Diffuse Scleroderma, Systemic Sclerosis
Conditions
Keywords
Systemic Sclerosis, Diffuse Scleroderma, SSc
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.
Interventions
BIOLOGICALPrivigen
Subjects will receive 2 g/kg/mo of Privigen® for 6 months if randomized to the treatment group.
Sponsors
CSL Behring
Georgetown University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To study the effects of IVIG on the skin in patients with scleroderma | At the 4 month visit | The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To determine any toxicity of IVIG in scleroderma | At 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. |
| To evaluate the effects of IVIG on pulmonary function | At the 4 month visit | Pulmonary Function Tests (PFTs) will be performed to determine these changes. |
| Muscle, joint, and inflammatory parameters | At the 4 month visit | Done by performing a physical exam |
Other
| Measure | Time frame | Description |
|---|---|---|
| To study the effects of IVIG on the skin in patients with scleroderma | At the 6 month visit | The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin |
| Muscle, joint, and inflammatory parameters | At the 6 month visit | A physical exam will be performed to determine these changes |
| To determine any toxicity of IVIG in scleroderma | At the 6 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. |
| To evaluate the effects of IVIG on pulmonary function | At the 6 month visit | Pulmonary Function Tests (PFTs) will be performed to determine these changes. |
| To study the biologic effects of IVIG on gene arrays in skin biopsies | At the 6 month visit | A skin biopsy will be taken at the Baseline and Month 6 visit to determine the biological effects of IVIG on the skin. |
Countries
United States
Outcome results
None listed