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

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

Official Title
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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

Conditions
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Pediatric Chronic Inflammatory Demyelinating Polyneuropathy

Intervention / Treatment
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* Drug: Panzyga


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age ≥2 years and ≤17 years. Patients with a diagnosis of definite or possible CIDP based on European Neuromuscular Center (ENMC) criteria 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). Exclusion Criteria: 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. 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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

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

What is the study measuring?
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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 CIPD 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 |
| --- | --- | --- |
| 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. The Modified Rankin Score (mRS) is a 6 point disability scale with possible scores ranging from 0 to 6. | Up to 24 weeks | 
| 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 |