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IVIG Treatment in Systemic Sclerosis

Not Applicable
Conditions
Diffuse Scleroderma
Systemic Sclerosis
Interventions
Biological: Privigen
Registration Number
NCT01785056
Lead Sponsor
Georgetown University
Brief Summary

The purpose of this research is to study the effects of Privigen (intravenous immunoglobulin) on the skin in patients with scleroderma. Approximately 24 subjects will take part in this investigator-initiated study at Georgetown University Hospital and Johns Hopkins Hospital. This study will last for one year (12 months). This research is being done because systemic sclerosis can cause severe, progressive organ involvement. The investigators hope this study treatment will improve the outcomes in this disease, including skin, muscle, joint, gastrointestinal, and lung involvement.

Detailed Description

The investigators propose a double-blind controlled trial of 24 patients with Privigen® (Immune Globulin Intravenous (Human), 10% liquid treatment) with 3:1 randomization. Subjects with scleroderma will be given 2 g/kg/mo of Privigen® or placebo (Albuminar®-5) for 6 months. Scleroderma is generally a progressive disease, and while the skin does improve with time in some patients, others have progressive disease in spite of aggressive treatment. Also, spontaneous improvement in other organ systems is even less likely. Patients entered into the trial will have failed to respond to standard of care treatment over the past 4 months. Thus, the investigators feel that any actual improvement observed can be attributed to the IVIG treatment. Since this is a pilot study, future larger controlled trials will be necessary to clearly demonstrate the effectiveness, but the investigators are hoping that this study will give us signals that will guide the future clinical trial.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
14
Inclusion Criteria
  • Diffuse systemic sclerosis with active skin involvement as defined as a mRSS >12, with no improvement or worsening in the previous 4 months in spite of treatment with methotrexate, cellcept, imuran, or anti-TNF agent;
  • 18 years of age or older;
  • Disease duration of less than 5 years from the first non-Raynaud's symptom of scleroderma.
Exclusion Criteria
  • Use of more than 10 mg of prednisone, or any dose of cytoxan, d-penicillamine, or rituximab in the last 3 months;
  • History of deep vein thrombosis (DVT), stroke, or other thromboembolic phenomenon;
  • History of anaphylaxis or other serious reaction to human blood or blood products.
  • Absolute IgA deficiency
  • A prior receipt of IVIg treatment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PrivigenPrivigenPrivigen is a ready-to-use, sterile, 10% protein liquid preparation of polyvalent human immunoglobulin G (IgG) for intravenous administration. Subjects will be given 2 g/kg/month of IVIGfor 6 months. Each dose will be split into 2 to 4 infusions on consecutive days to achieve the total monthly dose.
Placebo (Albuminar-5)PrivigenAlbuminar-5 is a sterile solution of albumin obtained from large pools of adult human venous plasma and used as the placebo in this study. Albuminar-5 will be administered by the intravenous route and each dose will be split into 2 to 4 infusions on consecutive days to achieve the total monthly dose.
Primary Outcome Measures
NameTimeMethod
To study the effects of IVIG on the skin in patients with sclerodermaAt the 4 month visit

The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin

Secondary Outcome Measures
NameTimeMethod
To evaluate the effects of IVIG on pulmonary functionAt the 4 month visit

Pulmonary Function Tests (PFTs) will be performed to determine these changes.

To determine any toxicity of IVIG in sclerodermaAt the 4 month visit

Laboratory assessments (Complete Blood Count (CBC) including liver and electrolytes with differentiation, Complete Metabolic Profile (CMP) including creatinine, and Coombs test) will be performed monthly to monitor toxicity.

Muscle, joint, and inflammatory parametersAt the 4 month visit

Done by performing a physical exam

Trial Locations

Locations (2)

Georgetown University Hospital

🇺🇸

Washington, District of Columbia, United States

Johns Hopkins Hospital

🇺🇸

Baltimore, Maryland, United States

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