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Pyruvate Kinase Deficiency Global Longitudinal Registry
Sponsor: Agios Pharmaceuticals, Inc.
Summary
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia. This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to 9 years. Data will be collected from participating Registry Physicians, participants, and, where appropriate, parents/guardians who have provided informed consent or assent (where relevant) and authorization pursuant to applicable laws and regulations. Data should include demographic, clinical, and treatment data; and other data of relevance to the management of patients with PK deficiency. Annual chart review and data entry are expected in order to enhance longitudinal understanding of PK deficiency; however, no specific protocol schedule of assessment is required by this Registry protocol.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
500
Start Date
2018-04-23
Completion Date
2027-05-31
Last Updated
2026-03-30
Healthy Volunteers
No
Conditions
Locations (52)
Phoenix Childrens Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Children's Hospital of Orange County
Orange, California, United States
Stanford University Medical Center
Palo Alto, California, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St Jude Children's Research Hospital
Memphis, Tennessee, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
University of Vermont Medical Center
Burlington, Vermont, United States
Saint Josephs Healthcare System
Hamilton, Ontario, Canada
Toronto General Hospital
Toronto, Ontario, Canada
St. Justine Hospital
Montreal, Quebec, Canada
Fakultni nemocnice Olomouc
Olomouc, Czechia
Ustav hematologie a krevni transfuze
Prague, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
Copenhagen University Hospital
Herlev, Denmark
Hopital Necker
Paris, France
Charite - Universitatsmedizin Berlin
Berlin, Germany
Evangelisches Krankenhaus Bielefeld gGmbH
Bielefeld, Germany
Universitatsklinikum Heidelberg
Heidelberg, Germany
Kinder- und Jugendarztpraxis
Munich, Germany
Universitatsklinikum Wurzburg
Würzburg, Germany
St James's Hospital
Dublin, Ireland
Presidio Ospedaliero di Pescara
Pescara, Abruzzo, Italy
AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
Naples, Campania, Italy
E O Ospedali Galliera
Genoa, Liguria, Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, Italy
Ospedale S Eugenio
Roma, Italy
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Centro Hospitalar E Universitario de Coimbra EPE
Coimbra, Portugal
Centro Hospitalar Lisboa Central- Hospital Dona Estefania
Lisbon, Portugal
Centro Hospitalar de Vila Nova de Gaia / Espinho E.P.E
Porto, Portugal
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, South Korea
Hospital Universitario Germans Trias i Pujol
Badalona, Barcelona, Spain
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Spain
Hospital de La Santa Creu i Sant Pau
Barcelona, Spain
Hospital Infantil Universitario Nino Jesus
Madrid, Spain
Hospital Universitario La Paz
Madrid, Spain
Hospital de Tortosa Verge de la Cinta
Tortosa, Spain
Centre Hospitalier Universitaire Vaudois
Lausanne, Switzerland
Siriraj Hospital Mahidol University
Bangkok, Thailand
Hacettepe University Medical Faculty
Ankara, Turkey (Türkiye)
Hammersmith Hospital
London, London, City of, United Kingdom
Kings College Hospital
London, United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom