)

Secondary Outcomes (4)

• Change 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.

  Change from Baseline (Visit 1) at Final Assessment (Visit 12, week 20).

• Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Patient Calcinosis Visual Analog Scales.

  Change from Visit 2 to Visit 12 (week 20)

• Change in Size of Dermatomyositis-associated Calcinosis Area on Lesions as Measured by a Difference in Plain Film (X-ray) Studies.

  Change from Visit 2 to Visit 12 (week 20)

• Change in Patient Functionality and/or Quality of Life.

  Change from Visit 1 to Visit 12 (week 20)

Study Arms (2)

Treatment lesion

EXPERIMENTAL

Lesion A, target lesion, 2cm x 2cm (+/- 1cm) treated with 1 pass Fractionated Carbon Dioxide (FCO2) Laser followed by application of 4 ml of 5% topical sodium thiosulfate solution (STS), 8 to 10 treatments over 6 months.

Device: Fractionated Carbon Dioxide (FCO2) Laser; Drug: Sodium thiosulfate

No Treatment Lesion

NO INTERVENTION

Lesion B, similar area of calcinosis on the same patient, which did not receive treatment is evaluated.

Interventions

Fractionated Carbon Dioxide (FCO2) Laser DEVICE

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.

Also known as: Candela QuadraLase (TM)

Treatment lesion

Sodium thiosulfate DRUG

Following treatment with FCO2 laser, 4 ml of 5% Sodium Thiosulfate solution will be applied to the treatment site only. Subjects will receive a total of 8-10 treatments over a 6 month period.

Treatment lesion

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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, District of Columbia clinic over the 20 week period.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alison Ehrlich: lead

Study Sites (1)

The George Washington University Medical Faculty Associates Departments of Dermatology and Rheumatology

Washington D.C., District of Columbia, 20037, United States

Location

MeSH Terms

Conditions

Calcinosis; Dermatomyositis

Interventions

Lasers; sodium thiosulfate

Condition Hierarchy (Ancestors)

Calcium Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Optical Devices; Equipment and Supplies; Radiation Equipment and Supplies

Results Point of Contact

• Title: Alison Ehrlich, MD, Chair of Department of Dermatology
• Organization: GW Medical Faculty Associates

aehrlich@mfa.gwu.edu

202-741-2625

Study Officials

• Alison Ehrlich, MD

  George Washington University

  PRINCIPAL INVESTIGATOR

• Gary Simon, MD

  George Washington University

  PRINCIPAL INVESTIGATOR

• 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

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 4, 2012
• First Posted: April 6, 2012
• Study Start: August 1, 2012
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: November 29, 2017
• Results First Posted: November 29, 2017

Record last verified: 2017-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)