IgPro10 Dosage for Childhood CIDP
Recruiting in Palo Alto (17 mi)
+13 other locations
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 4
Recruiting
Sponsor: CSL Behring
Disqualifiers: Inherited neuropathy, Developmental delay, Thrombotic episode, others
No Placebo Group
Prior Safety Data
Approved in 2 Jurisdictions
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.
12345How 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.
12467Eligibility 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.
+5 more
Trial 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
Participant Groups
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.
2Treatment groups
Experimental Treatment
Group I: IgPro10 (dose level 2)Experimental Treatment1 Intervention
Group II: IgPro10 (dose level 1)Experimental Treatment1 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)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Mayo ClinicRochester, MN
University of PittsburghPittsburgh, PA
Seattle Children's HospitalSeattle, WA
Children's Hospital of Los AngelesLos Angeles, CA
More Trial Locations
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Who Is Running the Clinical Trial?
CSL BehringLead Sponsor
References
Safety and tolerability of IgPro10 in Japanese primary immunodeficiency patients: a registrational study. [2022]Studies investigating the safety of IgPro10 (Privigen®, CSL Behring, King of Prussia, PA, USA) in Japanese patients with primary immunodeficiency (PID) are lacking. This study evaluated safety and tolerability of IgPro10 in Japanese patients with PID. In this prospective, open-label, single-arm, registrational study for Japan, IgPro10 was administered intravenously at pre-study doses of 138-556 mg/kg body weight per 3-/4-weekly dosing cycle for up to 4 months. Frequency and intensity of adverse events (AEs), their relationship to IgPro10 and AE rate per infusion (AERI) were evaluated. Of 11 enrolled patients, 10 completed the study. The median (range) total duration of exposure was 16.14 (4.1-16.3) weeks. Eight patients reported 19 AEs, none severe (based on maximum severity), giving an AERI of 0.442. One AE was deemed related to IgPro10 treatment. Three patients experienced temporally associated AEs. No serious AEs or deaths were reported. Nine patients (90%) who completed the study tolerated flow rates of ≥ 8 mg/kg/min; 5 tolerated 12 mg/kg/min (7.2 mL/kg/h), translating into a threefold decrease in mean infusion time. IgPro10 was well tolerated at a flow rate of up to 12 mg/kg/min. Safety and tolerability findings were consistent with previously reported studies in non-Japanese patients with PID.
Stability over 36 months of a new liquid 10% polyclonal immunoglobulin product (IgPro10, Privigen) stabilized with L-proline. [2013]IgPro10 (Privigen) is a new liquid intravenous immunoglobulin (IVIG) product that is formulated with 250 mM L-proline at pH 4.8. A 3-year study was performed to assess its stability.
A 10% liquid immunoglobulin preparation for intravenous use (Privigen®) in paediatric patients with primary immunodeficiencies and hypersensitivity to IVIG. [2014]The objective of this study was to evaluate safety and efficacy of Privigen®, a 10% intravenous immunoglobulin (IVIG), in a particular group of paediatric patients (highly sensitive to previous IVIG infusion) affected with Primary Immunodeficiencies (PID).
Gammaplex® 5 and 10% in the treatment of primary immunodeficiency and chronic immune thrombocytopenic purpura. [2018]Immunoglobulin G is used to both prevent infection in primary immunodeficiency diseases (PIDs) and prevent bleeding in immune thrombocytopenic purpura. Gammaplex is a highly purified human intravenous immunoglobulin G available as 5 and 10% liquid formulations. Gammaplex 5% has proven efficacy and safety in PID and immune thrombocytopenic purpura, protecting against serious acute bacterial infections and treating bleeding by improving platelet counts, respectively. The therapeutic effect of Gammaplex 10% is expected to be similar to that of Gammaplex 5% based on demonstrated bioequivalence in a bridging study in PID. The availability of Gammaplex 10% provides another option to individualize therapy according to patient needs, allowing a 34% reduction in infusion time without compromising safety and tolerability.
