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

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT07142343
Phase
PHASE4
Status
Recruiting
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Conditions

  • CHAPLE Disease

Eligibility Criteria

Sex
ALL
Age
1 Year - 5 Years
Healthy Volunteers
Not accepted

Interventions

  • Pozelimab — DRUG
    Administered per the protocol

Study Details

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 also looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the study drug blocks Complement 5 (C5) in the body * Whether the study drug changes the level of a substance called CH50 measured in the blood * Whether the study drug changes the levels of albumin and other proteins * Whether the body makes antibodies against study drug, which could make the study drug less effective or could lead to side effects

Key Dates

Start date
Mar 19, 2026
Status verified
Apr 2026
Primary completion
Feb 12, 2030
Completion
Feb 12, 2030

Study Design

Enrollment
5 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Active CHAPLE

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Event (TEAEs) [ Time Frame: Through week 52 ]

Central Contacts