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

November 21, 2017 updated by: Alison Ehrlich

Novel Drug Delivery of Sodium Thiosulfate for Calcinosis Associated With Adult and Juvenile Dermatomyositis

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
3

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • The George Washington University Medical Faculty Associates Departments of Dermatology and Rheumatology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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, District of Columbia 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Treatment lesion
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
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 Names:
  • Candela QuadraLase (TM)
Drug: Sodium thiosulfate
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.
No Intervention: No Treatment Lesion
Lesion B, similar area of calcinosis on the same patient, which did not receive treatment is evaluated.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change 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: Change from Visit 2 to Visit 12 (week 20)
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
Time Frame: Change from Baseline (Visit 1) at Final Assessment (Visit 12, week 20).
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. The range of a durometer is from 0 to 100. The higher the durometer reading, the harder the lesion. Improvement in the lesion hardness would result in a negative change over time.
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.
Time Frame: Change from Visit 2 to 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. The scale ranges 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 change would indicate improvement in the disease activity. A positive change would indicate worsening of disease activity.
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.
Time Frame: Change from Visit 2 to Visit 12 (week 20)
Change in dermatomyositis-associated calcinosis as measured by a difference in centimeters in plain film (x-ray) studies. X rays were compared between baseline and final assessment for any changes.
Change from Visit 2 to Visit 12 (week 20)
Change in Patient Functionality and/or Quality of Life.
Time Frame: Change from Visit 1 to Visit 12 (week 20)
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. DLQI index scores range from 0 to 30. The higher the score, the more quality of life is impaired.Change in scores from baseline to final assessment yielding a negative percent change indicate an improvement in DLQI. HAQ scales range from 0 to 3. The higher the score, the greater the disability. Change in scores from baseline to final assessment yielding a negative percent change indicate an improvement in the HAQ. MMT8 testing results in a score between 0 and 150. The higher the score, the more normal the function of the muscle. A positive percent change would indicate an improvement in MMT8 testing results.
Change from Visit 1 to Visit 12 (week 20)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alison Ehrlich

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 1, 2012

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 4, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 5, 2012

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 6, 2012

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 29, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 21, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 121131

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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