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

CD55-Deficient Protein-Losing Enteropathy Clinical Trial

— CHAPLE Disease  

Official title:

An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT06003881
• Other study ID #: R3918-PLE-21110
• Status: Available

Study information

• Verified date: August 2023
• Source: Regeneron Pharmaceuticals
• Contact: Requests for compassionate use must be initiated by a treating p
• Phone: +44(0) 1932 824 100
• Email: medicineaccess[@]clinigengroup.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 1 Year and older

Eligibility

Key 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)

Key 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 NOTE: Other protocol defined inclusion / exclusion criteria apply

Related Conditions & MeSH terms

• CD55-Deficient Protein-Losing Enteropathy
• Intestinal Diseases
• Protein-Losing Enteropathies

Intervention

Drug:
• Pozelimab
Subcutaneous (SC) administration

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Regeneron Pharmaceuticals