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Two drugs, eculizumab or ravulizumab,in combination with cemdisiran and pozelimab, to treat paroxysmal nocturnal hemoglobinuria (PNH)


18 and older

Phase 3

1 Location


Clinical Trial Goal

To find out if eculizumab or ravulizumab, in combination with cemdisiran and pozelimab, is safe and works well to treat PNH.

You may be able to join this trial if you:

  • Are 18 years old or older
  • Have PNH
  • Have not been treated with a C5 inhibitor in the last 6 months. Your doctor can tell you this
  • Have not had a blood or marrow transplant (BMT)
  • Agree to have other standard tests done to see if you can be in the clinical trial 

Trial Details

Cemdisiran is a drug that targets C5 in certain cells.
Eculizumab, pozelimab and ravulizumab are monoclonal antibodies that target C5 in certain cells. 

In this trial, you’ll be randomized to 1 of 2 groups. Once you’re randomized, you’ll be told what group you’re in:
  • Group 1 – Cemdisiran and pozelimab plus eculizumab
  • Group 2 – Cemdisiran and pozelimab plus ravulizumab

Randomized means doctors will use a computer to assign you to either group. A computer assigns you by chance, like flipping a coin or drawing a name out of a hat. You, your doctor or the clinical trial doctor won’t have any control over which group you’ll be assigned. This means you won’t be able to choose your group.
You’ll get: 
  • Cemdisiran - Given as a shot under your skin
  • Eculizumab - Group 1 only - Given as intravenous (IV) infusions 
  • Pozelimab - Given as IV infusions or as a shot under your skin
  • Ravulizumab - Group 2 only - Given as IV infusions

You may continue treatment for 6 months. have blood tests to see how well the treatment is working. The clinical trial doctors will check your health for about 1 year.

The Food and Drug Administration (FDA) has approved eculizumab and ravulizumab to treat PNH. The FDA has not yet approved cemdisiran or pozelimab.


Clinical Trials Administrator, 844-734-6643,


The Oncology Institute of Hope and InnovationRecruiting

Whittier, California record

NCT05133531. First posted on 11/24/21

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