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

Multicenter, Prospective, Double-Blinded, Parallel Group, Randomized Phase III Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

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

Study Overview

• Status: Recruiting
• Conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy
• Intervention / Treatment: Drug: Panzyga

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Patrick Murphy; Phone Number: 866-337-1868; Email: ctgov@clinicalresearchmgt.com

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Recruiting
      • Octapharma Research Site
  • California
    • Orange, California, United States, 92868
      • Recruiting
      • Octapharma Research Site
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Recruiting
      • Octapharma Research Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Octapharma Research Site
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Octapharma Research Site
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • Recruiting
      • Octapharma Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥2 years and ≤17 years.
2. Patients with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EANPNS) 2021 guidelone [1]
3. Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.
4. 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).

Exclusion Criteria:

1. Patients with previously diagnosed CIDP who lack any CIDP symptoms.
2. Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.
3. Patients who have previously failed immunoglobulin therapy for CIDP.
4. 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.
5. Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.
6. Patients on unstable (change in prescribed dose within the last eight weeks) corticosteroids or rituximab use.
7. Patients with known or suspected hypersensitivity, anaphylaxis, or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.
8. 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).
9. 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.
10. 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.
11. Presence of medical history information or clinical symptoms suggestive of immunoglobulin (IgA) deficiency and antibodies against IgA.
12. History of alcohol or drug abuse in the previous year, per Investigator's opinion.
13. Unable or unwilling to comply with the study protocol.
14. 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.
15. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Panzyga High Dose; 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 is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.
Experimental: Panzyga Low Dose; 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; PANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to CIDP Relapse	Time to CIDP relapse or withdrawal for any other reason with with relapse defined as increase in Modified Rankin Score score by ≥1 point from the baseline score. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.	Up to 24 weeks
Percentage of Patients With Good/Excellent Response	The percentage of patients with good/excellent response, defined by a Modified Rankin Score score of 0 or 1 in each arm at Week 24. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.	Up to 24 weeks
CIDP Relapse	Evaluate percentage of patients with CIDP relapse between 2 doses of Panzyga with relapse defined as increase in Modified Rankin score by ≥1 point from the baseline score related to CIDP. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6.	Up to 24 weeks

Collaborators and Investigators

• Sponsor: Octapharma

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: June 11, 2021
• First Submitted That Met QC Criteria: June 11, 2021
• First Posted (Actual): June 18, 2021

Study Record Updates

• Last Update Posted (Actual): June 6, 2025
• Last Update Submitted That Met QC Criteria: June 3, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Polyradiculoneuropathy
• Other Study ID Numbers: NGAM-11

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No