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Trial record 1 of 56 for:    "Paroxysmal nocturnal hemoglobinuria"
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Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01374360
First received: April 15, 2011
Last updated: February 17, 2017
Last verified: February 2017
  Purpose
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Condition
Paroxysmal Nocturnal Hemoglobinuria

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

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. In addition, all serious adverse events (SAEs), regardless of causality, will be collected in order to search for unknown safety concerns.


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.


Enrollment: 4700
Actual Study Start Date: January 2007
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
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

Detailed Description:
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
  Eligibility

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360

Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
Study Director: Jing Marantz Alexion Pharmaceuticals
  More Information

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
Study First Received: April 15, 2011
Last Updated: February 17, 2017
Individual Participant Data  
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

ClinicalTrials.gov processed this record on June 28, 2017