A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT05744921
Phase
PHASE3
Status
Recruiting
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Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

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

Interventions

  • Pozelimab — DRUG
    Administered per the protocol
  • Cemdisiran — DRUG
    Administered per the protocol

Study Details

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)

Key Dates

Start date
Mar 7, 2023
Status verified
Mar 2026
Primary completion
Feb 26, 2029
Completion
Feb 26, 2029

Study Design

Enrollment
202 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: PNH Transition Patients
    Patients with PNH who completed treatment/ protocol requirements (as applicable) in the parent study (R3918-PNH-2021 \[NCT05133531\])
  • Experimental: C5 Polymorphism Patients
    Patients who have not been treated in either parent study but who have a documented complement component 5 (C5) variation rendering them refractory to eculizumab/ravulizumab. Note: Loading dose of pozelimab administered IV on Day 1.

Primary Outcome Measure

Incidence of treatment-emergent serious adverse events (SAEs) [ Time Frame: Up to week 108 ]

Central Contacts

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