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

Octapharma

Status and phase: Enrolling; Phase 3

Conditions: Pediatric Chronic Inflammatory Demyelinating Polyneuropathy

Treatments: Drug: Panzyga

Study type: Interventional

Funder types: Industry

Identifiers

NCT04929236

NGAM-11

Details and patient eligibility

About

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

Enrollment: 30 estimated patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age ≥2 years and ≤17 years.
2. Patients with a diagnosis of definite or possible CIDP based on European Neuromuscular Center (ENMC) criteria
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.
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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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30 participants in 2 patient groups

Panzyga High Dose

Experimental group

Description:

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

Treatment:

Drug: Panzyga

Panzyga Low Dose

Experimental group

Description:

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

Treatment:

Drug: Panzyga