Systemic SclerosisNCT01785056
IVIG Treatment in Systemic Sclerosis
A study testing Privigen in people with Systemic Sclerosis and Diffuse Scleroderma, run by Georgetown University at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis, Diffuse Scleroderma
- What is being tested?
- Privigen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2013to Jan 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21224
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
About this study
Official title: A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety and Efficacy of Intravenous Immunoglobulin Treatment in Patients With Systemic Sclerosis
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.
Read the 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.
- Conditions
- Systemic Sclerosis, Diffuse Scleroderma
- Being tested
- Privigen
- Keywords
- Systemic Sclerosis, Diffuse Scleroderma, SSc
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrivigenBiological
Subjects will receive 2 g/kg/mo of Privigen® for 6 months if randomized to the treatment group.
| Study group | Role | What they receive |
|---|---|---|
| Privigen | Experimental | 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 IVIGfor 6 months. Each dose will be split into 2 to 4 infusions on consecutive days to achieve the total monthly dose. Receives: Biological: Privigen |
| Placebo (Albuminar-5) | Placebo comparator | 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. Receives: Biological: Privigen |
What is being measured
The primary outcome is the main question the study is designed to answer.
To study the effects of IVIG on the skin in patients with scleroderma
The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin
Measured: At the 4 month visit
Secondary outcomes (3)
To determine any toxicity of IVIG in scleroderma
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.
Measured: At the 4 month visit
To evaluate the effects of IVIG on pulmonary function
Pulmonary Function Tests (PFTs) will be performed to determine these changes.
Measured: At the 4 month visit
Muscle, joint, and inflammatory parameters
Done by performing a physical exam
Measured: At the 4 month visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2013 (estimated)
- Study startApr 2013
- Primary completionJan 2018
- Study completionJan 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Georgetown University
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring
- Responsible party
- Sponsor
- Investigators
- Virginia D Steen, MD, Principal investigator, Georgetown University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01785056
- Sponsor's protocol ID
- Pro00000052
- Status last verified
- Jan 2018
- First submitted
- 28 Jan 2013
- Last updated
- 17 Jul 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety and Efficacy of Intravenous Immunoglobulin Treatment in Patients With Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT01785056
- Acronym / sponsor ID
- Pro00000052
- Conditions
- Systemic Sclerosis, Diffuse Scleroderma
- Keywords
- Systemic Sclerosis, Diffuse Scleroderma, SSc
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Georgetown UniversityUniversity, hospital or other
- Collaborators
- CSL Behring
- Study start
- Apr 2013
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jan 2019 (estimated)
- First posted
- 6 Feb 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Privigen | Experimental | Biological: PrivigenPrivigen 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) | Placebo comparator | Biological: 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PrivigenSubjects will receive 2 g/kg/mo of Privigen® for 6 months if randomized to the treatment group. | Biological | Placebo (Albuminar-5), Privigen |
Primary outcome
To study the effects of IVIG on the skin in patients with scleroderma
Time frame: At the 4 month visit
The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin
3 secondary outcomes
To determine any toxicity of IVIG in scleroderma
Time frame: At the 4 month visit
To evaluate the effects of IVIG on pulmonary function
Time frame: At the 4 month visit
Muscle, joint, and inflammatory parameters
Time frame: At the 4 month visit
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2018.
Related trials for Systemic Sclerosis
Active, not recruiting
Patient Reported Outcomes With UVA-1 Therapy for Treatment of Sclerosing Skin Diseases
Scleroderma, Systemic, Morphea
Not yet recruitingPhase 1
A Phase 1 Study of QLS2416 Injection in Participants With Autoimmune Diseases
Systemic Lupus Erythematosus, Rheumatoid Arthritis (RA)
RecruitingPhase 1/2
A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2)
Systemic Sclerosis, Idiopathic Inflammatory Myopathies
Active, not recruitingObservational study
Cross-cultural Adaptation and Validation of the Localised Scleroderma Quality of Life Instrument (LoSQI) in Juvenile Localised Scleroderma (JLS)
Scleroderma
Recruiting
Tissue Modeling in Systemic Sclerosis Using Induced Pluripotent Stem Cells (iPSCs)
Systemic Sclerosis (SSc)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Virginia D Steen, MD principal investigator
Georgetown University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Privigen in people with Systemic Sclerosis and Diffuse Scleroderma, run by Georgetown University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To study the effects of IVIG on the skin in patients with scleroderma (time frame: At the 4 month visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jul 2018.