This study will find out more about calcinosis, the development of calcium deposits in various parts of the body, in people with dermatomyositis (DM) and juvenile dermatomyositis (JDM). Calcinosis can be painful and cause disabilities and other problems, so scientists want to find treatments for it. This study is examining the safety and efficacy of a medication to treat calcinosis, called sodium thiosulfate.
- Participants aged 7 older
- Meets Bohan and Peter criteria, as modified by the International Myositis Assessment and Clinical Studies Group (IMACS), for probable or definite DM or JDM
- Has extensive calcinosis, defined as calcinosis involving at least 2 extremities or the torso
- Has moderate to severe calcinosis, defined as having a calcinosis activity visual analogue scale score of greater than or equal to 3.5 cm out of 10 cm
- Is willing and able to comply with the requirements of the protocol and to undergo all testing
- Can have IV access established to receive study infusions
- Myositis disease activity is stable
- Medications for myositis are stable for at least 6 weeks prior to study entry
- Men and women of reproductive potential must agree to use a reliable form of birth control during the 62-week duration of the study
- Subjects or their legal guardian must sign a written informed consent
- Stable myositis disease activity will be defined by physician global and patient/parent global VAS that are < 4 cm, as well as creatine kinase (CK), lactate dehydrogenase (LDH), aldolase, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) that are less than or equal to 2X upper limit of normal (ULN).
- If a patient has a medication for myositis changed in this window for reasons besides their myositis activity and has returned to their baseline medication use prior to enrollment they will still be eligible.
Adam I. Schiffenbauer, M.D.
Staff Clinician, Environmental Autoimmunity Group
10 Center Dr
NIHBC 10 - CLINICAL CENTER - MAGNUSON
Bethesda, MD 20892-1627