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Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

NCT04888507 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-08-29

Study results available
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Summary

The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy

The secondary objectives of the study are:

* To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of CH50
* To evaluate the effect of the combination treatment on the stability of LDH during the transition period from eculizumab monotherapy to combination with pozelimab and cemdisiran
* To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements
* To evaluate the effect of the combination treatment on hemoglobin levels
* To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life (HRQoL)
* To assess the concentrations of total pozelimab and eculizumab in serum; and total cemdisiran and C5 protein in plasma
* To assess the immunogenicity of pozelimab and cemdisiran
* To assess safety after dose intensification
* To evaluate the long-term safety and efficacy of the combination treatment in an optional open-label extension period (OLEP)

Conditions

Interventions

DRUG

Pozelimab

Intravenous (IV) loading dose (once) followed after 30 minutes by sub-cutaneous (SC) administration

DRUG

Cemdisiran

SC administration

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-08
Primary Completion
2022-05-05
Completion
2023-05-04
FDA Drug
Yes

Countries

  • United Kingdom

Study Locations

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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 NCT04888507 on ClinicalTrials.gov