CD55-Deficient Protein-Losing EnteropathyCHAPLE DiseaseNCT06003881
Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
A study testing Pozelimab in people with CD55-Deficient Protein-Losing Enteropathy, run by Regeneron Pharmaceuticals.
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. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- CD55-Deficient Protein-Losing Enteropathy
- What is being tested?
- Pozelimab
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
About this study
Official title: An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
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.
- Being tested
- Pozelimab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PozelimabDrug
Also called REGN3918
Subcutaneous (SC) administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CD55-Deficient Protein-Losing EnteropathyStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06003881
- Sponsor's protocol ID
- R3918-PLE-21110
- Acronym
- CHAPLE Disease
- Status last verified
- Oct 2025
- First submitted
- 14 Aug 2023
- Last updated
- 10 Nov 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
- ClinicalTrials.gov ID
- NCT06003881
- Acronym / sponsor ID
- CHAPLE Disease / R3918-PLE-21110
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 22 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PozelimabAlso: REGN3918Subcutaneous (SC) administration | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2025.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Pozelimab in people with CD55-Deficient Protein-Losing Enteropathy, run by Regeneron Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2025; AzTrials last refreshed its copy on 8 Nov 2026.