The purpose of the study is to evaluate the effectiveness and safety of human immunoglobulin SCIg in the form of Hizentra (Immune globulin Subcutaneous) in patients with Dermatomyositis. Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body) in patients with primary immunodeficiency. At present, patients with steroid resistant dermatomyositis can only be treated with IVIg (The healthy antibodies in IVIG can block the damaging antibodies that attack muscle and skin in dermatomyositis) treatment. An evaluation can then be made to see if SCIg is a suitable replacement and exerts immunomodulatory effect on complement antibodies.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
2
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
Time frame: The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.
The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Secondary outcome measures will be a) patient preference, based on the number of participants who prefer SCIg to IVIg (a home-made questionnaire will be utilized to capture preference in the most unbiased way; b) average change in Quality of life scores between week 12 and 28; c) effect on the complement consumption in serum; d) Immunological parameters on the repeated skin biopsies; and e) adverse events as reported at each visit. Accordingly, information will be obtained on the superiority of SCIg vs. IVIg
Time frame: The main secondary outcome is the preference of the participant for SCIg compared with IVIg
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