Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Regeneron Pharmaceuticals

Status and phase

Completed

Phase 3

Phase 2

Conditions

CHAPLE

CD55-deficient Protein-losing Enteropathy

Treatments

Drug: Pozelimab

Study type

Interventional

Funder types

Industry

Identifiers

R3918-PLE-1878

Details and patient eligibility

About

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

Enrollment

10 patients

Sex

All

Ages

1+ year old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

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.