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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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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.

Detailed Description

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Conditions

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CD55-Deficient Protein-Losing Enteropathy

Interventions

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Pozelimab

Subcutaneous (SC) administration

Intervention Type DRUG

Other Intervention Names

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REGN3918

Eligibility Criteria

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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)

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

Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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R3918-PLE-21110

Identifier Type: -

Identifier Source: org_study_id