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

NCT04929236 | Recruiting Phase 3 FDA Drug

• 1 other identifier: NGAM-11
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 6

Brief Summary

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

Conditions

Pediatric Chronic Inflammatory Demyelinating Polyneuropathy

Outcome Measures

Primary Outcomes (1)

• Change in CIDP Baseline

  Evaluate the efficacy of two PANZYGA dose regimens in pediatric CIDP patients based on change in CIDP symptoms, measured by the Modified Rankin Score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.

  Up to 24 weeks

Secondary Outcomes (3)

• CIDP Relapse

  Up to 24 weeks

• Time to CIDP Relapse

  Up to 24 weeks

• Percentage of Patients With Good/Excellent Response

  Up to 24 weeks

Study Arms (2)

Panzyga High Dose

EXPERIMENTAL

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

Drug: Panzyga

Panzyga Low Dose

EXPERIMENTAL

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

Drug: Panzyga

Interventions

Panzyga DRUG

PANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.

Panzyga High Dose; Panzyga Low Dose

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age ≥2 years and ≤17 years.
• Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone \[1\]
• Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
• 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).

You may not qualify if:

• Patients with previously diagnosed CIDP who lack any CIDP symptoms.
• Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
• Patients who have previously failed immunoglobulin therapy for CIDP.
• Patients who received immunoglobulin or plasma exchange (PEX) within eight weeks prior to the Baseline Visit (washout phase). However, if a patient has clinical evidence of confirmed CIDP relapse during the washout phase (consistent with an increase in mRS of ≥1), they are eligible for trial enrolment.
• Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
• Patients on unstable (change in prescribed dose within the last eight weeks) corticosteroids or rituximab use.
• Patients with known or suspected hypersensitivity, anaphylaxis, or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
• Female patients who are breastfeeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method while on the study (acceptable methods of birth control for this study include: intrauterine device \[IUD\], hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap).
• Presence of medical history information or clinical symptoms suggestive of human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.
• Severe liver and/or kidney disease (alanine aminotransferase \[ALT\] \> 3 × upper limit of normal \[ULN\]; aspartate aminotransferase \[AST\] \> 3 × ULN; and/or creatinine levels \>44 µmol/L for children ages 2-3 years, \>62 µmol/L for children ages 4-10 years, and \>89 µmol/L for children ages 11-17 years.
• Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
• History of alcohol or drug abuse in the previous year, per Investigator's opinion.
• Unable or unwilling to comply with the study protocol.
• Receipt of any other investigational medicinal product (IMP) within three months before study entry or participating in another interventional clinical study. Prior participation in an observational or open-label study involving an approved product may be allowed but require prior consultation with the Medical Monitor to assess eligibilty.
• 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.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (6)

Octapharma Research Site

Birmingham, Alabama, 35233, United States

RECRUITING

Octapharma Research Site

Orange, California, 92868, United States

RECRUITING

Octapharma Research Site

Louisville, Kentucky, 40202, United States

RECRUITING

Octapharma Research Site

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Octapharma Research Site

Houston, Texas, 77030, United States

RECRUITING

Octapharma Research Site

Charlottesville, Virginia, 22908, United States

RECRUITING

MeSH Terms

Interventions

Panzyga

Central Study Contacts

Patrick Murphy

CONTACT

866-337-1868; ctgov@clinicalresearchmgt.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-Blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2021
• First Posted: June 18, 2021
• Study Start: October 1, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: June 6, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)