Pozelimab safety in children 1 to 5 with CHAPLE disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 4 interventional study administers pozelimab to young children with genetically confirmed CD55-deficient CHAPLE disease to monitor safety and tolerability. Participants attend regular clinic visits for blood draws to measure drug levels, complement activity (C5 blockade and CH50), albumin and other protein levels, and anti-drug antibodies, while investigators record adverse events. Enrollment requires documented or protocol-administered meningococcal vaccination and, where applicable, Haemophilus influenzae and pneumococcal vaccination before dosing. The trial is sponsored by Regeneron and conducted at Marmara University Hospital in Istanbul.

Why it matters

Who can join: the full rules

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

Where it's running

• Marmara University Hospital
  Istanbul, Turkey (Türkiye) Recruiting

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.