IgPro10 Dosage for Childhood CIDP

Recruiting at 12 trial locations

Overseen By Trial Registration Coordinator

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: CSL Behring

Disqualifiers: Inherited neuropathy, Developmental delay, Thrombotic episode, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

30 Participants Needed

Trial Summary

What is the purpose of this trial?

A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed to IVIG treatment

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug IgPro10 for treating childhood CIDP?

IgPro10, also known as Privigen, has been shown to be effective in increasing platelet counts and reducing bleeding in patients with chronic immune thrombocytopenic purpura (ITP), and it is well tolerated in patients with primary immunodeficiencies (PID). This suggests it may have potential benefits for other immune-related conditions like childhood CIDP. ^{1 2 3 4 5}

How is the drug IgPro10 unique in treating childhood CIDP?

IgPro10 is a liquid intravenous immunoglobulin product stabilized with L-proline, which helps maintain its stability over time. It is administered intravenously and can be infused at higher flow rates, reducing the infusion time compared to some other treatments. ^{1 2 4 6 7}

Research Team

Study Director

Principal Investigator

CSL Behring

Eligibility Criteria

This trial is for children and teenagers aged 2 to 17 with confirmed or possible Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). They can be new or past users of IVIG therapy. Those ineligible include individuals without CIDP symptoms, a history of inherited neuropathies, developmental delays, severe reactions to blood products, pregnant/breastfeeding females, and those not using contraception.

Inclusion Criteria

I am between 2 and 17 years old with a diagnosis or suspicion of CIDP.

Exclusion Criteria

Pregnant or breastfeeding mother

I or my family have a history of inherited nerve damage.

I do not have symptoms of CIDP.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Exploration Phase

Participants receive one of two dose levels of Privigen to explore efficacy and safety

24 weeks

Randomization Phase

Participants are randomized to receive one of two dose levels of Privigen to assess CIDP improvement and recovery

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• IgPro10

Trial Overview The study is testing two different doses of IgPro10 (Privigen) in young patients with CIDP. It's an open-label trial meaning everyone knows what treatment they're getting. The goal is to see how well the different doses work and how safe they are over time.

Participant Groups

Group I: IgPro10 (dose level 2) Experimental Treatment 1 Intervention

Group II: IgPro10 (dose level 1) Experimental Treatment 1 Intervention

IgPro10 is already approved in United States, European Union for the following indications:

Approved in United States as Privigen for:

• Primary humoral immunodeficiency (PI)
• Chronic immune thrombocytopenic purpura (ITP) in patients age 15 years and older
• Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults

Approved in European Union as Privigen for:

• Primary immunodeficiency syndromes
• Chronic immune thrombocytopenic purpura (ITP)
• Chronic inflammatory demyelinating polyneuropathy (CIDP)

Who Is Running the Clinical Trial?

CSL Behring

Lead 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

In a study of 11 Japanese patients with primary immunodeficiency, IgPro10 (Privigen®) was found to be well tolerated, with only 19 adverse events reported, none of which were severe, resulting in a low adverse event rate per infusion of 0.442.

The study demonstrated that 90% of participants could tolerate high infusion rates of up to 12 mg/kg/min, significantly reducing infusion time, which aligns with safety profiles observed in previous studies of IgPro10 in non-Japanese patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and tolerability of IgPro10 in Japanese primary immunodeficiency patients: a registrational study. Morio, T., Gotoh, K., Imagawa, T., et al.[2022]

IgPro10 (Privigen) demonstrated stability for up to 36 months at room temperature (25 degrees C), indicating its potential for safe storage and use without refrigeration.

The formulation maintained high purity (>98%) of the IgG fraction and preserved its biological activity and effector functions throughout the study, suggesting its efficacy as an intravenous immunoglobulin product.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stability over 36 months of a new liquid 10% polyclonal immunoglobulin product (IgPro10, Privigen) stabilized with L-proline. Cramer, M., Frei, R., Sebald, A., et al.[2013]

In a study involving 8 pediatric patients with Primary Immunodeficiencies who were sensitive to previous IVIG infusions, Privigen® was found to be safe, with only three patients experiencing mild adverse events compared to more severe reactions with previous treatments.

