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Lifesaving drug now available for kids with rare gut disease

NCT ID NCT06003881

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved This study
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program gives patients with CHAPLE disease, a rare and life-threatening immune disorder that causes protein loss from the gut, access to the experimental drug pozelimab. The goal is to provide treatment and monitor long-term safety. Participants must have a confirmed genetic diagnosis of CD55-deficient protein-losing enteropathy.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year and older

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. 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 (frameshift, nonsense mutations) as defined in the protocol 2. Written informed consent from parent/guardian for minor patients 3. Written assent from minor patients as appropriate (eg, above the age of 6 years or the applicable age per local regulatory requirements) Key Exclusion Criteria: 1. Patients who discontinued the prior pozelimab study due to safety or lack of efficacy 2. Considered by the treating physician as inappropriate for this program for any reason as defined in the protocol NOTE: Other protocol defined inclusion / exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

protein-losing enteropathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CD55-deficient protein-losing enteropathy

More trials for these conditions

Other studies related to the condition(s) this trial covers.