Abstract Details

Title Evaluating the Safety and Efficacy of Different Intravenous Immunoglobulin 10 Percent Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 9-008

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

Finding safe and effective doses of intravenous immunoglobulin 10% for CIDP in the pediatric population can improve their quality of life.

Authors/Disclosures
Julian Thomas, MD PRESENTER	Dr. Thomas has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Scholar Rock. Dr. Thomas has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Thomas has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catalyst. Dr. Thomas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ITF Therapeutics. Dr. Thomas has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Thomas has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen.
Syed Rizvi	Syed Rizvi has nothing to disclose.
Huub Kreuwel	Huub Kreuwel has received personal compensation for serving as an employee of Octapharma.

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