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

Conditions

• CD55-deficient Protein-losing Enteropathy
• CHAPLE

Eligibility Criteria

Sex

ALL

Age

1 Year - N/A

Healthy Volunteers

Not accepted

Interventions

• Pozelimab — DRUG
  Single loading intravenous (IV) dose on day 1, then fixed doses sub-cutaneous (SC) (based on body weight) QW (±2 days) over the treatment period.

Study Details

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

Key Dates

Start date

Jan 27, 2020

Status verified

Aug 2025

Primary completion

Nov 9, 2021

Completion

May 2, 2024

Study Design

Enrollment

10 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Active PLE
  Patients aged 1 year and older with a clinical diagnosis of CD55-deficient PLE disease

Primary Outcome Measure

Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24 [ Time Frame: At Week 24 ]

Locations (1)

Find similar trials in Bethesda, MD