Trial Outcomes & Findings for Topical Sodium Thiosulfate and Fractional Carbon Dioxide Laser in Treating Dermatomyositis Associated Calcinosis (NCT NCT01572844)

Last Updated: 2017-11-29

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Change from Visit 2 to Visit 12 (week 20)

Results posted on

2017-11-29

Participant Flow

All participants enrolled required 2 similar calcinosis lesions; one for the treatment arm and one for the no treatment arm.

Unit of analysis: lesion

Participant milestones

Participant milestones
Measure	Treatment Lesion Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Overall Study STARTED	3 3	3 3
Overall Study COMPLETED	2 2	2 2
Overall Study NOT COMPLETED	1 1	1 1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment Lesion Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Overall Study Physician Decision	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Study Participants n=3 Participants This Arm includes all participants who were enrolled in the study
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Change from Visit 2 to Visit 12 (week 20)

Outcome measures

Outcome measures
Measure	Treatment Lesion n=2 lesion Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion n=2 lesion Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Physician Calcinosis Visual Analog Scales. Subject 1	-38.0 percentage change	3.3 percentage change
Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Physician Calcinosis Visual Analog Scales. Subject 2	-53.1 percentage change	2.0 percentage change

SECONDARY outcome

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

Population: 2 subjects were enrolled and each subject has 2 lesions that were followed through the study. Each subject has one area that was marked as treatment lesion and also has an area that was used as a control (no treatment lesion). Overall, there were 2 subjects, 2 treatment lesions and 2 no treatment lesions.

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.

Outcome measures

Outcome measures
Measure	Treatment Lesion n=2 lesions Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion n=2 lesions Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
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. Subject 1	-35.8 percentage of change	-18.4 percentage of change
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. Subject 2	7.4 percentage of change	23.8 percentage of change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment Lesion n=2 lesions Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion n=2 lesions Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Patient Calcinosis Visual Analog Scales. Subject 1	104.7 Percentage of change	136.5 Percentage of change
Change in Dermatomyositis-associated Calcinosis of a Single Lesion by Assessing Its Severity as Measured by a Difference in Patient Calcinosis Visual Analog Scales. Subject 2	0 Percentage of change	1.9 Percentage of change

SECONDARY outcome

Timeframe: Change from Visit 2 to Visit 12 (week 20)

Population: No change was observed for any lesion.

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.

Outcome measures

Outcome measures
Measure	Treatment Lesion n=2 lesion Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion n=2 lesion Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Change in Size of Dermatomyositis-associated Calcinosis Area on Lesions as Measured by a Difference in Plain Film (X-ray) Studies.	0 cm Standard Deviation 0	0 cm Standard Deviation 0

SECONDARY outcome

Timeframe: Change from Visit 1 to Visit 12 (week 20)

Population: The arms/groups does not apply to this outcome measure. Lesions were not analyzed in these assessments. Overall Patient functionality and quality of life were evaluated via questionnaires, manual muscle testing, and range of motion evaluations. No changes in range of motion were noted for either subject.

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.

Outcome measures

Outcome measures
Measure	Treatment Lesion n=2 Participants Lesion A, target lesion Fractionated Carbon Dioxide (FCO2) Laser: At each treatment visit, the target area (2cm x 2cm) (+/- 1cm) of the calcinosis lesions will be treated with one pass of the Candela QuadraLase (TM) FCO2 laser. 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 Treatment Lesion Lesion B, similar area of calcinosis on the same patient, which did not receive treatment, is evaluated.
Change in Patient Functionality and/or Quality of Life. DLQI : Subject 1	-37.5 percentage of change from baseline	—
Change in Patient Functionality and/or Quality of Life. DLQI : Subject 2	57.1 percentage of change from baseline	—
Change in Patient Functionality and/or Quality of Life. HAQ : Subject 1	-44.4 percentage of change from baseline	—
Change in Patient Functionality and/or Quality of Life. HAQ : Subject 2	0 percentage of change from baseline	—
Change in Patient Functionality and/or Quality of Life. MMT8 : Subject 1	3.4 percentage of change from baseline	—
Change in Patient Functionality and/or Quality of Life. MMT8 : Subject 2	-6.7 percentage of change from baseline	—

Adverse Events

Treatment Lesion

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

No Treatment Lesion

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Alison Ehrlich, MD, Chair of Department of Dermatology

GW Medical Faculty Associates

Phone: 202-741-2625

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place