Clinical trial NCT01785056: IVIG Treatment in Systemic Sclerosis
Official title: A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety and Efficacy of Intravenous Immunoglobulin Treatment in Patients With Systemic Sclerosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Systemic Sclerosis
Diffuse Scleroderma
Keywords: Systemic Sclerosis
Diffuse Scleroderma
SSc
Lead sponsor: Georgetown University
Sponsor class: OTHER
Collaborators: name: CSL Behring
First posted: 2013-02-06
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: To study the effects of IVIG on the skin in patients with scleroderma
timeFrame: At the 4 month visit
Secondary outcomes:
measure: To determine any toxicity of IVIG in scleroderma
timeFrame: At the 4 month visit
measure: To evaluate the effects of IVIG on pulmonary function
timeFrame: At the 4 month visit
measure: Muscle, joint, and inflammatory parameters
timeFrame: At the 4 month visit
Other outcomes:
measure: To study the effects of IVIG on the skin in patients with scleroderma
timeFrame: At the 6 month visit
measure: To determine any toxicity of IVIG in scleroderma
timeFrame: At the 6 month visit
measure: To evaluate the effects of IVIG on pulmonary function
timeFrame: At the 6 month visit
measure: To study the biologic effects of IVIG on gene arrays in skin biopsies
timeFrame: At the 6 month visit
measure: Muscle, joint, and inflammatory parameters
timeFrame: At the 6 month visit
Locations:
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
Study officials:
affiliation: Georgetown University Hospital
name: Virginia D Steen, MD
role: PRINCIPAL_INVESTIGATOR
References: