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NCT05133531 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, sponsored by Regeneron Pharmaceuticals.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 190
- Enrollment target
- 20
- Study locations
NCT05133531: Recruiting Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, sponsored by Regeneron Pharmaceuticals.
NCT05133531 is a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria that is actively recruiting participants, run by Regeneron Pharmaceuticals. The registered enrollment target is 190 participants, below the 251-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (24% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05133531, a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria, is actively recruiting participants, sponsored by Regeneron Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 190 participants
- Enrollment target
- 20
- Study locations
Study Summary
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)
Primary Outcome
Cohort A
Conditions Studied
Interventions
- DRUG Ravulizumab
- DRUG Pozelimab
- DRUG Cemdisiran
- DRUG Eculizumab
Study Locations (20)
Other
- A Beneficencia Portuguesa de Sao Paulo, BP Mirante - São Paulo
- Casa de Saude Santa Marcelina - São Paulo
- George Papanikolaou Hospital - Thessaloniki
- Semmelweis University - Budapest
- Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC) - Jaipur
- Hematology Citta della Salute e della Scienza di Torino - Turin
Kerala
- Malabar Cancer Center, Kerala - Kannur
- Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims - Kochi
Uttar Pradesh
- King George Hospital - Lucknow
- Sanjay Gandhi Postgraduate Institute of Medical Sciences - Lucknow
California
- The Oncology Institute of Hope & Innovation - Whittier
São Paulo
- Centro de Estudos e Pesquisas em Hematologia e Oncologia - Santo André
Ontario
- Toronto General Hospital - Toronto
Beijing Municipality
- Peking Union Medical College Hospital - Beijing
Antioquia
- Hospital Pablo Tobon Uribe - Medellín
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 190 participants |
| Start Date | 2022-08-01 |
| Est. Completion | 2027-01-28 |
| Phase | Phase 3 |
What NCT05133531 shows while recruiting
NCT05133531 is an interventional study that assigns participants to a tested intervention. The registered 190 participants enrollment target is mid-sized for trials with a published cap, below the 251-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (24% lower).
The record links to 1 condition, with Paroxysmal Nocturnal Hemoglobinuria appearing as the primary indexed condition, and to 4 interventions - of which Ravulizumab is the first listed.
NCT05133531 names 20 study sites across 13 states, led by Other, Kerala, Uttar Pradesh.
Frequently Asked Questions
What is clinical trial NCT05133531 about?
NCT05133531 is a clinical study titled "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". 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 p...
What is the current status of trial NCT05133531?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 190 participants. The study started on 2022-08-01. Estimated completion is 2027-01-28.
What conditions does trial NCT05133531 study?
This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria.
What interventions are being tested in trial NCT05133531?
The interventions under investigation include: Ravulizumab (DRUG), Pozelimab (DRUG), Cemdisiran (DRUG), Eculizumab (DRUG).
Who is sponsoring clinical trial NCT05133531?
This trial is sponsored by Regeneron Pharmaceuticals, which has 280 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05133531 being conducted?
This trial has 20 study locations across California, São Paulo, Ontario, Beijing Municipality, Antioquia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Paroxysmal Nocturnal Hemoglobinuria
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05133531's enrollment target sits among peer trials
190 4th of 14 higher than 10 of 14 other Paroxysmal Nocturnal Hemoglobinuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Paroxysmal Nocturnal Hemoglobinuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
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-
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
ACTIVE NOT RECRUITING · Phase 3
-
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
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-
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
COMPLETED · Phase 3
-
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
COMPLETED · Phase 3
-
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
COMPLETED · Phase 3
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