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 (NCT05744921)
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
🩺 Plain-English Summary (8th-Grade Level)
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)
Who can join: Review the eligibility criteria below.
Location: Toronto General Hospital — Toronto, Ontario
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (46 Sites)
Showing the first 25 of 46 sites.
- Toronto General Hospital — Toronto, Ontario
- Hospital Pablo Tobon Uribe — Medellín, Antioquia
- Semmelweis University/Semmelweis Egyetem — Budapest,
- Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims — Kochi, Kerala
- K J Somaiya Super Specialty Hospital & Research Centre — Mumbai, Maharashtra
- Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus — New Delhi, National Capital Territory of Delhi
- Postgraduate Institute of Medical Education & Research — Chandigarh,
- Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC) — Jaipur,
- Aou Careggi — Florence, Firenze
- SC Hematology, AOU Città della Salute e della Scienza di Torino — Torino,
- Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital — Nagoya, Aichi-ken
- University of Tsukuba Hospital — Tsukuba, Ibaraki
- Jordan University Hospital (JUH) — Amman,
- Hospital Tg Ampuan Afzan — Kuantan, Pahang
- Hospital Queen Elizabeth — Kota Kinabalu, Sabah
- Hospital Ampang — Ampang, Selangor
- Clinica San Felipe — Lima,
- St Lukes Medical Center — Quezon City, National Capital Region
- In-Vivo Sp. z o.o. — Bydgoszcz, Kuyavian-Pomeranian Voivodeship
- University Clinical Center Medical University of Gdansk — Gdansk, Pomeranian Voivodeship
- Szpital Uniwersytecki Nr2 Bydgoszcz — Bydgoszcz,
- Prof Dr Ion Chiricuta Cancer Institute — Cluj-Napoca, Cluj
- National University Hospital — Singapore,
- St. Vincent Hospital — Suwon, Gyeonggi-do
- Ajou University Medical Center — Suwon, Gyeonggi-do
📞 Contact the Study Coordinator
Phone: (844) 734-6643
Email: clinicaltrials@regeneron.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Paroxysmal Nocturnal Hemoglobinuria
- Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data — Boston, Massachusetts
- A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors — Ciudad Autonoma Buenos Aires,
- Long-term Safety of Danicopan: IPIG Registry-based Cohort Study — Boston, Massachusetts
- A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH — Los Angeles, California
- Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria — Duarte, California
- A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment — Whittier, California
Explore by condition: All Paroxysmal Nocturnal Hemoglobinuria clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.