CalcinosisPhase 2NCT01572844
Topical Sodium Thiosulfate and Fractional Carbon Dioxide Laser in Treating Dermatomyositis Associated Calcinosis
A Phase 2 study testing Fractionated Carbon Dioxide (FCO2) Laser and Sodium thiosulfate in people with Calcinosis, run by Alison Ehrlich at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Calcinosis
- What is being tested?
- Fractionated Carbon Dioxide (FCO2) Laser and Sodium thiosulfate
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to Jun 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
The George Washington University Medical Faculty Associates Departments of Dermatology and Rheumatology
Washington D.C., District of Columbia, 20037
About this study
Official title: 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.
Read the 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.
- Conditions
- Calcinosis
- Being tested
- Fractionated Carbon Dioxide (FCO2) Laser, Sodium thiosulfate
- Keywords
- Dermatomyositis, Superficial calcinosis, Fractional carbon dioxide laser, Sodium thiosulfate
- Medical subject headings
- Calcinosis, Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fractionated Carbon Dioxide (FCO2) LaserDevice
Also called 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.
Sodium thiosulfateDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 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.
Measured: 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 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.
Measured: 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 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.
Measured: Change from Visit 2 to Visit 12 (week 20)
Change in Patient Functionality and/or Quality of Life.
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.
Measured: Change from Visit 1 to Visit 12 (week 20)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2012 (estimated)
- Study startAug 2012
- Primary completionJun 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for CalcinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alison Ehrlich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Alison Ehrlich, George Washington University
- Investigators
- Alison Ehrlich, MD, Principal investigator, George Washington UniversityGary Simon, MD, Principal investigator, George Washington UniversityJames Katz, MD, Study chair, George Washington UniversityGulnara Mamayrova, MD, Study chair, George Washington UniversityLaura Roosa, Study chair, George Washington UniversityAndrea Morris, MD, Study chair, George Washington University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01572844
- Sponsor's protocol ID
- 121131
- Status last verified
- Nov 2017
- First submitted
- 4 Apr 2012
- Last updated
- 29 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Novel Drug Delivery of Sodium Thiosulfate for Calcinosis Associated With Adult and Juvenile Dermatomyositis
- ClinicalTrials.gov ID
- NCT01572844
- Acronym / sponsor ID
- 121131
- Conditions
- Calcinosis
- Keywords
- Dermatomyositis, Superficial calcinosis, Fractional carbon dioxide laser, Sodium thiosulfate
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Alison EhrlichUniversity, hospital or other
- Study start
- Aug 2012
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 6 Apr 2012 (estimated)
- Results
- Posted 29 Nov 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment lesion | Experimental | Device: Fractionated Carbon Dioxide (FCO2) Laser, Drug: Sodium thiosulfateLesion 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. |
| No Treatment Lesion | No intervention | –Lesion B, similar area of calcinosis on the same patient, which did not receive treatment is evaluated. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fractionated Carbon Dioxide (FCO2) LaserAlso: 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. | Device | Treatment lesion |
| Sodium thiosulfateFollowing 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. | Drug | Treatment lesion |
Primary outcome
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.
4 secondary outcomes
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 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 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 Patient Functionality and/or Quality of Life.
Time frame: Change from Visit 1 to Visit 12 (week 20)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Calcinosis
Active, not recruiting
Shockwave™ vs Surgical Endarterectomy for Calcified Severe Common Femoral Artery Stenosis: Comparison of Efficacy, Safety and Long-Term Outcomes
Common Femoral Artery Stenosis, Calcification; Heart
RecruitingObservational study
Quantification of Calcinosis Cutis Disease Burden Using Computed Tomography Images
Systemic Sclerosis (SSc), Calcinosis Cutis
Not yet recruitingPhase 2
REducing Systemic Inflammation With LOw-dose Colchicine to ImproVE Cardiovascular Calcification in Chronic Kidney Disease: A Pilot Randomized Open-Label Trial
Chronic Kidney Disease Mineral and Bone Disorder, Cognitive Function
Enrolling by invitationObservational study
Advanced Ultrasound Signal Processing of Suspicious Breast Images
Breast Cancer, Breast Cyst
RecruitingObservational study
A Natural History Study of Bone and Mineral Disorders
Tumor Induced Osteomalcia, Osteomalacia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alison Ehrlich, MD principal investigator
George Washington University
Gary Simon, MD principal investigator
George Washington University
James Katz, MD study chair
George Washington University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fractionated Carbon Dioxide (FCO2) Laser and Sodium thiosulfate in people with Calcinosis, run by Alison Ehrlich at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Nov 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2017; AzTrials last refreshed its copy on 7 Nov 2026.