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 (NCT05133531)
A Randomized, Open-Label, C5 Inhibitor-Controlled 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
🩺 Plain-English Summary (8th-Grade Level)
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab. The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug". The study is looking at several research questions, including: * How effective is the pozelimab + cemdisiran combination compared to ravulizumab? * How effective is pozelimab + cemdisiran combination compared to eculizumab? * What side effects may happen from taking the study drugs? * How much study drugs are in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)
Who can join: Review the eligibility criteria below.
Location: The Oncology Institute of Hope & Innovation — Whittier, California
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 (68 Sites)
Showing the first 25 of 68 sites.
- The Oncology Institute of Hope & Innovation — Whittier, California
- Centro de Estudos e Pesquisas em Hematologia e Oncologia — Santo André, São Paulo
- A Beneficencia Portuguesa de Sao Paulo, BP Mirante — São Paulo,
- Casa de Saude Santa Marcelina — São Paulo,
- Toronto General Hospital — Toronto, Ontario
- Peking Union Medical College Hospital — Beijing, Beijing Municipality
- Hospital Pablo Tobon Uribe — Medellín, Antioquia
- George Papanikolaou Hospital — Thessaloniki,
- Semmelweis University — Budapest,
- Malabar Cancer Center, Kerala — Kannur, Kerala
- 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 (PGIMER) — Chandigarh, Punjab
- King George Hospital — Lucknow, Uttar Pradesh
- Sanjay Gandhi Postgraduate Institute of Medical Sciences — Lucknow, Uttar Pradesh
- Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC) — Jaipur,
- Aou Careggi — Florence, Firenze
- Fondazione Policlinico Universitario A. Gemelli - IRCCS — Rome, Roma
- Hematology Citta della Salute e della Scienza di Torino — Turin,
- Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital — Nagoya, Aichi-ken
- Ogaki Municipal Hospital — Ōgaki, Gifu
- University of Tsukuba Hospital — Tsukuba, Ibaraki
- Matsushita Memorial Hospital — Moriguchi, Osaka
- NTT Medical Center Tokyo — Shinagawa-ku, Tokyo
💰 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
Explore by condition: All Paroxysmal Nocturnal Hemoglobinuria clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.