IVIG Treatment in Systemic Sclerosis

Recruiting at 1 trial location

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

14 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing an antibody solution given through an IV in patients with scleroderma who haven't improved with standard treatments. The goal is to see if this treatment can help their immune system work better and reduce disease symptoms. The study will last for several months and involve a small group of patients.

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.

What safety data exists for the treatment known as Privigen?

The safety of a drug is monitored through systems like the FDA's Spontaneous Reporting System, which collects data on adverse drug events (unwanted or harmful effects). This helps identify potential risks and ensure patient safety after the drug is marketed. ^{1 2 3 4 5}

Research Team

Virginia D Steen, MD

Principal Investigator

Georgetown University Hospital

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;

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

Timeline

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

Treatment Details

Interventions

• Privigen

Participant Groups

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

Findings from Research

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Adverse event reporting in publications compared with sponsor database for cancer clinical trials. [2007]

2. United States pubmed.ncbi.nlm.nih.gov

Adverse drug events: identification and attribution. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

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

4. United States pubmed.ncbi.nlm.nih.gov

Serious Adverse Drug Events Reported to the FDA: Analysis of the FDA Adverse Event Reporting System 2006-2014 Database. [2023]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Adverse event reporting and patient safety at a University Hospital: Mapping, correlating and associating events for a data-based patient risk management. [2017]