A Randomized, Open-Label, Ravulizumab-Controlled,Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy ACCESS-1

What's the purpose of this trial?

The primary objective of the study is: To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 26-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy The secondary objectives of the study are to: * Evaluate the effect of pozelimab and cemdisiran combination treatment versus ravulizumab treatment on the following: * Measures of hemolysis * Transfusion parameters * Hemoglobin levels * Fatigue as assessed by Clinical Outcome Assessments (COAs) * Health-related quality of life (HRQoL) as assessed by COAs * Safety and tolerability * Complement activation * To assess the concentrations of total pozelimab and total ravulizumab in serum and cemdisiran and total complement factor 5 (C5) protein in plasma * To assess the immunogenicity of pozelimab and cemdisiran

This trial is currently open and accepting patients.

What will happen during the trial?

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Key Inclusion Criteria:

1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes described in the protocol
2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms described in the protocol
3. LDH level ≥2 × ULN at the screening visit

Key Exclusion Criteria:

1. Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
3. Body weight \<40 kilograms at screening visit
4. Planned use of any complement inhibitor therapy other than study drugs during the treatment period
5. Not meeting meningococcal vaccination requirements for ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit
6. Any contraindication for receiving Neisseria meningitidis vaccination
7. Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab prescribing information, where available, or national guidelines/local practice or if necessary when vaccination is less than 2 weeks from study treatment initiation)
8. Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Additional Trial Information

Phase 3

Enrollment: 148 patients (estimated)

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Trial Locations

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Innovative Clinical Research Institute

Whittier, CA

Open and Accepting
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