Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
- Conditions
- CD55-Deficient Protein-Losing Enteropathy
- Registration Number
- NCT06003881
- Lead Sponsor
- Regeneron Pharmaceuticals
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
- 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)
Key
Exclusion Criteria
- 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
NOTE: Other protocol defined inclusion / exclusion criteria apply
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method