Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Paroxysmal Nocturnal Hemoglobinuria
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry|
- The PNH Registry will collect data to evaluate safety data specific to the use of Soliris. [ Time Frame: Ongoing (up to 13 years) ] [ Designated as safety issue: Yes ]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.
- 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) ] [ Designated as safety issue: No ]Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.
|Study Start Date:||January 2007|
|Estimated Study Completion Date:||December 2020|
|Estimated Primary Completion Date:||December 2020 (Final data collection date for primary outcome measure)|
PNH patients of any age, including minors, that are receiving Soliris
Not receiving Soliris
PNH patients of any age, including minors, that are not receiving Soliris
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360
|Contact: Lynn Sandersemail@example.com|
|United States, Maryland|
|Johns Hopkins University Medical Center||Recruiting|
|Baltimore, Maryland, United States|
|Study Director:||Stephen Squinto, PhD||Alexion Pharmaceuticals|