Clinical Trial: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

Official Title: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Brief Summary: This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Detailed Summary: Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: The PNH Registry will collect data to evaluate safety data specific to the use of Soliris. [ Time Frame: Ongoing (up to 13 years) ]

Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of HAHA to Soliris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, serious hemolysis, pregnancies, infusion reactions, targeted adverse events, bone marrow transplant, and mortality. In addition, all serious adverse events (SAEs), regardless of causality, will be collected in order to search for unknown safety concerns.


Original Primary Outcome: The PNH Registry will collect data to evaluate safety data specific to the use of Soliris. [ Time Frame: Ongoing (up to 13 years) ]

Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of HAHA to Soliris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, serious hemolysis, pregnancies, infusion reactions, targeted adverse events, bone marrow transplant, and mortality.


Current Secondary Outcome: PNH Registry will collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris and non-Soliris treated patients. [ Time Frame: Ongoing (up to 13 years) ]

Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.


Original Secondary Outcome: Same as current

Information By: Alexion Pharmaceuticals

Dates:
Date Received: April 15, 2011
Date Started: January 2007
Date Completion: December 2020
Last Updated: February 17, 2017
Last Verified: February 2017