Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
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ClinicalTrials.gov Identifier: NCT01374360 |
Recruitment Status :
Recruiting
First Posted : June 16, 2011
Last Update Posted : December 15, 2021
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Sponsor:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexion Pharmaceuticals
Tracking Information | |||||
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First Submitted Date | April 15, 2011 | ||||
First Posted Date | June 16, 2011 | ||||
Last Update Posted Date | December 15, 2021 | ||||
Actual Study Start Date | January 2007 | ||||
Estimated Primary Completion Date | December 2025 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures |
Evaluate safety data specific to the use of Soliris and Ultomiris [ 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 and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.
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Original Primary Outcome Measures |
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.
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Change History | |||||
Current Secondary Outcome Measures |
Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris 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.
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Original Secondary Outcome Measures |
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.
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified 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 |
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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 |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status | Recruiting | ||||
Estimated Enrollment |
5000 | ||||
Original Estimated Enrollment |
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:
Exclusion Criteria:
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Sex/Gender |
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Ages | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers | No | ||||
Contacts |
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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 |
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Responsible Party | Alexion Pharmaceuticals | ||||
Study Sponsor | Alexion Pharmaceuticals | ||||
Collaborators | Not Provided | ||||
Investigators |
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PRS Account | Alexion Pharmaceuticals | ||||
Verification Date | December 2021 |