Evaluating different doses of Panzyga for children with CIDP

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

This study is testing different doses of Panzyga to see which one works best for children aged 2 to 17 with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP).

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Octapharma (Industry-sponsored)
• Drugs / interventions: rituximab
• Locations: 6 sites (Birmingham, Alabama and 5 other locations)
• Trial ID: NCT04929236 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and efficacy of various dose regimens of Panzyga in pediatric patients diagnosed with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). The study will include children aged 2 to 17 years who exhibit functional impairment due to CIDP. Participants will receive different doses of Panzyga, and their responses will be monitored to determine the most effective regimen. The trial follows a structured approach to ensure that the results are reliable and can inform future treatment options for this condition.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 2 to 17 years with a confirmed diagnosis of CIDP and functional impairment.

Not a fit: Patients who have previously failed immunoglobulin therapy for CIDP or those without current CIDP symptoms may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more effective treatment option for children suffering from CIDP, potentially improving their quality of life.

How similar studies have performed: While this approach is being evaluated in this specific context, similar studies have shown promise in treating CIDP with immunoglobulin therapies.

Eligibility criteria

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.

Where this trial is running

Birmingham, Alabama and 5 other locations

• Octapharma Research Site — Birmingham, Alabama, United States (Recruiting)
• Octapharma Research Site — Orange, California, United States (Recruiting)
• Octapharma Research Site — Louisville, Kentucky, United States (Recruiting)
• Octapharma Research Site — Philadelphia, Pennsylvania, United States (Recruiting)
• Octapharma Research Site — Houston, Texas, United States (Recruiting)
• Octapharma Research Site — Charlottesville, Virginia, United States (Recruiting)

Study contacts

• Study coordinator: Patrick Murphy

Conditions Pediatric Chronic Inflammatory Demyelinating Polyneuropathy