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Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01374360
First received: April 15, 2011
Last updated: February 17, 2017
Last verified: February 2017
History of Changes
April 15, 2011
February 17, 2017
January 2007
December 2020   (Final data collection date for primary outcome measure)
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.
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.
Complete list of historical versions of study NCT01374360 on ClinicalTrials.gov Archive Site
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.
Same as current
Not Provided
Not Provided
 
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Probability Sample
PNH Patients
Paroxysmal Nocturnal Hemoglobinuria
Not Provided
  • Receiving Soliris
    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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
4700
December 2020
December 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris and withdrawn from treatment. Patients who are minors must have parent/legal guardian consent and must be willing and able to give assent, if applicable as determined by the Ethics Committees/Institutional Review Boards. Upon attaining adulthood, these patients must be re-consented.
  • Ability to comprehend and sign consent to have data entered in the PNH Registry.

Exclusion Criteria:

  • Inability or unwillingness to sign informed consent.
  • Patients currently enrolled in an interventional clinical trial for treatment of PNH cannot be enrolled in the Registry at the same time.
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
United States
 
NCT01374360
M07-001
Yes
Not Provided
Plan to Share IPD: No
Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Study Director: Jing Marantz Alexion Pharmaceuticals
Alexion Pharmaceuticals
February 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP