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
Recruiting
Phase 4
Interventional Study
CHAPLE Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 1 – 5
- Sex
- Any
- Trial phase
- Phase 4
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 5 (estimated)
- Sponsor
- Regeneron Pharmaceuticals · Industry
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About This Trial
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 sym…
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
2. Parent(s)/legal guardian(s) are willing and able to comply with participant's clinic visits and study-related procedures, including participant's completion of the full series of meningoco…
Contacts