Objective:

This phase III study aims to evaluate the safety and efficacy of different IVIg 10% dose regimens in pediatric CIDP patients.

Background:

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an acquired chronic progressive or relapsing-remitting neuropathy. CIDP can be diagnosed throughout life, from early childhood (<1 year) to old age (>80 years). Over the last 20 years, high-dose IVIG has become an effective and safe therapeutic option for CIDP in adults. To date, there are no FDA-approved medications for pediatric patients with CIDP and this organization hypothesizes that IVIg could prove to be beneficial in this population.

Design/Methods:

This is a multicenter, prospective, double-blinded, parallel-group, randomized phase III study to evaluate safety and efficacy of different IVIg 10% (PANZYGA^®) dose regimens in pediatric CIDP patients. The primary objective is to evaluate the efficacy of two IVIg dose regimens in pediatric CIDP patients based on the percentage of patients showing improvement. Approximately 30 patients age ≥2 years and ≤17 years, with a diagnosis of CIDP based on European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021 criteria will be randomized in a 1:1 ratio to receive 1.0g/kg or 2.0g/kg of IVIg 10%. Patients will receive 6 total infusions administered every 4 weeks. 

Results:

The primary efficacy endpoint will be the percentage of patients with CIDP improvement at week 24, defined as a decrease in mRS of ≥1 point from the baseline score. Secondary outcomes include the percentage of patients with CIDP relapse (increase in mRS score by ≥1 point from the baseline score related to CIDP), time to CIDP relapse, and the percentage of patients with good or excellent response (mRS score of 0 or 1 in each arm at Week 24). 

Conclusions: