Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Part of paid clinical trials in Whittier, California.

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT05131204
Phase
PHASE3
Status
Terminated

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

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

Interventions

  • Cemdisiran — DRUG
    Administered per protocol
  • Eculizumab — DRUG
    Administered per protocol
  • Pozelimab — DRUG
    Administered per protocol
  • Ravulizumab — DRUG
    Administered per protocol

Study Details

The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy The secondary objectives of the study are to: * Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following: * Transfusion requirements and transfusion parameters * Measures of hemolysis: LDH control, breakthrough hemolysis, and inhibition of CH50 * Hemoglobin levels * Fatigue as assessed by Clinical Outcome Assessments (COAs) * Health-related quality of life (HRQoL) as assessed by COAs * Safety and tolerability * To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma * To assess the immunogenicity of pozelimab and cemdisiran

Key Dates

Start date
Oct 6, 2022
Status verified
Apr 2025
Primary completion
Jul 12, 2023
Completion
Jul 12, 2023

Study Design

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

Arms

  • Experimental: Pozelimab and Cemdisiran
    Randomized 1:1
  • Experimental: Anti-C5 standard-of-care
    Randomized 1:1

Primary Outcome Measure

Percent Change in Lactate Dehydrogenase (LDH) From Baseline to Week 36 [ Time Frame: From baseline to week 36 ]

Locations (1)

FacilityCityStateZIPSite coordinators
Regeneron Research FacilityWhittierCalifornia90603-

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By research site
Regeneron Research Facility· Whittier, CA

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