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Clinical Trial Details — Status: Available

Administrative data

NCT number NCT06003881
Other study ID # R3918-PLE-21110
Secondary ID
Status Available
Phase
First received
Last updated

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
Health authority
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
Est. completion date
Est. primary completion date
Accepts healthy volunteers
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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Pozelimab
Subcutaneous (SC) administration

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Regeneron Pharmaceuticals
See also
  Status Clinical Trial Phase
Completed NCT04209634 - Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) Phase 2/Phase 3