IVIG Treatment in Systemic Sclerosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by Georgetown University
Sponsor:
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Georgetown University
ClinicalTrials.gov Identifier:
NCT01785056
First received: January 28, 2013
Last updated: June 26, 2014
Last verified: December 2013
  Purpose

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.


Condition Intervention
Systemic Sclerosis
Diffuse Scleroderma
Biological: Privigen

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety and Efficacy of Intravenous Immunoglobulin Treatment in Patients With Systemic Sclerosis

Resource links provided by NLM:


Further study details as provided by Georgetown University:

Primary Outcome Measures:
  • To study the effects of IVIG on the skin in patients with scleroderma [ Time Frame: At the 4 month visit ] [ Designated as safety issue: No ]
    The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin


Secondary Outcome Measures:
  • To determine any toxicity of IVIG in scleroderma [ Time Frame: At the 4 month visit ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: At the 4 month visit ] [ Designated as safety issue: No ]
    Pulmonary Function Tests (PFTs) will be performed to determine these changes.

  • Muscle, joint, and inflammatory parameters [ Time Frame: At the 4 month visit ] [ Designated as safety issue: No ]
    Done by performing a physical exam


Other Outcome Measures:
  • To study the effects of IVIG on the skin in patients with scleroderma [ Time Frame: At the 6 month visit ] [ Designated as safety issue: No ]
    The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin

  • To determine any toxicity of IVIG in scleroderma [ Time Frame: At the 6 month visit ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: At the 6 month visit ] [ Designated as safety issue: No ]
    Pulmonary Function Tests (PFTs) will be performed to determine these changes.

  • To study the biologic effects of IVIG on gene arrays in skin biopsies [ Time Frame: At the 6 month visit ] [ Designated as safety issue: No ]
    A skin biopsy will be taken at the Baseline and Month 6 visit to determine the biological effects of IVIG on the skin.

  • Muscle, joint, and inflammatory parameters [ Time Frame: At the 6 month visit ] [ Designated as safety issue: No ]
    A physical exam will be performed to determine these changes


Estimated Enrollment: 24
Study Start Date: April 2013
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Privigen
Privigen 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 IVIG (or placebo) for 6 months. Each dose will be split into 2 to 4 infusions on consecutive days to achieve the total monthly dose.
Biological: Privigen
Subjects will receive 2 g/kg/mo of Privigen® for 6 months if randomized to the treatment group.
Placebo Comparator: Placebo (Albuminar-5)
Albuminar-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.

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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diffuse systemic sclerosis with active skin involvement as defined as a mRSS >16, 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 6 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01785056

Contacts
Contact: Virginia D Steen, MD 202-444-6200
Contact: Maia Zulmatashvili 202-444-6210 mz381@georgetown.edu

Locations
United States, District of Columbia
Georgetown University Hospital Recruiting
Washington, District of Columbia, United States, 20007
Contact: Virginia D Steen, MD    202-444-6200      
Contact: Maia Zulmatashvili    202-444-6210    mz381@georgetown.edu   
Principal Investigator: Virginia D Steen, MD         
United States, Maryland
Johns Hopkins Hospital Not yet recruiting
Baltimore, Maryland, United States, 21224
Contact: Laura Hummers, MD    410-550-2400      
Contact: Gwendolyn Leatherman    410-550-8582    gleathe@jhmi.edu   
Principal Investigator: Laura Hummers, MD         
Sponsors and Collaborators
Georgetown University
CSL Behring
Investigators
Principal Investigator: Virginia D Steen, MD Georgetown University Hospital
  More Information

No publications provided

Responsible Party: Georgetown University
ClinicalTrials.gov Identifier: NCT01785056     History of Changes
Other Study ID Numbers: Pro00000052
Study First Received: January 28, 2013
Last Updated: June 26, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Georgetown University:
Systemic Sclerosis
Diffuse Scleroderma
SSc

Additional relevant MeSH terms:
Scleroderma, Diffuse
Scleroderma, Systemic
Sclerosis
Connective Tissue Diseases
Pathologic Processes
Skin Diseases
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 20, 2014