Pozelimab + cemdisiran for adults with PNH whose current treatment isn't working

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, interventional trial testing a combination of two investigational agents, pozelimab and cemdisiran, in adults with PNH who have persistent hemolysis despite treatment with approved C5 inhibitors. Eligible participants must have been on labeled doses of eculizumab, ravulizumab, or crovalimab for at least six months and have persistently elevated LDH (>1.5× ULN) attributed to intravascular hemolysis. The treatment program includes a 28-week primary treatment period followed by a 52-week extension, with monitoring for hemolysis markers, safety, drug levels, and anti-drug antibodies. The study will collect adverse event data and pharmacokinetic/pharmacodynamic measures to determine whether the combination improves disease control and is tolerable.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
3. LDH persistently \> 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis
4. At least 2 screening LDH values from different visits as described in the protocol
5. Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study

Key Exclusion Criteria:

1. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
2. Body weight \<40 kilograms at screening visit
3. Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
4. Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
5. Known hereditary complement deficiency

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Where it's running

• Hospital das Clinicas da FMRP USP
  Ribeirão Preto, São Paulo, Brazil Recruiting
• Faculdade de Medicina do ABC
  Santo André, São Paulo, Brazil Recruiting
• Hospital Santa Marcelina
  São Paulo, Brazil Recruiting
• Cisss-Ca
  Lévis, Quebec, Canada Recruiting
• Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli
  Rome, Lazio, Italy Recruiting
• AOU Careggi
  Florence, Tuscany, Italy Recruiting

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term treatment for patients with paroxysmal nocturnal hemoglobinuria using pozelimab and cemdisiran

  This study is testing a new combination of two experimental drugs, pozelimab and cemdisiran, to see if they can safely help adults with paroxysmal nocturnal hemoglobinuria over a long period of time.

  Recruiting at 48 sites Phase 3 Regeneron Pharmaceuticals · Curitiba, Jardim Santo Andre and 49 other locations · NCT05744921

• Oral HS-10542 for paroxysmal nocturnal hemoglobinuria

  This study will test whether the oral drug HS-10542 can reduce hemolysis and raise hemoglobin in adults with PNH, including those not fully helped by C5 inhibitor therapy.

  Recruiting Phase 1/2 Jiangsu Hansoh Pharmaceutical Co., Ltd. · Hangzhou, Zhejiang · NCT07470762

• Danicopan added to ravulizumab or eculizumab for PNH in Korea

  This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.

  Recruiting at 3 sites AstraZeneca · Busan and 3 other locations · NCT07457151

• Iptacopan use in Korean patients with PNH or C3G

  This post-marketing surveillance study tests how well iptacopan works and how safe it is for Korean adults with PNH or C3G in real-world care.

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• Eye injection of pozelimab for geographic atrophy in adults

  This study will test whether injections of pozelimab into the eye are safe and well tolerated in adults with geographic atrophy from age-related macular degeneration.

  Recruiting at 9 sites Phase 1 Regeneron Pharmaceuticals · Lakewood, Colorado and 8 other locations · NCT07230834

• Pozelimab safety in children 1 to 5 with CHAPLE disease

  This trial will test whether pozelimab is safe and tolerable in children ages 1 to 5 who have CHAPLE disease.

  Recruiting Phase 4 Regeneron Pharmaceuticals · Istanbul · NCT07142343

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.