None of the patients required premedication with Privigen®, and the treatment maintained stable IgG levels without any episodes of bacterial infection, indicating both safety and efficacy in this sensitive population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A 10% liquid immunoglobulin preparation for intravenous use (Privigen®) in paediatric patients with primary immunodeficiencies and hypersensitivity to IVIG. Lozano-Blasco, J., Martín-Mateos, MA., Alsina, L., et al.[2014]

Gammaplex 5% has been shown to be effective and safe in preventing serious infections and treating bleeding in patients with primary immunodeficiency diseases and immune thrombocytopenic purpura.

Gammaplex 10% is expected to have similar therapeutic effects as Gammaplex 5%, while allowing for a 34% reduction in infusion time, providing a more convenient treatment option without compromising safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gammaplex&#174; 5 and 10% in the treatment of primary immunodeficiency and chronic immune thrombocytopenic purpura. Wasserman, RL.[2018]

In a study involving 57 patients with chronic immune thrombocytopenic purpura (ITP), Privigen, a 10% liquid human IgG formulation, effectively increased platelet counts in 80.7% of patients by day 7 after a two-day treatment regimen.

Privigen was well tolerated, with most infusions completed at the maximum rate and only mild to moderate adverse events reported, indicating a favorable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Privigen, a novel liquid intravenous immunoglobulin formulation, in adolescent and adult patients with chronic immune thrombocytopenic purpura. Robak, T., Salama, A., Kovaleva, L., et al.[2022]

Intravenous immune globulin (IVIG) is primarily composed of immunoglobulin G (IgG) and is effective in treating conditions like idiopathic thrombocytopenic purpura (ITP) and primary humoral immunodeficiencies (PHI), with specific dosages recommended for each condition.

While IVIG is a valuable treatment option for various immunodeficiencies and autoimmune disorders, its high cost and the need for more conclusive efficacy data in some conditions are important considerations for its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical uses of intravenous immune globulin. Knapp, MJ., Colburn, PA.[2019]

The pharmacokinetics of IgPro10, a new 10% liquid intravenous immunoglobulin product, were studied in patients with common variable immunodeficiency (CVID) and X-linked agammaglobulinaemia (XLA) over a median treatment duration of 4 months, showing a significant increase in total IgG serum concentrations from 10.2 g/l to 23.2 g/l after infusion.

IgPro10 demonstrated a median terminal half-life of 36.6 days, with pharmacokinetics similar to existing intact IgG products, ensuring adequate plasma concentrations of IgG and specific antibodies against key pathogens when administered at 3- or 4-week intervals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics of a new 10% intravenous immunoglobulin in patients receiving replacement therapy for primary immunodeficiency. Wasserman, RL., Church, JA., Peter, HH., et al.[2009]

References

1. Japan pubmed.ncbi.nlm.nih.gov

Safety and tolerability of IgPro10 in Japanese primary immunodeficiency patients: a registrational study. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Stability over 36 months of a new liquid 10% polyclonal immunoglobulin product (IgPro10, Privigen) stabilized with L-proline. [2013]

3. Singapore pubmed.ncbi.nlm.nih.gov

A 10% liquid immunoglobulin preparation for intravenous use (Privigen®) in paediatric patients with primary immunodeficiencies and hypersensitivity to IVIG. [2014]

4. England pubmed.ncbi.nlm.nih.gov

Gammaplex&#174; 5 and 10% in the treatment of primary immunodeficiency and chronic immune thrombocytopenic purpura. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Privigen, a novel liquid intravenous immunoglobulin formulation, in adolescent and adult patients with chronic immune thrombocytopenic purpura. [2022]

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

Clinical uses of intravenous immune globulin. [2019]

7. Netherlands pubmed.ncbi.nlm.nih.gov

Pharmacokinetics of a new 10% intravenous immunoglobulin in patients receiving replacement therapy for primary immunodeficiency. [2009]