Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT04811716
Phase
PHASE2
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Pozelimab — DRUG
    Administered Sub-cutaneous (SC) per protocol
  • Cemdisiran — DRUG
    Administered SC per protocol

Study Details

The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) 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 total complement hemolysis activity (CH50) * To evaluate the effect of the combination treatment on hemoglobin levels * To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements * To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life * To assess the concentrations of total pozelimab in serum and total complement component (C) 5 and cemdisiran in plasma * To assess immunogenicity to pozelimab and cemdisiran * To evaluate the long-term safety and efficacy of pozelimab and cemdisiran in an optional open-label extension period (OLEP) * To assess safety after treatment intensification with pozelimab and cemdisiran

Key Dates

Start date
Jul 29, 2021
Status verified
Apr 2025
Primary completion
Oct 25, 2022
Completion
Oct 18, 2023

Study Design

Enrollment
24 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Pozelimab Q4W + Cemdisiran
  • Experimental: Pozelimab Q2W + Cemdisiran

Primary Outcome Measure

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Through Week 28 ]

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