Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients
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).
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.
Parallel
Panzyga
DrugPANZYGA is a human immunoglobin solution with 10% protein content for intravenous (IV) administration.
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.
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.
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.
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.
