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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

Study acronym: ACCESS-EXT
StatusRecruiting
PhasePhase 3
Started2023-03-07
View on ClinicalTrials.gov ↗

Amendment history

2026-07-29
Study Status, Contacts/Locations v30
Primary completion moved earlier: 2029-02-26 → 2028-10-10
Completion moved earlier: 2029-02-26 → 2028-10-10
Study sites45→46
2026-07-10
Study Status, Contacts/Locations v29
Study sites44→45
2026-06-16
Study Status, Contacts/Locations v28
Study sites43→44
2026-03-16
Study Status, Contacts/Locations v27
Study sites42→43
2026-03-03
Study Status, Contacts/Locations v26
Study sites41→42
Show 25 earlier versions
2026-02-20
Study Status, Contacts/Locations v25
Study sites41 entries, revised
SrinagarindFaculty Hospitalof Medicine Khon Kaen University
2026-02-04
Study Status, Contacts/Locations v24
Study sites37→41
2025-11-21
Study Status, Contacts/Locations v23
Study sitesdetails revised at 5 of 37 sites
2025-10-08
Study Status v22
Re-verified, no change to tracked fields
2025-10-07
Study Status, Contacts/Locations v21
Study sitesdetails revised at 1 of 37 sites
2025-09-16
Study Status, Contacts/Locations v20
Study sites36→37
2025-08-18
Study Status, Contacts/Locations v19
Study sites35→36
2025-06-19
Study Identification, Study Status, Study Description, Arms and Interventions, Outcome Measures, Contacts/Locations v18
Primary completion date2029-02-05→2029-02-26
Completion date2029-02-05→2029-02-26
Study sites34→35
Secondary endpoints57 to 53 entries
Maintenance of adequate control of hemolysis (LDH ≤1.5 × ULN) Maintenance of adequate control of hemolysis (LDH ≤1.5 × ULN) Maintenance of adequate control of hemolysis (LDH ≤1.5 × ULN) Maintenance of adequate control of hemolysis (LDH ≤1.5 × ULN)
Study title-1 characters
[...] ab + Cemdisiran Combination Therapy is and How Well it Works.
2025-04-28
Study Status, Contacts/Locations, IPDSharing v17
Study sites32→34
2025-04-08
Study Status, Contacts/Locations v16
Study sitesdetails revised at 1 of 32 sites
2025-03-07
Study Status, Contacts/Locations v15
Study sitesdetails revised at 1 of 32 sites
2025-02-04
Study Status, Contacts/Locations v14
Study sitesdetails revised at 1 of 32 sites
2025-01-29
Study Status, Contacts/Locations v13
Study sitesdetails revised at 1 of 32 sites
2024-12-17
Study Status, Contacts/Locations v12
Study sites31→32
2024-11-20
Study Status, Contacts/Locations v11
Study sites29→31
2024-10-21
Study Status, Contacts/Locations v10
Study sites28→29
2024-09-11
Study Status, Contacts/Locations v9
Study sites27→28
2024-06-27
Study Status, Eligibility v8
Eligibility criteria-17 characters
[...] n within 5 years prior to enrollment and serotype B vaccine (when available) within 3 years prior to enrollment as described in the proto [...]
2024-06-06
Study Status, Eligibility, Contacts/Locations v7
Study sites25→27
Eligibility criteria-468 characters
Key Inclusion Criteria: 1. All patients will require completion of the primary vaccination series of both meningococcal vaccines (serotypes ACWY and B) in accordance with Advisory Committee on Immunization Practices (ACIP) guidelines for patients receiving a complement inhibitor. Patients Entering from the Parent Study Patients with PNH wh [...] vaccine (when available) within 3 years prior to enrollment. Note: Patientsas withoutdescribed prior vaccination will be eligible provided they are willing to undergo vaccination prior to initiation of study treatment and, at a minimum,in the first dose of v [...] [...]
2024-05-29
Study Identification, Study Status, Study Description, Arms and Interventions, Eligibility v6
Primary completion date2029-05-23→2029-02-05
Completion date2029-05-23→2029-02-05
Eligibility criteria+920 characters
Key Inclusion Criteria: 1. All patients will require completion of the primary vaccination series of both meningococcal vaccines (serotypes ACWY and B) in accordance with Advisory Committee on Immunization Practices (ACIP) guidelines for patients receiving a complement inhibitor. Patients Entering from the Parent Study 1. Patients with PNH who have completed, without permanent disc [...] l treatment period (OLTP) transition period, if applicable. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol. Patients Entering wi [...] [...]
Study title+8 characters
A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy 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.
2024-04-22
Study Status, Contacts/Locations v5
Study sites24→25
2024-01-30
Study Status, Contacts/Locations v4
Study sites18→24
2023-11-28
Study Status, Outcome Measures, Eligibility, Contacts/Locations v3
Study sites13→18
Eligibility criteria+10 characters
Key Inclusion Criteria: Patients Entering from the Parent StudiesStudy 1. Patients with PNH who have completed, without permanent [...] t Key Exclusion Criteria: Patients Entering from the Parent StudiesStudy Significant protocol deviation(s) in the parent study based [...] evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferas [...]
Primary endpoints6 to 7 entries
TreatmentIncidence emergentof adverse events (AEs) leading to permanent treatment discontinuation Severity of adverse events (AEs) leading to permanent treatment discontinuation
2023-11-01
Study Status, Study Description, Study Design, Arms and Interventions, Eligibility, Contacts/Locations v2
Primary completion date2028-03-23→2029-05-23
Completion date2028-03-23→2029-05-23
Enrollment target300→202
Study sites1→13
Eligibility criteria+131 characters
[...] eted, without permanent discontinuation, study treatment in at least 1 of the parent studiesstudy (R3918-PNH-2021[NCT05133531]), and/orincluding R3918the post-PNHOpen-2022label [NCT05131204]treatment period (OLTP) transition period, if applicable. Patients Entering with C5 polymorphism Pati [...] of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes Active disease, as defined by the presence of 1 or more PNH [...] t Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening Patients with known HIV with history of op [...] vidence of c [...]
2023-04-07
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Primary completion date2028-03-27→2028-03-23
Completion date2028-03-27→2028-03-23
Start dateestimated 2023-03-09→confirmed 2023-03-07
Study sites0→1
2023-02-22
Original filing
Regeneron
148 trials monitored
See 16 trials at risk
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
Trial Details
NCT Number NCT05744921
Lead Sponsor Regeneron Pharmaceuticals
Conditions Paroxysmal Nocturnal Hemoglobinuria
Enrollment 202 participants
Start Date 2023-03-07
Primary Completion 2028-10-10 (estimated)
Study Completion 2028-10-10 (estimated)
Updated on ClinicalTrials.gov 2026-09-28