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Trial record 1 of 1 for:    NCT01374360
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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
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Condition or disease
Paroxysmal Nocturnal Hemoglobinuria

Detailed Description:
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Study Design
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Study Type : Observational
Estimated Enrollment : 5000 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Actual Study Start Date : January 2007
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
Receiving Soliris or Ultomiris
PNH patients of any age, including minors, that are receiving Soliris or Ultomiris
Not receiving Soliris or Ultomiris
PNH patients of any age, including minors, that are not receiving Soliris or Ultomiris


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
PNH Patients
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 or Ultomiris 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01374360


Contacts
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Contact: Alexion Pharmaceuticals, Inc. ClinicalTrials@alexion.com

Locations
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United States, Massachusetts
Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations. Recruiting
Boston, Massachusetts, United States, 02210
Contact: PNH Registry         
Contact       ClinicalTrials@alexion.com   
Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
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Study Director: Phillipe Gustovic Alexion Pharmaceuticals
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01374360    
Other Study ID Numbers: M07-001
First Posted: June 16, 2011    Key Record Dates
Last Update Posted: December 15, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Alexion Pharmaceuticals:
PNH
Paroxysmal Nocturnal hemoglobinuria
Soliris
 Eculizumab
Ravulizumab
Ultomiris
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
 Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases