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Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Primary Purpose

CD55-Deficient Protein-Losing Enteropathy

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Pozelimab
Sponsored by
Regeneron Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for CD55-Deficient Protein-Losing Enteropathy

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All Sexes

Key Inclusion Criteria: 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 Written informed consent from parent/guardian for minor patients 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: Patients who discontinued the prior pozelimab study due to safety or lack of efficacy 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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 14, 2023
    Last Updated
    August 14, 2023
    Sponsor
    Regeneron Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06003881
    Brief Title
    Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
    Acronym
    CHAPLE Disease
    Official Title
    An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Regeneron Pharmaceuticals

    4. Oversight

    5. Study Description

    Brief Summary
    The program is to provide access to an experimental drug called pozelimab and to document the long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE). CD55-deficient PLE/CHAPLE disease is a rare inherited disease of the immune system. This can be a life-threatening condition that is usually found when patients are children.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    CD55-Deficient Protein-Losing Enteropathy

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Pozelimab
    Other Intervention Name(s)
    REGN3918
    Intervention Description
    Subcutaneous (SC) administration

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Year
    Eligibility Criteria
    Key Inclusion Criteria: 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 Written informed consent from parent/guardian for minor patients 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: Patients who discontinued the prior pozelimab study due to safety or lack of efficacy 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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Requests for compassionate use must be initiated by a treating physician Physicians with questions about access can contact Clinigen
    Phone
    +44(0) 1932 824 100
    Email
    medicineaccess@clinigengroup.com

    12. IPD Sharing Statement

    Learn more about this trial

    Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy

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