Status:
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
Lead Sponsor:
Regeneron Pharmaceuticals
Conditions:
CHAPLE Disease
Eligibility:
All Genders
1-5 years
Phase:
PHASE4
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...
Eligibility Criteria
Inclusion
- Key
- Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
- 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.
- Parent(s)/legal guardian(s) are willing to provide written informed consent
- Participant has active CHAPLE disease as described in the protocol
- Key
Exclusion
- History of meningococcal infection
- 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
- 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
- Prior treatment with a complement inhibitor as described in the protocol
- 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
Key Trial Info
Start Date :
December 23 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 24 2029
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT07142343
Start Date
December 23 2025
End Date
August 24 2029
Last Update
November 12 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Marmara University Hospital
Istanbul, Turkey (Türkiye), 10 34899