NCT07142343 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
| NCT ID | NCT07142343 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Regeneron Pharmaceuticals |
| Condition | CHAPLE Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 5 participants |
| Start Date | 2026-03-19 |
| Primary Completion | 2030-02-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 5 participants in total. It began in 2026-03-19 with a primary completion date of 2030-02-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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
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 meningococcal vaccinations required per protocol. 3. Parent(s)/legal guardian(s) are willing to provide written informed consent 4. Participant has active CHAPLE disease as described in the protocol Key Exclusion Criteria: 1. History of meningococcal infection 2. No documented meningococcal quadrivalent (serotype ACWY) vaccination prior to screening and participant's parent(s)/legal guardian(s) are unwilling for participant to undergo vaccination during the study as described in the protocol 3. No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening as described in the protocol 4. Prior treatment with a complement inhibitor as described in the protocol 5. Presence of a concomitant disease that leads to hypoproteinemia or secondary intestinal lymphangiectasia as described in the protocol Note: Other Protocol Defined Inclusion/ Exclusion Criteria Apply
Contact & Investigator
Clinical Trial Management
STUDY DIRECTOR
Regeneron Pharmaceuticals
Frequently Asked Questions
Who can join the NCT07142343 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 5 Years, studying CHAPLE Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07142343 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT07142343 currently recruiting?
Yes, NCT07142343 is actively recruiting participants. Contact the research team at clinicaltrials@regeneron.com for enrollment information.
Where is the NCT07142343 trial being conducted?
This trial is being conducted at Istanbul, Turkey (Türkiye).
Who is sponsoring the NCT07142343 clinical trial?
NCT07142343 is sponsored by Regeneron Pharmaceuticals. The principal investigator is Clinical Trial Management at Regeneron Pharmaceuticals. The trial plans to enroll 5 participants.