Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug hopes to tame rare Gut-Heart disease in toddlers

NCT ID NCT07142343

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 study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called pozelimab in children aged 1 to 5 who have CHAPLE disease, a very rare genetic condition that causes severe stomach and heart problems. The main goal is to see if the drug is safe and how it affects the body. Only 5 children will take part, and the drug works by blocking a part of the immune system to control symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pozelimab (a lab-made antibody that blocks a part of the immune system called C5)
What this could lead to
If it works, this could provide a treatment option for young children with CHAPLE disease, helping control life-threatening gastrointestinal and cardiovascular symptoms.
What could go wrong
This is a very small, early-phase study with only 5 children, so results may not apply to everyone. The drug blocks part of the immune system, which could increase infection risk.

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.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 5 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2026

Expected to finish

Feb 2030

An estimate. End dates often move.

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 to 5 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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 CHAPLE disease as described in the protocol 2. Parent(s)/legal guardian(s) are willing and able to comply with participant's clinic visits and study-related procedures, including participant's completion of the full series of meningococcal vaccinations required per protocol. 3. Parent(s)/legal guardian(s) are willing to provide written informed consent 4. Participant has active CHAPLE disease as described in the protocol Key Exclusion Criteria: 1. History of meningococcal infection 2. No documented meningococcal quadrivalent (serotype ACWY) vaccination prior to screening and participant's parent(s)/legal guardian(s) are unwilling for participant to undergo vaccination during the study as described in the protocol 3. No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening as described in the protocol 4. Prior treatment with a complement inhibitor as described in the protocol 5. Presence of a concomitant disease that leads to hypoproteinemia or secondary intestinal lymphangiectasia as described in the protocol Note: Other Protocol Defined Inclusion/ Exclusion Criteria Apply

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Chaple disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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.

Chaple disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Marmara University Hospital

    RECRUITING

    Istanbul, 10 34899, Turkey (Türkiye)