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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety and effectiveness of Panzyga, an immune globulin therapy, for children with CIDP, a condition affecting the nerves that can cause weakness or numbness. Researchers are testing two different dose levels of Panzyga to determine which is most effective. Children diagnosed with CIDP who experience difficulties with daily activities may be suitable candidates for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

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.

Is there any evidence suggesting that PANZYGA is likely to be safe for children with CIDP?

Research has shown that Panzyga, administered through an IV, is generally safe for adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). One study found that doses up to 2.0 g/kg were safe for these patients. Although this study focused on adults, it provides some insight into the treatment's safety.

The FDA has approved Panzyga for treating CIDP in adults, indicating a known safety record for this use. However, its safety and effectiveness in children with CIDP remain unknown, highlighting the importance of this clinical trial.

In this trial, participants will receive either a high dose (2.0 g/kg) or a low dose (1.0 g/kg) of Panzyga. The trial aims to assess how well these doses are tolerated in children. While existing research suggests safety, the trial will offer specific information for children. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Panzyga for childhood CIDP because it offers a targeted approach using immunoglobulin therapy. Unlike traditional treatments that may involve the use of corticosteroids or plasma exchange, Panzyga is administered intravenously at a high or low dose every four weeks, potentially offering more consistent and manageable results. This method may reduce inflammation and improve nerve function more precisely, which is particularly beneficial for young patients needing effective, yet gentle, treatment options. With the possibility of fewer side effects and a streamlined administration schedule, Panzyga stands out as a promising alternative to existing therapies.

What evidence suggests that PANZYGA might be an effective treatment for CIDP in children?

Research has shown that Panzyga, a type of intravenous immunoglobulin (IVIg), effectively treats Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). Studies have found that IVIg therapy can significantly reduce disability and symptoms in people with CIDP. The FDA has approved Panzyga for treating adults with CIDP, confirming its effectiveness in managing this condition. Clinical reviews have reported positive results, with patients noticing improvements in their symptoms. In this trial, researchers are studying both high and low doses of Panzyga to better understand its effectiveness for children with CIDP. ^{2 4 6 7 8}

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

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 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?

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

Published Research Related to This Trial

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 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]

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 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]

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]

Citations

1. 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 ...

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. fda.gov fda.gov/media/146188/download

Clinical Review (125587/70)- PANZYGA To provide confirmatory data on the effect of 1.0 g/kg Panzyga (NewGam) every three weeks in patients with active CIDP based on the percentage ...

4. pfizer.com pfizer.com/news/press-release/press-release-detail/us-fda-approves-panzygar-treatment-adults-chronic

U.S. FDA Approves PANZYGA® for the Treatment of Adults ... PANZYGA is the only intravenous immunoglobulin (IVIg) with two FDA-approved maintenance dosing options for CIDP, helping to meet the clinical needs of patients.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3090105/

Intravenous immunoglobulin for the treatment of chronic ... IVIg therapy was statistically superior to placebo in reducing disability and impairment among patients with CIDP. The effectiveness of IVIg was similar to that ...

6. fda.gov fda.gov/files/vaccines,%20blood%20&%20biologics/published/Package-Insert-PANZYGA.pdf

Package Insert - PANZYGA Treatment of Immune Thrombocytopenia (ITP) in children. The safety and effectiveness of PANZYGA have not been established in pediatric patients with ITP.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10442284/

Safety and Tolerability of Intravenous Immunoglobulin in ... Infusions of 10% IVIg at doses up to 2.0 g/kg with high infusion rates were safe and well tolerated in patients with CIDP.

8. panzyga.pfizerpro.com panzyga.pfizerpro.com/efficacy/pi-study-designs

ifas, 10% solution) | PI Study Design A 1-year, prospective, open-label, noncontrolled, nonrandomized, multicenter, phase 3 study evaluated the efficacy, safety, and tolerability of PANZYGA in 51 ...