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

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 study evaluates the safety and effectiveness of a combination therapy using two experimental drugs, pozelimab and cemdisiran, in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). It aims to assess how well this treatment works over the long term, including monitoring for side effects and measuring drug levels in the blood. Additionally, the study will investigate whether patients develop antibodies against the drugs, which could impact their effectiveness. Participants must have completed a prior study or have specific genetic markers related to their PNH condition.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

Patients Entering from the Parent Study

1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021\[NCT05133531\]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
2. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol.

Patients Entering with C5 polymorphism

1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit
5. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol

Key Exclusion Criteria:

Patients Entering from the Parent Study

1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study

Patients Entering with C5 polymorphism

1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol
4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
6. Known hereditary complement deficiency
7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Where it's running

• Liga Paranaense de Combate Ao Cancer - Hospital Erasto Gaertner
  Curitiba, Jardim Santo Andre, Brazil Recruiting
• A Beneficencia Portuguesa de Sao Paulo, BP Mirante
  São Paulo, Brazil Recruiting
• Toronto General Hospital
  Toronto, Ontario, Canada Recruiting
• Hospital Pablo Tobon Uribe
  Medellín, Antioquia, Colombia Recruiting
• Semmelweis University/Semmelweis Egyetem
  Budapest, Hungary Recruiting
• Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
  Kochi, Kerala, India 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.

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

  This trial tests whether combining pozelimab and cemdisiran can better reduce hemolysis in adults with PNH whose C5 inhibitor treatment isn’t controlling their disease.

  Recruiting at 20 sites Phase 3 Regeneron Pharmaceuticals · Ribeirão Preto, São Paulo and 19 other locations · NCT07154745

• 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.

  Recruiting at 3 sites Novartis · Jeollanam and 2 other locations · NCT07416162

• 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.