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

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT04888507
Phase
PHASE2
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

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

Interventions

  • Pozelimab — DRUG
    Intravenous (IV) loading dose (once) followed after 30 minutes by sub-cutaneous (SC) administration
  • Cemdisiran — DRUG
    SC administration

Study Details

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)

Key Dates

Start date
Jul 8, 2021
Status verified
Aug 2025
Primary completion
May 5, 2022
Completion
May 4, 2023

Study Design

Enrollment
6 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Pozelimab+Cemdisiran

Primary Outcome Measure

OLTP: Number of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to Day 225 ]

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