A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT07154745
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 a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH). The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs? * How much of the study drugs are in the blood at different times? * Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)

Key Dates

Start date
May 30, 2026
Status verified
May 2026
Primary completion
Jan 6, 2030
Completion
Nov 22, 2031

Study Design

Enrollment
35 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Pozelimab + Cemdisiran Combo

Primary Outcome Measure

Percent change in Lactate Dehydrogenase (LDH) during TP [ Time Frame: From baseline to week 28 ]

Central Contacts

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