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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 : January 16, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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 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 2020
Estimated Study Completion Date : December 2020


Group/Cohort
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



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
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 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.

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
Contact: Alexion Pharmaceuticals, Inc. PNHregistry@alxn.com

Locations
United States, Connecticut
Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations. Recruiting
New Haven, Connecticut, United States, 06510
Contact: PNH Registry         
Contact       PNHRegistry@Alxn.com   
Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
Study Director: Jing Marantz Alexion Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01374360     History of Changes
Other Study ID Numbers: M07-001
First Posted: June 16, 2011    Key Record Dates
Last Update Posted: January 16, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Alexion Pharmaceuticals:
PNH
Paroxysmal Nocturnal hemoglobinuria
Soliris
Eculizumab

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases