30 Participants Needed

PANZYGA for Childhood CIDP

Recruiting at 6 trial locations
MR
PM
Overseen ByPatrick Murphy
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) for those who have received immunoglobulin or plasma exchange within eight weeks before the study. Additionally, patients on unstable doses of corticosteroids or rituximab may not be eligible. It's best to discuss your specific medications with the study team.

How is the drug Panzyga unique for treating childhood CIDP?

Panzyga is a high purity intravenous immunoglobulin (IVIG) that is stable for long periods and has a low level of additional plasma proteins, which may reduce the risk of side effects. It is designed to be similar to normal plasma, making it potentially safer and more effective for treating conditions like childhood CIDP.12345

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

Research shows that Panzyga, a type of intravenous immunoglobulin (IVIG), is effective in treating conditions like primary immunodeficiency diseases and chronic inflammatory demyelinating polyneuropathy (CIDP) in adults, suggesting it may also help children with CIDP.12356

Are You a Good Fit for This Trial?

This trial is for children aged 2 to 17 with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). They must have functional impairment due to CIDP and not have a history of inherited neuropathies, previous immunoglobulin therapy failure, or recent treatments that could affect the study. Participants need parental consent and may require birth control if applicable.

Inclusion Criteria

Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by the patient, if age appropriate per Independent Ethics Committee [IEC]/Institutional Research Board [IRB] requirements)
I am between 2 and 17 years old.
Patients with a diagnosis of definite or possible CIDP based on European Neuromuscular Center (ENMC) criteria
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Exclusion Criteria

I am not pregnant, breastfeeding, or planning to become pregnant and will use effective birth control during the study.
Any other condition(s) that, in the Investigator's opinion, makes it undesirable for the patient to participate in the study or may interfere with protocol compliance
I am willing and able to follow the study rules.
See 12 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive PANZYGA intravenously every four weeks over a period of sixteen weeks

16 weeks
5 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Panzyga
Trial Overview The trial tests different dosing regimens of PANZYGA, an immunoglobulin therapy, in pediatric patients with CIDP. It aims to determine the safest and most effective dosage by monitoring how well the treatment works and any side effects it causes in these young patients.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Panzyga Low DoseExperimental Treatment1 Intervention
Group II: Panzyga High DoseExperimental Treatment1 Intervention

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

🇺🇸
Approved in United States as Panzyga for:
🇪🇺
Approved in European Union as Panzyga for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Octapharma

Lead Sponsor

Trials
86
Recruited
11,300+

Wolfgang Marguerre

Octapharma

Chief Executive Officer since 1983

MBA from INSEAD

Wolfgang Frenzel

Octapharma

Chief Medical Officer since 2010

MD from University of Vienna

Published Research Related to This Trial

In a study involving 25 children with polyarticular juvenile rheumatoid arthritis, 76% experienced significant clinical improvement after receiving intravenous immune globulin (IVIG) treatment, indicating its efficacy for this condition.
The treatment was found to be safe, with no serious adverse effects reported, and those who continued IVIG showed sustained improvement, although the benefits diminished quickly after stopping the treatment.
Intravenous immunoglobulin in the treatment of polyarticular juvenile rheumatoid arthritis: a phase I/II study. Pediatric Rheumatology Collaborative Study Group.Giannini, EH., Lovell, DJ., Silverman, ED., et al.[2013]
In a study involving 25 children with primary immunodeficiency, IVIG 10% demonstrated a very low rate of serious bacterial infections at 0.04 per patient-year, indicating its efficacy in preventing infections.
The treatment was found to be safe, with common side effects including abdominal pain, headache, and chills, while maintaining effective immunoglobulin G levels between infusions.
Intravenous immunoglobulin 10% in children with primary immunodeficiency diseases.Ochs, HD., Melamed, I., Borte, M., et al.[2019]
In a phase 3 study involving 51 patients with primary immunodeficiency diseases, panzyga® (intravenous immunoglobulin 10%) effectively prevented serious bacterial infections (SBIs), achieving a low rate of 0.08 SBIs per patient-year.
The treatment was generally well tolerated, with no adverse events leading to withdrawal or death, indicating a favorable safety profile while meeting efficacy standards set by regulatory agencies.
Efficacy and Safety of Human Intravenous Immunoglobulin 10% (Panzyga®) in Patients with Primary Immunodeficiency Diseases: a Two-Stage, Multicenter, Prospective, Open-Label Study.Borte, M., Melamed, IR., Pulka, G., et al.[2018]

Citations

Intravenous immunoglobulin in the treatment of polyarticular juvenile rheumatoid arthritis: a phase I/II study. Pediatric Rheumatology Collaborative Study Group. [2013]
Intravenous immunoglobulin 10% in children with primary immunodeficiency diseases. [2019]
Efficacy and Safety of Human Intravenous Immunoglobulin 10% (Panzyga®) in Patients with Primary Immunodeficiency Diseases: a Two-Stage, Multicenter, Prospective, Open-Label Study. [2018]
Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. [2023]
Biochemical characterization and stability of immune globulin intravenous 10% liquid (Panzyga®). [2017]
Efficacy, safety and pharmacokinetics of a new 10% normal human immunoglobulin for intravenous infusion, BT595, in children and adults with primary immunodeficiency disease. [2022]
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