A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

Part of paid clinical trials in Whittier, California.

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT05133531
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

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

Interventions

  • Ravulizumab — DRUG
    Administered Intravenous (IV) per the protocol
  • Pozelimab — DRUG
    Administered IV and subcutaneous (SC) per the protocol
  • Cemdisiran — DRUG
    Administered SC per the protocol
  • Eculizumab — DRUG
    Administered IV per the protocol

Study Details

This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab. The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug". The study is looking at several research questions, including: * How effective is the pozelimab + cemdisiran combination compared to ravulizumab? * How effective is pozelimab + cemdisiran combination compared to eculizumab? * What side effects may happen from taking the study drugs? * How much study drugs are in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)

Key Dates

Start date
Aug 1, 2022
Status verified
Apr 2026
Primary completion
Oct 12, 2026
Completion
Oct 26, 2026

Study Design

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

Arms

  • Experimental: Cohort A
    Randomized 1:1
  • Experimental: Cohort B
    Randomized 1:1

Primary Outcome Measure

Percent change in lactate dehydrogenase (LDH) [ Time Frame: From baseline to week 26 ]

Locations (1)

FacilityCityStateZIPSite coordinators
The Oncology Institute of Hope & InnovationWhittierCalifornia90602-

Find similar trials in Whittier, CA

By research site
The Oncology Institute of Hope & Innovation· Whittier, CA

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