What side effects are associated with this type of intervention?

Treatments for Protein-Losing Enteropathy (PLE) that target complement component C5, such as pozelimab and eculizumab, commonly have side effects including increased susceptibility to infections, particularly meningococcal infections, due to complement system inhibition. Other potential side effects include infusion reactions, headache, hypertension, and gastrointestinal symptoms. The immunosuppressive nature of these therapies also raises the risk of other opportunistic infections.

What prior FDA approvals exist?

Eculizumab, a monoclonal antibody that inhibits the cleavage of complement component 5 (C5), has been FDA-approved for conditions involving complement-mediated damage, such as Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS). While not specifically approved for Protein-Losing Enteropathy (PLE), its mechanism of action is similar to pozelimab, which is currently being studied for CD55-deficient PLE. Another related drug is ravulizumab, a longer-acting C5 inhibitor, approved for PNH and aHUS, offering extended dosing intervals.

What other types of research exist?

As of 2024, promising alternatives to Pozelimab for Protein-Losing Enteropathy (PLE) patients include Eculizumab, another monoclonal antibody targeting C5, and Ravulizumab, a long-acting C5 inhibitor. Both have shown efficacy in complement-mediated diseases and could be considered for PLE treatment.

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Monoclonal Antibodies

Pozelimab for Protein-Losing Enteropathy

Phase 2 & 3

Waitlist Available

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing pozelimab, a medication that targets specific proteins, in patients with CD55-deficient PLE, a rare condition causing severe protein loss from the intestines. The goal is to see if pozelimab can reduce symptoms and improve health by blocking a problematic protein.

Who is the study for?

This trial is for patients with a condition called CD55-deficient Protein-Losing Enteropathy (CHAPLE Disease). Participants must have a clinical diagnosis confirmed by genetic testing and either be currently experiencing symptoms or on eculizumab therapy but willing to switch to pozelimab.

What is being tested?

The study aims to test the effectiveness and safety of a drug named Pozelimab in treating CHAPLE Disease. It will look at how well it manages symptoms, improves quality of life, affects lab values like albumin levels, reduces hospital stays, and impacts growth among other factors.

What are the potential side effects?

While specific side effects are not listed here, the trial will monitor how patients tolerate Pozelimab. This includes any adverse reactions they might have during treatment as well as changes in their overall health.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with CD55-deficient PLE/CHAPLE disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24

Secondary study objectives

Absolute Value of Total Albumin at Specified Timepoints

Absolute Values of Total Ferritin at Baseline and Week 24

Absolute Values of Total Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24

+61 more

Side effects data

From 2024 Phase 2 & 3 trial • 10 Patients • NCT04209634

20%

Rhinitis

20%

Iron deficiency

20%

Urticaria

20%

Pyrexia

10%

Vomiting

10%

Dehydration

10%

Acarodermatitis

10%

Nasopharyngitis

10%

Tonsillitis

10%

Hypokalaemia

10%

Metabolic acidosis

10%

Alopecia

10%

Alopecia areata

10%

Dermatitis contact

10%

Abdominal pain

10%

Constipation

10%

Gingival bleeding

10%

Blood glucose increased

10%

Blood uric acid increased

10%

Hepatic enzyme increased

10%

Anaemia folate deficiency

10%

Immunisation reaction

10%

Headache

10%

Haematuria

10%

Proteinuria

100%

80%

60%

40%

20%

Study treatment Arm

Pozelimab

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Active PLEExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pozelimab

2020

Completed Phase 3

~130

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Protein-Losing Enteropathy (PLE) often involve targeting the complement system to reduce inflammation and tissue damage. Pozelimab, a monoclonal antibody, inhibits the cleavage of complement component C5 into C5a and C5b, thereby preventing the activation of the complement pathway. This is significant for PLE patients as it helps to control the excessive immune response that contributes to the disease, potentially improving symptoms and reducing the need for other medications or hospitalizations.

Pharmacokinetics and pharmacodynamics of pozelimab alone or in combination with cemdisiran in non-human primates.Inhibition of complement pathway activation with Pozelimab, a fully human antibody to complement component C5.Characterizing the original anti-C5 function-blocking antibody, BB5.1, for species specificity, mode of action and interactions with C5.