Menu
Recruiting NCT07142343

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

Phase IV Interventional CHAPLE Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Pozelimab.
Who it may be relevant to
Registry conditions: CHAPLE Disease. Basic parameters: 1 year — 5 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Turkey (Türkiye)
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label, Single-Arm Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Pozelimab in Pediatric Patients 1 to 5 Years of Age With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Overview

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

Interventions

  • Drug Pozelimab
    Administered per the protocol

Primary outcome measures

  • Incidence of Treatment-Emergent Adverse Event (TEAEs) [Time frame: Through week 52]
  • Severity of TEAEs [Time frame: Through week 52]
Secondary outcome measures (12)
  • Concentrations of total pozelimab [Time frame: Through week 52]
  • Concentrations of total C5 in serum [Time frame: Through week 52]
  • Absolute change from baseline of albumin concentration in serum [Time frame: Through week 52]
  • Percent change from baseline of albumin concentration in serum [Time frame: Through week 52]
  • Absolute change from baseline of total protein [Time frame: Through week 52]
  • Percent change from baseline of total protein [Time frame: Through week 52]
  • Absolute change from baseline of total Immunoglobulin G (IgG) [Time frame: Through week 52]
  • Percent change from baseline of total IgG [Time frame: Through week 52]
  • Incidence of Anti-Drug Antibody (ADA) to pozelimab over time [Time frame: Through week 52]
  • Titer of ADA to pozelimab over time [Time frame: Through week 52]
  • Change from baseline of total complement activity CH50 over time [Time frame: Through week 52]
  • Percent change from baseline of CH50 over time [Time frame: Through week 52]

Eligibility criteria

Inclusion criteria

  • Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
  • 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.
  • Parent(s)/legal guardian(s) are willing to provide written informed consent
  • Participant has active CHAPLE disease as described in the protocol

Exclusion criteria

  • History of meningococcal infection
  • 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
  • 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
  • Prior treatment with a complement inhibitor as described in the protocol
  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Turkey (Türkiye) · 1 center
  • Marmara University Hospital — Istanbul

Identifiers

NCT: NCT07142343 · R3918-PLE-2399

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