Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study
Information source: Thomas Jefferson University
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Dermatomyositis
Intervention: Immunoglobulin (Hizentra) (Drug)
Phase: Phase 0
Status: Recruiting
Sponsored by: Thomas Jefferson University Overall contact:
Marinos Dalakas, MD, Phone: 215-955-4673, Email: marinos.dalakas@jefferson.edu
Summary
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.
Clinical Details
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: 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.
Secondary outcome: The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Eligibility
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. Men or woman aged >18 years
2. Diagnosis of DM based on standard criteria
3. Receiving the equivalent of at least 0. 4 g/kg IVIg every 4 weeks (IVIg group only)
4. Established response to IVIg or dependence on IVIg to maintain status established
either by symptomatic worsening of condition at the end of the inter-dose interval
for both groups or by worsening after reduction of the dose within the previous 12
months (IVIg group only)
5. IVIg regimen stable for 12 weeks while on IVIg (minor changes are permitted provided
that the dose change is 15% or less) (IVIg group only)
6. Stable dosing with steroids and/or other immunosuppressives for 12 weeks with no
changes schedule or intended.
Exclusion Criteria:
1. Pregnancy, planned pregnancy, breast feeding or unwillingness to practice
contraception
2. Severe concurrent medical conditions which would prevent treatment or assessment,
including significant hematological, renal or liver dysfunction or malignancies
3. Initiation or immunomodulatory treatment other than IVIg in the past 24 weeks or
modification of immunomodulatory treatment other than IVIg in the past 12 weeks.
4. Participation in trial of an investigational medicinal product in the past 12 weeks
5. Presence of skin infection unrelated to dermatomyositis, severe skin involvement
Presence of any other medical condition, which in the opinion of the investigator might
interfere with performance or interpretation of this study.
Locations and Contacts
Marinos Dalakas, MD, Phone: 215-955-4673, Email: marinos.dalakas@jefferson.edu
Thomas Jefferson University, Philadelphia, Pennsylvania 19107, United States; Recruiting
Marinos Dalakas, MD, Phone: 215-955-7865, Email: marinos.dalakas@jefferson.edu
Goran Rakocevic, MD, Phone: 215 955-7952, Email: goran.rakocevic@jefferson.edu
Additional Information
Starting date: November 2014
Last updated: June 23, 2015
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