Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT ID: NCT05131204
Last Updated: 2025-04-08
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
3 participants
INTERVENTIONAL
2022-10-06
2023-07-12
Brief Summary
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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
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Pozelimab and Cemdisiran
Randomized 1:1
Cemdisiran
Administered per protocol
Eculizumab
Administered per protocol
Pozelimab
Administered per protocol
Ravulizumab
Administered per protocol
Anti-C5 standard-of-care
Randomized 1:1
Eculizumab
Administered per protocol
Ravulizumab
Administered per protocol
Interventions
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Cemdisiran
Administered per protocol
Eculizumab
Administered per protocol
Pozelimab
Administered per protocol
Ravulizumab
Administered per protocol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Treated with eculizumab or ravulizumab prior to screening visit as described in the protocol Note: Biosimilars are not permitted, unless approved by the Sponsor
Exclusion Criteria
2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
3. Body weight \< 40 kilograms at screening visit
4. Any use of complement inhibitor therapy other than eculizumab or ravulizumab in the 26 weeks prior to the screening visit or planned use during the study with the exception of study treatments
5. Not meeting meningococcal vaccination requirements for eculizumab or ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit.
6. Any contraindication for receiving Neisseria meningitidis vaccination.
7. Positive for hepatitis B, and/ or hepatitis C as described in the protocol
8. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
9. Participation in another interventional clinical study (except R3918-PNH-2021) or use of any experimental therapy within 30 days before screening visit or within 5 half-lives of that investigational product, whichever is greater, with the exception of eculizumab or ravulizumab.
10. Patients with functional or anatomic asplenia
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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Regeneron Research Facility
Whittier, California, United States
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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A Plain Language Summary is available on TrialSummaries.com
Other Identifiers
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2020-002761-33
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
R3918-PNH-2022
Identifier Type: -
Identifier Source: org_study_id
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