Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
About the study
The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).
The secondary objectives of the study are:
To evaluate the safety and tolerability of pozelimab in patients with CD55-deficient PLE disease
To evaluate the effect of pozelimab on CD55-deficient PLE (both patients with active disease at baseline and those with inactive disease on eculizumab, switching to pozelimab)
To determine the effects of pozelimab on albumin and other serum proteins (total protein, immunoglobulins)
To determine the effects of pozelimab on ascites
To determine the effects of pozelimab on stool consistency
To determine the effect of pozelimab on health-related quality of life
To determine the effect of pozelimab on lab abnormalities observed in CD55-deficient PLE such as hypertriglyceridemia, thrombocytosis, and hypovitaminosis B12
To describe the effects of pozelimab on the sparing of concomitant medications and reduction in hospitalization days
To determine the effects of pozelimab on growth
To characterize the concentration of pozelimab in patients with CD55-deficient PLE
To assess the incidence of treatment-emergent ADA for pozelimab in patients with CD55-deficient PLE disease
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Key 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
- Active disease as defined by the protocol or inactive disease on eculizumab therapy (and whose treating physician has the expectation of future access to renewed eculizumab treatment should this be required), and is willing to discontinue eculizumab during screening and start pozelimab at baseline with no eculizumab wash-out
EXCLUSION CRITERIA
Key Exclusion Criteria:
- History of meningococcal infection
- No documented meningococcal vaccination within 3 years prior to screening and patient unwilling to undergo vaccination during the study
- No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening and patient unwilling to undergo vaccination during the study if required per local practice or guidelines
- Presence of a concomitant disease that leads to hypoproteinemia at the time of starting pozelimab
- A concomitant disease that leads to secondary intestinal lymphangiectasia such as a fontan procedure for congenital heart disease
Note: Other protocol-defined Inclusion/Exclusion criteria apply.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
CD55-deficient Protein-losing Enteropathy,CHAPLE
Age (in years)
1+
Phase
Phase 2/Phase 3
Participants needed
10
Est. Completion Date
May 2, 2024
Treatment type
Interventional
Sponsor
Regeneron Pharmaceuticals
ClinicalTrials.gov identifier
NCT04209634
Study number
R3918-PLE-1878
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is a clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?