Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Tracking Information
First Submitted Date	April 15, 2011
First Posted Date	June 16, 2011
Last Update Posted Date	February 26, 2018
Actual Study Start Date	January 2007
Estimated Primary Completion Date	December 2025 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures; (submitted: February 22, 2018)	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 human anti-drug antibodies (ADA) to Soliris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.
Original Primary Outcome Measures; (submitted: June 15, 2011)	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.
Change History	Complete list of historical versions of study NCT01374360 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures; (submitted: February 22, 2018)	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 Measures; (submitted: June 15, 2011)	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.
Current Other Outcome Measures	Not Provided
Original Other Outcome Measures	Not Provided
Descriptive Information
Brief Title	Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
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 Description	Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Study Type	Observational
Study Design	Observational Model: Other; Time Perspective: Prospective
Target Follow-Up Duration	Not Provided
Biospecimen	Not Provided
Sampling Method	Probability Sample
Study Population	PNH Patients
Condition	Paroxysmal Nocturnal Hemoglobinuria
Intervention	Not Provided
Study Groups/Cohorts	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
Publications *	Chou WC, Huang WH, Wang MC, Chang CS, Yeh SP, Chiou TJ, Chen YC, Lin TH, Shen MC; Taiwan PNH study group. Characteristics of Taiwanese patients of PNH in the international PNH registry. Thromb J. 2016 Oct 4;14(Suppl 1):39. eCollection 2016.; Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. doi: 10.1056/NEJMoa1502950.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status	Recruiting
Estimated Enrollment; (submitted: January 11, 2018)	5000
Original Estimated Enrollment; (submitted: June 15, 2011)	2000
Estimated Study Completion Date	December 2025
Estimated Primary Completion Date	December 2025 (Final data collection date for primary outcome measure)
Eligibility Criteria	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.
Sex/Gender	Sexes Eligible for Study: All
Ages	Child, Adult, Senior
Accepts Healthy Volunteers	No
Contacts	Contact: Alexion Pharmaceuticals, Inc. ClinicalTrials@alexion.com
Listed Location Countries	United States
Removed Location Countries
Administrative Information
NCT Number	NCT01374360
Other Study ID Numbers	M07-001
Has Data Monitoring Committee	No
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Plan to Share IPD: No
Responsible Party	Alexion Pharmaceuticals
Study Sponsor	Alexion Pharmaceuticals
Collaborators	Not Provided
Investigators	Study Director: Jing Marantz Alexion Pharmaceuticals
PRS Account	Alexion Pharmaceuticals
Verification Date	February 2018