Efficacy and safety of Privigen, a novel liquid intravenous immunoglobulin formulation, in adolescent and adult patients with chronic immune thrombocytopenic purpura. [2022]Intravenous immunoglobulin (IVIG) has become a mainstay of treatment for acute and chronic immune thrombocytopenic purpura (ITP). The efficacy and safety of Privigen, a new, ready-to-use, 10% liquid human IgG formulation, was evaluated in this open-label, multicentre study. Privigen infusions (1 g/kg per day for 2 consecutive days, days 1 and 2) were given to 57 adolescent and adult patients with chronic ITP and platelet counts or =50 x 10(9)/l. Correspondingly, haemorrhage number and severity were significantly reduced. Adverse events were generally mild or moderate and typical of underlying disease and IVIG treatment. Privigen was well tolerated - 104 of 114 infusions were performed at the maximum permitted infusion rate (4 mg/kg/min). 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.
Clinical uses of intravenous immune globulin. [2019]The preparation, pharmacokinetics, clinical uses, dosage and administration, and adverse effects of intravenous immune globulin (IVIG) are reviewed. IVIG, which consists primarily of immunoglobulin G (IgG), is initially prepared from pooled human plasma by using the Cohn-Oncley fractionation procedure. Secondary treatments render the preparation suitable for i.v. use. The specific antibody content of IVIG depends on the geographic location of the plasma donors, the product, and the product lot. The metabolism of IgG appears to follow a multicompartmental, first-order process. The half-life of IgG is dependent on the half-lives of the IgG subclasses; three of the four subclasses have half-lives in the range of 23-25 days. IVIG is indicated in the treatment of idiopathic thrombocytopenic purpura (ITP) and as replacement therapy in primary humoral immunodeficiencies (PHI). IVIG has also been used for antimicrobial prophylaxis in bone marrow transplant and burn patients and in patients with malignancies. Patients with HIV infection, cystic fibrosis, neonatal sepsis, and respiratory syncytial virus infection may also benefit from prophylaxis or treatment with IVIG. The recommended dosage of IVIG in ITP is 400 mg/kg/day for two to five days. For the treatment of PHI, the usual dosage is 100-400 mg/kg every three or four weeks. Adverse reactions are often mild and are usually related to the infusion rate. Intravenous immune globulin is a valuable therapeutic tool in several immunodeficiency and autoimmune states, but IVIG products are expensive, and conclusive data on their efficacy in the treatment of many disorders remain to be obtained.
Pharmacokinetics of a new 10% intravenous immunoglobulin in patients receiving replacement therapy for primary immunodeficiency. [2009]Intravenous immunoglobulin (IVIg) is used in treating immunodeficiencies and autoimmune or inflammatory disorders. As manufacturing processes and storage can alter IgG molecules, pharmacokinetic assessments are important for new preparations. Thus, we studied pharmacokinetics of IgPro10, a new 10% liquid IVIg product stabilised with l-proline, in patients with common variable immunodeficiency (CVID) and X-linked agammaglobulinaemia (XLA). Patients received IgPro10 for >or=4 months (median dose of 444mg/kg, at 3- or 4-week intervals). Median total IgG serum concentrations increased from 10.2g/l pre-infusion to 23.2g/l at infusion end. Serum IgG concentrations decreased in a biphasic manner; median terminal half-life was 36.6 days. Median half-lives were 33.2 for IgG(1), 36.3 for IgG(2), 25.9 for IgG(3) and 36.4 days for IgG(4). Specific antibody concentrations (anti-CMV, anti-Hemophilus influenzae type B, anti-tetanus toxoid and anti-Streptococcus pneumoniae) decreased with median half-lives of 22.3-30.5 days. IgPro10 pharmacokinetics were similar in patients with CVID and XLA, although patients with CVID showed higher levels of anti-tetanus and anti-S. pneumoniae antibodies than patients with XLA, suggesting residual specific antibody production. IgPro10 pharmacokinetics fulfilled expectations for and were similar to intact IgG products. Administration of IgPro10 at 3- or 4-week intervals achieved sufficient plasma concentrations of total IgG, IgG subclasses and antibodies specific to important pathogens.