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A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

NCT07142343 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-04-13

No results posted yet for this study

Summary

This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug.

The study is focused on young children 1 to 5 years of age, who have CHAPLE disease. CHAPLE is a very rare hereditary disease that can cause potentially life-threatening symptoms related to the stomach and intestines (gastrointestinal symptoms), and symptoms related to the heart and blood vessels (cardiovascular symptoms).

The study is also looking at several other research questions, including:

* What side effects may happen from taking the study drug
* How much study drug is in the blood at different times
* Whether the study drug blocks Complement 5 (C5) in the body
* Whether the study drug changes the level of a substance called CH50 measured in the blood
* Whether the study drug changes the levels of albumin and other proteins
* Whether the body makes antibodies against study drug, which could make the study drug less effective or could lead to side effects

Conditions

  • CHAPLE Disease

Interventions

DRUG

Pozelimab

Administered per the protocol

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-19
Primary Completion
2030-02-12
Completion
2030-02-12
FDA Drug
Yes

Countries

  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07142343 on ClinicalTrials.gov