Paroxysmal Nocturnal HemoglobinuriaNCT06028594
Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
A study testing Pozelimab and Cemdisiran in people with Paroxysmal Nocturnal Hemoglobinuria, run by Regeneron Pharmaceuticals.
Trial summary
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in your body to break apart. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Pozelimab and Cemdisiran
- 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 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- Patients who have completed either the end of treatment visit of the open-label treatment period or open-label extension period in one of the following parent studies:
- An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (R3918-PNH-2050 [NCT05744921])
- A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy (R3918-PNH-2092 [NCT04811716])
- A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from Eculizumab Therapy (R3918-PNH-20105 [NCT04888507]). Note: In some countries, patients that would normally enter into the R3918-PNH-2050 (NCT05744921) parent study before entering this expanded access program, may be given the opportunity to skip R3918-PNH-2050 (NCT05744921) and go straight into this expanded access program, with Regeneron's permission
- Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission
- Willing and able to comply with clinic visits and related standard-of-care procedures
- With Regeneron's permission, patients who have been enrolled in other PNH studies as appropriate
Who cannot join
- Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician
- Any new condition or worsening of an existing condition which, in the opinion of the treating physician, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the program
- NOTE: Other protocol defined inclusion / exclusion criteria apply
About this study
Official title: An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in your body to break apart.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Pozelimab, Cemdisiran
- Medical subject headings
- Hemoglobinuria, Paroxysmal
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
CemdisiranDrug
Also called ALN-CC5
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 posted8 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies 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
- NCT06028594
- Sponsor's protocol ID
- R3918-PNH-2238
- Status last verified
- Oct 2025
- First submitted
- 31 Aug 2023
- Last updated
- 20 Oct 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT06028594
- Acronym / sponsor ID
- R3918-PNH-2238
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 8 Sep 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.
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in your body to break apart.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients who have completed either the end of treatment visit of the open-label treatment period or open-label extension period in one of the following parent studies:
- Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission
- Willing and able to comply with clinic visits and related standard-of-care procedures
- With Regeneron's permission, patients who have been enrolled in other PNH studies as appropriate
Key exclusion criteria (3 listed)
- Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician
- Any new condition or worsening of an existing condition which, in the opinion of the treating physician, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the program
- 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 | – |
| CemdisiranAlso: ALN-CC5Subcutaneous (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 20 Oct 2025.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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 and Cemdisiran in people with Paroxysmal Nocturnal Hemoglobinuria, 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 7 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 20 Oct 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 20 Oct 2025.