Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy

NCT06003881 | Unknown

• 1 other identifier: R3918-PLE-21110
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The program is to provide access to an experimental drug called pozelimab and to document the long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE). CD55-deficient PLE/CHAPLE disease is a rare inherited disease of the immune system. This can be a life-threatening condition that is usually found when patients are children.

Conditions

CD55-Deficient Protein-Losing Enteropathy

Interventions

Pozelimab DRUG

Subcutaneous (SC) administration

Also known as: REGN3918

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of CD55-deficient PLE/CHAPLE disease (based on a history of PLE), confirmed by biallelic CD55 loss-of-function mutation detected by genotype analysis (frameshift, nonsense mutations) as defined in the protocol
• Written informed consent from parent/guardian for minor patients
• Written assent from minor patients as appropriate (eg, above the age of 6 years or the applicable age per local regulatory requirements)

You may not qualify if:

• Patients who discontinued the prior pozelimab study due to safety or lack of efficacy
• Considered by the treating physician as inappropriate for this program for any reason as defined in the protocol

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2023
• First Posted: August 22, 2023
• Last Updated: November 10, 2025

Record last verified: 2025-10