Actively 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
Led by Regeneron Pharmaceuticals · Updated on 2026-04-13
5
Participants Needed
1
Research Sites
203 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
- Parent(s)/legal guardian(s) willing and able to follow clinic visits and study procedures
- Parent(s)/legal guardian(s) willing to give written informed consent
- Participant has active CHAPLE disease as described in the protocol
You will not qualify if you...
- History of meningococcal infection
- No documented meningococcal quadrivalent (serotype ACWY) vaccination before screening and unwillingness to vaccinate during the study
- No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local guidelines
- Prior treatment with a complement inhibitor as described in the protocol
- Presence of another disease causing hypoproteinemia or secondary intestinal lymphangiectasia as described in the protocol
- Other protocol-defined exclusion criteria apply
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Marmara University Hospital
Istanbul, Turkey (Türkiye), 10 34899
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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