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RECRUITING 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

NCT05133531 · View on ClinicalTrials.gov ↗

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)

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

FieldValue
Enrollment Target 190 participants
Start Date 2022-08-01
Est. Completion 2027-01-28
Phase Phase 3

Sponsor

Regeneron Pharmaceuticals

290 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05133531

The ClinicalTrials.gov registry entry for NCT05133531 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 190 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Regeneron Pharmaceuticals, which has 290 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT05133531 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, Kerala, Uttar Pradesh. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT05133531?

The interventions under investigation include: Ravulizumab (DRUG), Pozelimab (DRUG), Cemdisiran (DRUG), Eculizumab (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT05133531?

This trial is sponsored by Regeneron Pharmaceuticals, which has 290 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial