Trial record 1 of 42 for:
"paroxysmal nocturnal hemoglobinuria"
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Verified April 2015 by Alexion Pharmaceuticals
ICON Clinical Research
Information provided by (Responsible Party):
First received: April 15, 2011
Last updated: April 1, 2015
Last verified: April 2015
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Paroxysmal Nocturnal Hemoglobinuria
||Observational Model: Cohort
Time Perspective: Prospective
||Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
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:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||December 2020 (Final data collection date for primary outcome measure)
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
Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris and withdrawn from treatment.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.
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
|Johns Hopkins University Medical Center
|Baltimore, Maryland, United States |
|Roswell Park Cancer Institute
|Buffalo, New York, United States |
|Contact: Erin Kittelman 716-845-1516 Erin.Kittelman@RoswellPark.org |
ICON Clinical Research
||Stephen Squinto, PhD
No publications provided by Alexion Pharmaceuticals
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in Pregnant Patients with Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-9. doi: 10.1056/NEJMoa1502950.
History of Changes
|Other Study ID Numbers:
|Study First Received:
||April 15, 2011
||April 1, 2015
||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
Netherlands: Independent Ethics Committee
Norway: Ethics Committee
Spain: Ethics Committee
Sweden: Regional Ethical Review Board
United Kingdom: Research Ethics Committee
United States: Food and Drug Administration
Keywords provided by Alexion Pharmaceuticals:
Paroxysmal Nocturnal hemoglobinuria
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on October 13, 2015
Bone Marrow Diseases
Signs and Symptoms