IgPro10 Dosage for Childhood CIDP

Recruiting at 11 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests different doses of a treatment called IgPro10 (also known as Privigen or Immune Globulin Intravenous) for children with CIDP, a rare nerve condition causing weakness and sensory problems. Researchers aim to evaluate how two different dose levels of IgPro10 work for children who have or haven't previously tried IVIG treatments. Children aged 2 to 17 with symptoms of CIDP might be suitable for this study. As a Phase 4 trial, this study involves an FDA-approved treatment and seeks to understand how it can benefit more patients.

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 is the safety track record for IgPro10?

Research has shown that IgPro10 has been tested for safety in treating chronic inflammatory demyelinating polyneuropathy (CIDP). In earlier studies, most participants tolerated IgPro10 well. However, some risks exist, including rare but serious side effects like blood clots and kidney problems. Many adults using IgPro10 have experienced improvements without major issues.

This trial for children is in a late stage, indicating that the treatment is considered relatively safe based on past research. Prospective participants should consult a doctor for more personalized information. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

IgPro10 is unique because it offers a specialized approach to treating childhood CIDP (Chronic Inflammatory Demyelinating Polyneuropathy) with immunoglobulin therapy. Unlike standard treatments that often include corticosteroids or other forms of IVIG (intravenous immunoglobulin), IgPro10 is being tested at different dosage levels, which may optimize its effectiveness and minimize side effects. Researchers are excited because the precise dosing could lead to better management of CIDP symptoms in children, potentially improving their quality of life significantly.

What is the effectiveness track record for IgPro10 in treating CIDP in children?

Research has shown that IgPro10, a type of intravenous immunoglobulin (IVIG), can effectively treat conditions like chronic inflammatory demyelinating polyneuropathy (CIDP). One study found that 76.9% of patients who had previously received IVIG responded well to the treatment, while 46.7% of those new to IVIG also showed improvement. This suggests that IgPro10 can help reduce symptoms in people with CIDP. Additionally, improvements appeared in the INCAT score, a test that measures changes in nerve-related conditions. This trial will evaluate different dosages of IgPro10 to determine its potential for managing CIDP in children. ^{4 5 6 7 8}

Who Is on the Research Team?

Study Director

Principal Investigator

CSL Behring

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

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]

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]

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]

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]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06752356?term=Human%20immunoglobulin%20G&rank=10

A Study Investigating Intravenous Human Normal Immune ...

The current study is being conducted to assess the efficacy and safety of KIg10 (Intravenous Human Immune globulin 10%) at two different dosages as maintenance ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12006664/

The Results of ADVANCE‐CIDP IVIG Trial

ADVANCE‐CIDP IVIG evaluated the efficacy and safety of immune globulin infusion (human) 10% solution (IVIG 10%; GAMMAGARD LIQUID, also known as Kiovig)

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT00220740

NCT00220740 | Immune Globulin Intravenous (IGIV) For ...

The intent of this study is to demonstrate the efficacy and safety of Immune Globulin Intravenous (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in ...

4.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/23781960/

Efficacy and safety of Privigen(®) in patients with chronic ...

IVIG-pretreated patients demonstrated a higher responder rate than IVIG-naïve patients (76.9% vs. 46.7%). The median (25%-75% quantile) INCAT score improved ...

5.

rarediseaseadvisor.com

rarediseaseadvisor.com/therapies/privigen/

Privigen

Privigen® (immune globulin intravenous, [human], 10% liquid) is an intravenous immunoglobulin (IVIg) stabilized with the amino acid proline.

6.

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.

7.

privigen.com

privigen.com/safe-and-effective/cidp

Safety & Efficacy for CIDP

Clinically proven to improve motor function in adults with CIDP · Safety and efficacy studied in the largest IgG study in CIDP · Most study participants ...

8.

ema.europa.eu

ema.europa.eu/en/documents/product-information/privigen-epar-product-information_en.pdf

Privigen, INN-human normal immunoglobulin - EMA

Chronic inflammatory demyelinating polyneuropathy (CIDP)*. The recommended starting dose is 2 g/kg bw divided over 2 to 5 consecutive days followed by.