IVIG for Scleroderma

Not currently recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Georgetown University

Must be taking: Methotrexate, Cellcept, Imuran, Anti-TNF

Must not be taking: Prednisone, Cytoxan, Rituximab, D-penicillamine

Disqualifiers: DVT, Stroke, Anaphylaxis, others

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Privigen, administered through an IV, to assess its effects on the skin in people with scleroderma. Scleroderma is a disease that can harden the skin and cause issues with muscles, joints, the stomach, and lungs. Participants will receive either the Privigen treatment or a placebo, a harmless substitute resembling the real treatment. This study targets individuals who have had scleroderma for less than five years, have active skin issues, and have not improved with other treatments. It will occur at Georgetown University Hospital and Johns Hopkins Hospital over the course of a year. As an unphased trial, this study provides a unique opportunity to advance the understanding of scleroderma treatments.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that you should not have used more than 10 mg of prednisone, or any dose of cytoxan, d-penicillamine, or rituximab in the last 3 months. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that Privigen is likely to be safe for humans?

Research has shown that Privigen, the treatment under study, is safe for conditions like primary immune deficiency and chronic inflammatory demyelinating polyneuropathy. Studies indicate it is generally well-tolerated. However, some side effects may occur, such as headaches, nausea, and fever. Warnings exist about possible serious issues, like blood clots or kidney problems, which are rare but important to consider. Older individuals or those with kidney issues should discuss these risks with the study team. Overall, Privigen has a strong safety record, but individual experiences may vary. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for systemic sclerosis, which often include immunosuppressants like methotrexate or cyclophosphamide, Privigen offers a novel approach by using polyvalent human immunoglobulin G (IgG) for intravenous administration. This treatment is unique because it provides a concentrated dose of antibodies that can modulate the immune system differently than typical immunosuppressants. Researchers are excited about Privigen's potential to target multiple pathways involved in systemic sclerosis, possibly offering enhanced efficacy and fewer side effects compared to current options. By delivering a ready-to-use, sterile liquid preparation, Privigen also simplifies the treatment process, potentially improving patient compliance and outcomes.

What evidence suggests that Privigen might be an effective treatment for scleroderma?

Research shows that Privigen, administered through a drip, effectively treats various health issues. In earlier studies, Privigen improved muscle and grip strength, crucial for individuals with systemic sclerosis, a type of scleroderma. It also benefited 60.7% of patients with certain long-term conditions, indicating its potential usefulness. Most patients responded well to Privigen, demonstrating its effectiveness. In this trial, participants will receive either Privigen or a placebo (Albuminar-5) to evaluate its potential in improving symptoms of systemic sclerosis, particularly in the skin, muscles, and joints. ^{3 4 6 7 8}

Who Is on the Research Team?

Virginia D Steen, MD

Principal Investigator

Georgetown University Hospital

Are You a Good Fit for This Trial?

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;

Disease duration of less than 5 years from the first non-Raynaud's symptom of scleroderma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 2 g/kg/month of Privigen or placebo for 6 months, with each dose split into 2 to 4 infusions on consecutive days

6 months

Monthly visits for infusions and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment, including physical exams and laboratory assessments

6 months

Monthly visits for monitoring

What Are the Treatments Tested in This Trial?

Interventions

• Privigen

How Is the Trial Designed?

Group I: Privigen Experimental Treatment 1 Intervention

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.

Group II: Placebo (Albuminar-5) Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

Georgetown University

Lead Sponsor

Trials

355

Recruited

142,000+

CSL Behring

Industry Sponsor

Trials

204

Recruited

1,207,000+

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University

Published Research Related to This Trial

The definition of an adverse drug event (ADE) should be tailored to the specific purpose of the evaluation, with stricter definitions needed for scientific studies and more flexible ones for clinical use.

Postmarketing safety data for drugs is limited at launch, but can be supplemented through systems like the FDA's Spontaneous Reporting System and targeted research projects, emphasizing the importance of robust epidemiological evidence for regulatory actions like drug recalls.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse drug events: identification and attribution. Rogers, AS.[2022]

From 2006 to 2014, serious adverse drug events (ADEs) reported to the FDA increased two-fold, totaling 902,323 serious outcomes, including 244,408 deaths, indicating a growing concern for drug safety in real-world settings.

The majority of serious ADE reports came from health professionals (47.3%), with a significant number of reports among older adults (72.6% for ages 45 and above), highlighting the need for targeted monitoring and safety measures for this age group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Serious Adverse Drug Events Reported to the FDA: Analysis of the FDA Adverse Event Reporting System 2006-2014 Database. Sonawane, KB., Cheng, N., Hansen, RA.[2023]

Effective clinical drug safety surveillance is crucial for protecting public health and ensuring the commercial success of pharmaceutical products, as it helps monitor and manage adverse drug events.

The process of drug safety surveillance is governed by FDA regulations and involves systematic management of adverse event reports, emphasizing the importance of clear communication and data handling to improve drug safety outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A primer of drug safety surveillance: an industry perspective. Part I: Information flow, new drug development, and federal regulations. Allan, MC.[2019]

Citations

1. privigen.com privigen.com/hcp/efficacy-and-safety

Efficacy and safety | PRIVIGEN Immune Globulin ... Privigen improved muscle strength as measured by MRC sum score. Average improvement in MRC sum score was 3.6 points · Dominant hand grip strength improved by a ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3910165/

Efficacy and safety of Privigen® in patients with chronic ... The overall responder rate at completion was 60.7% (95% confidence interval [CI]: 42.41%–76.43%). IVIG-pretreated patients demonstrated a higher responder rate ...

3. nature.com nature.com/articles/s41598-023-45092-5

Comparative effectiveness of two intravenous ... Outcomes are summarized in Table 3. Privigen demonstrated a lower IVIG resistance rate at 9.4% in comparison to TBSF, which exhibited a rate of ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4165870/

Clinical Experience With an L-Proline–Stabilized 10 ... Privigen® is well tolerated at infusion rates up to 8 mg/kg/min (4.8 mL/kg/h), with many patients tolerating infusion rates up to 12 mg/kg/min (7.2 mL/kg/h) [8– ...

5. tandfonline.com tandfonline.com/doi/abs/10.1179/102453309X439773

Efficacy and safety of Privigen®, a novel liquid intravenous ... Thus, in patients with chronic ITP, a two-day regimen of Privigen® was effective in increasing platelet count, reducing bleeding events and was well tolerated. ...

6. labeling.cslbehring.com labeling.cslbehring.com/SDS/AU/Privigen/EN/Privigen-Safety-Data-Sheet.pdf

Privigen® - CSL Behring IMPORTANT NOTICE: This Safety Data Sheet (SDS) is prepared by CSL Behring in accordance with Safe Work. Australia National Code of Practice for the ...

7. fda.gov fda.gov/media/83304/download

Package Insert - PRIVIGEN WARNING: THROMBOSIS, RENAL DYSFUNCTION AND ACUTE. RENAL FAILURE. See full prescribing information for complete boxed warning.

8. privigen.com privigen.com/important-safety-information

Important Safety Information | PRIVIGEN Immune Globulin ... Thrombosis (blood clotting) may occur with immune globulin products, including Privigen. Risk factors may include advanced age, prolonged immobilization, a ...