Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
NCT ID: NCT04811716
Last Updated: 2025-04-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
24 participants
INTERVENTIONAL
2021-07-29
2023-10-18
Brief Summary
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The secondary objectives of the study are:
* To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of total complement hemolysis activity (CH50)
* To evaluate the effect of the combination treatment on hemoglobin levels
* To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements
* To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life
* To assess the concentrations of total pozelimab in serum and total complement component (C) 5 and cemdisiran in plasma
* To assess immunogenicity to pozelimab and cemdisiran
* To evaluate the long-term safety and efficacy of pozelimab and cemdisiran in an optional open-label extension period (OLEP)
* To assess safety after treatment intensification with 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 Q4W + Cemdisiran
Pozelimab
Administered Sub-cutaneous (SC) per protocol
Cemdisiran
Administered SC per protocol
Pozelimab Q2W + Cemdisiran
Pozelimab
Administered Sub-cutaneous (SC) per protocol
Cemdisiran
Administered SC per protocol
Interventions
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Pozelimab
Administered Sub-cutaneous (SC) per protocol
Cemdisiran
Administered SC per protocol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
2. Participants with documented history of liver cirrhosis or participants with liver disease with evidence of currently impaired liver function; or participants with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) as described in the protocol
3. Significant protocol deviation(s) in the parent study based on the investigator's judgment as described in the protocol
4. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the participant unsuitable for enrollment or would jeopardize the safety of the participant
5. Known hypersensitivity to cemdisiran or any component of cemdisiran formulation
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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Prince of Wales Hospital
Hong Kong, New Territories, Hong Kong
D l Pesti Centrumk rh z Orsz gos Hematol giai s Infektol giai Int zet
Budapest, Nagyvárad Tér 1, Hungary
Hospital Miri
Miri, Sarawak, Malaysia
Hospital Sibu
Sibu, Sarawak, Malaysia
Hospital Sultanah Nur Zahirah
Kuala Terengganu, Terengganu, Malaysia
Samsung Medical Center
Seoul, Seoul Teugbyeolsi, South Korea
Pusan National University Hospital
Busan, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Yonsei University College of Medicine, Severance Hospital
Seoul, , South Korea
Ewha Womans University Medical Centre
Seoul, , South Korea
National Taiwan University Hospital
Taipei, , Taiwan
Chang Gung Memorial Hospital
Taoyuan, , Taiwan
St. James's University Hospital
Leeds, West Yorkshire, United Kingdom
Countries
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References
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Jang JH, Wong RSM, Hartford C, Pavani R, Aurand L, Nguyen Q, Mohan K, Meagher K, Sherman S, Rofail D, Perlee L, Souttou A, Kelly RJ. Safety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria. EJHaem. 2025 Jul 24;6(4):e70095. doi: 10.1002/jha2.70095. eCollection 2025 Aug.
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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2020-005005-17
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
R3918-PNH-2092
Identifier Type: -
Identifier Source: org_study_id
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