Clinical Trials Logo
  1. Home
  2. Other
  3. NCT04888507

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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:
A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Clinical Trial 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)

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04888507
Study type Interventional
Source Regeneron Pharmaceuticals
Contact
Status Completed
Phase Phase 2
Start date July 8, 2021
Completion date May 4, 2023
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT04432584 - A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors Phase 3
Completed NCT05828485 - Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects Phase 1
Recruiting NCT02179359 - Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies N/A
Active, not recruiting NCT04434092 - A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. Phase 3
Terminated NCT05131204 - Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Phase 3
Recruiting NCT01374360 - Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Active, not recruiting NCT05389449 - A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH Phase 3
Recruiting NCT06100900 - Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Phase 1
Completed NCT01272817 - Nonmyeloablative Allogeneic Transplant N/A
Completed NCT06326814 - A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults Phase 1
Completed NCT04463056 - Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH Phase 3
Recruiting NCT05476887 - To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 Phase 2
Completed NCT01192399 - Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Phase 2
Active, not recruiting NCT06051357 - Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria Phase 2
Recruiting NCT06154512 - A Real-world, Multi-center, Prospective, Observational Study for PNH in China
Completed NCT04128943 - Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Active, not recruiting NCT03329365 - Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
Recruiting NCT05755867 - Global PNH Patient Registry
Completed NCT04679103 - A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) Phase 3
Completed NCT05642585 - A Study of Single-dose MY008211A in Healthy Adults Phase 1