PANZYGA for Childhood CIDP

Recruiting at 6 trial locations

Overseen By Patrick Murphy

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Octapharma

Must not be taking: Corticosteroids, Rituximab

Disqualifiers: Inherited neuropathy, DVT, HIV, others

No Placebo Group All 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 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: 5

Duration: 16 weeks

30 Participants Needed

Trial Summary

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.

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. ^{1 2 3 4 5}

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. ^{2 3 4 5 6}

Eligibility Criteria

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

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.

Timeline

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

Treatment Details

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.

Participant Groups

Group I: Panzyga Low Dose Experimental Treatment 1 Intervention

1.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages.

Group II: Panzyga High Dose Experimental Treatment 1 Intervention

2.0g/kg of PANZYGA administered intravenously every four weeks over a period of sixteen weeks for a total of five treatment dosages.

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

Approved in United States as Panzyga for:

• Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
• Primary Immunodeficiency Syndrome
• Immune Thrombocytopenia

Approved in European Union as Panzyga for:

• Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
• Primary Immunodeficiency Syndrome
• Immune Thrombocytopenia

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

Findings from Research

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

The ProCID study involving 142 patients with chronic inflammatory demyelinating polyneuropathy (CIDP) found that infusions of 10% liquid intravenous immunoglobulin (IVIg) at doses up to 2.0 g/kg were safe and well tolerated, with a high tolerance rate of 94.4% for the maximum infusion rate.

While 60.5% of treatment-emergent adverse events (TEAEs) were related to treatment, most were mild, and serious TEAEs were rare, indicating a favorable safety profile for IVIg in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. Cornblath, DR., van Doorn, PA., Hartung, HP., et al.[2023]

Panzyga® is a new 10% intravenous immune globulin that is effective for treating immunodeficiencies and autoimmune diseases, showing high purity and functional IgG activity.

The product is stable for at least two years when refrigerated and can be stored at room temperature for up to twelve months, making it convenient for use in clinical settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Biochemical characterization and stability of immune globulin intravenous 10% liquid (Panzyga&#174;). Mersich, C., Ahrer, K., Buchacher, A., et al.[2017]

In a Phase III trial involving 67 patients with Primary Immunodeficiency Diseases (PID), the new 10% IVIg (BT595) demonstrated a very low rate of serious bacterial infections (0.01 per subject-year), indicating its efficacy in infection prevention.

The treatment was found to be safe and well tolerated, with only 8% of infusions resulting in mild adverse events like headache and fatigue, and no significant adverse effects at higher infusion rates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy, safety and pharmacokinetics of a new 10% normal human immunoglobulin for intravenous infusion, BT595, in children and adults with primary immunodeficiency disease. Kriván, G., Borte, M., Harris, JB., et al.[2022]

References

1. Canada pubmed.ncbi.nlm.nih.gov

Intravenous immunoglobulin in the treatment of polyarticular juvenile rheumatoid arthritis: a phase I/II study. Pediatric Rheumatology Collaborative Study Group. [2013]

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

Intravenous immunoglobulin 10% in children with primary immunodeficiency diseases. [2019]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Human Intravenous Immunoglobulin 10% (Panzyga®) in Patients with Primary Immunodeficiency Diseases: a Two-Stage, Multicenter, Prospective, Open-Label Study. [2018]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. [2023]

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

Biochemical characterization and stability of immune globulin intravenous 10% liquid (Panzyga&#174;). [2017]

6. England pubmed.ncbi.nlm.nih.gov

Efficacy, safety and pharmacokinetics of a new 10% normal human immunoglobulin for intravenous infusion, BT595, in children and adults with primary immunodeficiency disease. [2022]