MedTrace Logo

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

Last updated 2026-04-30

No results posted yet for this 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)

Conditions

Interventions

DRUG

Ravulizumab

Administered Intravenous (IV) per the protocol

DRUG

Pozelimab

Administered IV and subcutaneous (SC) per the protocol

DRUG

Cemdisiran

Administered SC per the protocol

DRUG

Eculizumab

Administered IV per the protocol

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-01
Primary Completion
2026-10-12
Completion
2026-10-26
FDA Drug
Yes

Countries

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

Study Locations

More Related Trials

Entities

Drugs
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 NCT05133531 on ClinicalTrials.gov