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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 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 202

Last updated 2026-03-18

No results posted yet for this study

Summary

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)

Conditions

Interventions

DRUG

Pozelimab

Administered per the protocol

DRUG

Cemdisiran

Administered per the protocol

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-07
Primary Completion
2029-02-26
Completion
2029-02-26
FDA Drug
Yes

Countries

  • Canada
  • Colombia
  • Hungary
  • India
  • Italy
  • Japan
  • Jordan
  • Malaysia
  • Peru
  • Philippines
  • Poland
  • Romania
  • Singapore
  • South Korea
  • Spain
  • Taiwan
  • Thailand
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05744921 on ClinicalTrials.gov