Status:

COMPLETED

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Lead Sponsor:

Regeneron Pharmaceuticals

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) The se...

Eligibility Criteria

Inclusion

  • Key
  • 1\. Participants with PNH who are receiving treatment with pozelimab monotherapy in the R3918- PNH-1868 study (NCT04162470)
  • Key

Exclusion

  • Documented, positive polymerase chain reaction (PCR) or equivalent test based on regional recommendations for COVID-19 or suspected SARS-CoV-2 infection as defined in the protocol
  • 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
  • Significant protocol deviation(s) in the parent study based on the investigator's judgment as described in the protocol
  • 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
  • Known hypersensitivity to cemdisiran or any component of cemdisiran formulation
  • NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply

Key Trial Info

Start Date :

July 29 2021

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

October 18 2023

Estimated Enrollment :

24 Patients enrolled

Trial Details

Trial ID

NCT04811716

Start Date

July 29 2021

End Date

October 18 2023

Last Update

April 8 2025

Active Locations (13)

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Page 1 of 4 (13 locations)

1

Prince of Wales Hospital

Hong Kong, New Territories, Hong Kong, 550540

2

D l Pesti Centrumk rh z Orsz gos Hematol giai s Infektol giai Int zet

Budapest, Nagyvárad Tér 1, Hungary, 1907

3

Hospital Miri

Miri, Sarawak, Malaysia, 98000

4

Hospital Sibu

Sibu, Sarawak, Malaysia, 96000