Ignite Creation Date:
2024-05-06 @ 2:04 PM
Last Modification Date:
2024-10-26 @ 1:24 PM
Study NCT ID:
NCT04209634
Status:
COMPLETED
Last Update Posted:
2024-05-13
First Post:
2019-12-20
Brief Title:
Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy CHAPLE Disease
Sponsor:
Regeneron Pharmaceuticals
Organization:
Regeneron Pharmaceuticals
Study Overview
Official Title:
An Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy CHAPLE Disease
Status:
COMPLETED
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None