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Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is currently recruiting participants.
Verified November 2012 by Alexion Pharmaceuticals
Sponsor:
Collaborator:
ICON
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01374360
First received: April 15, 2011
Last updated: November 12, 2012
Last verified: November 2012
History of Changes
  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:

Drug Information available for: Eculizumab
U.S. FDA Resources

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) ] [ Designated as safety issue: Yes ]
    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.


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) ] [ Designated as safety issue: No ]
    Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.


Estimated Enrollment: 2000
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

Genders Eligible for Study:   Both
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. (Subjects under the age of eighteen years must have parent/legal guardian consent. Upon turning eighteen years of age, these subjects must be re-consented).
  • Ability to comprehend and sign consent to have data entered in the PNH Registry.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360

Contacts
Contact: Lynn Sanders 203-439-9609 sandersl@alxn.com

Locations
United States, Maryland
Johns Hopkins University Medical Center Recruiting
Baltimore, Maryland, United States
Sponsors and Collaborators
Alexion Pharmaceuticals
ICON
Investigators
Study Director: Stephen Squinto, PhD Alexion Pharmaceuticals
  More Information

No publications provided

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: November 12, 2012
Health Authority: Argentina: Human Research Bioethics Committee
Australia: Human Research Ethics Committee
Australia: National Health and Medical Research Council
Belgium: Ethics Committee
Canada: Ethics Review Committee
Finland: Ethics Committee
France: Comité consultatif sur le traitement de l'information en matière de recherche dans le domaine de la santé
France: Conseil National de l'Ordre des Médecins
Germany: Paul-Ehrlich-Institut
Netherlands: Independent Ethics Committee
Norway: Ethics Committee
Spain: Ethics Committee
Sweden: Regional Ethical Review Board
Switzerland: Swissmedic
United Kingdom: Research Ethics Committee
United States: Food and Drug Administration

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 May 21, 2013