Trial Now Recruiting: A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease (NCT07142343)

WHY IT MATTERS

This is the first safety trial of pozelimab in very young children (ages 1-5) with CHAPLE disease, offering families of affected toddlers a potential treatment option when few alternatives exist for this life-threatening condition.

Researchers are testing a new medicine called pozelimab in young children ages 1 to 5 who have CHAPLE disease, a rare inherited condition that affects the stomach, intestines, heart, and blood vessels. This study is checking whether the medicine is safe and well-tolerated in this young age group. CHAPLE disease can cause serious symptoms, so finding safe treatments for young children is important.

NCT ID: NCT07142343 Status: RECRUITING Conditions: CHAPLE Disease Phase: PHASE4 Enrollment: 5 Sponsor: Regeneron Pharmaceuticals Summary: This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug. The study is focused on young children 1 to 5 years of age, who have CHAPLE disease. CHAPLE is a very rare hereditary disease that can cause potentially life-threatening symptoms related to the stomach and intestines (gastrointestinal symptoms), and symptoms related to the heart and blood vessels (cardiovascular symptoms). The study is

YOU CAN ACT ON THIS

If your child has CHAPLE disease and is between 1-5 years old, contact Regeneron Pharmaceuticals or your doctor immediately to ask about enrollment eligibility for trial NCT07142343, as only 5 participants are being recruited.