Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01374360
Recruitment Status : Recruiting
First Posted : June 16, 2011
Last Update Posted : April 12, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date April 15, 2011
First Posted Date June 16, 2011
Last Update Posted Date April 12, 2019
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 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
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 *

*   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, Older Adult
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: Phillipe Gustovic Alexion Pharmaceuticals
PRS Account Alexion Pharmaceuticals
Verification Date April 2019