Scientists track rare blood disorder in 500 patients worldwide

NCT ID NCT03481738

Ongoing Knowledge-focused Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

Summary

This study is creating a long-term registry to track people with pyruvate kinase deficiency, a rare inherited anemia. It will follow up to 500 patients for 2 to 9 years to collect information on their health, treatments, and daily life. The goal is to better understand how the disease progresses over time and what treatments work best, which will help doctors improve care for future patients.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

AOU dell'Universita degli Studi della Campania Luigi Vanvitelli

Naples, Campania, Italy
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Centre Hospitalier Universitaire Vaudois

Lausanne, CH 1011, Switzerland
Centro Hospitalar E Universitario de Coimbra EPE

Coimbra, 3041-853, Portugal
Centro Hospitalar Lisboa Central- Hospital Dona Estefania

Lisbon, 1169-045, Portugal
Centro Hospitalar de Vila Nova de Gaia / Espinho E.P.E

Porto, 4200-072, Portugal
Charite - Universitatsmedizin Berlin

Berlin, 13353, Germany
Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States
Children's Hospital of Michigan

Detroit, Michigan, 48201, United States
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Copenhagen University Hospital

Herlev, 2730, Denmark
Duke University Medical Center

Durham, North Carolina, 27710, United States
E O Ospedali Galliera

Genoa, Liguria, 16128, Italy
Evangelisches Krankenhaus Bielefeld gGmbH

Bielefeld, 33617, Germany
Fakultni nemocnice Olomouc

Olomouc, 779 00, Czechia
Fakultni nemocnice v Motole

Prague, 150 06, Czechia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Hacettepe University Medical Faculty

Ankara, Turkey (Türkiye)
Hammersmith Hospital

London, London, City of, W12 0HS, United Kingdom
Hopital Necker

Paris, 75743, France
Hospital Infantil Universitario Nino Jesus

Madrid, 28009, Spain
Hospital Sant Joan de Deu - PIN

Esplugues de Llobregat, Barcelona, 08950, Spain
Hospital Universitario Germans Trias i Pujol

Badalona, Barcelona, 08916, Spain
Hospital Universitario La Paz

Madrid, 28046, Spain
Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain
Hospital de La Santa Creu i Sant Pau

Barcelona, 8041, Spain
Hospital de Tortosa Verge de la Cinta

Tortosa, 43500, Spain
Kinder- und Jugendarztpraxis

Munich, 81377, Germany
Kings College Hospital

London, SE5 9RS, United Kingdom
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Ospedale S Eugenio

Roma, 144, Italy
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Presidio Ospedaliero di Pescara

Pescara, Abruzzo, 65125, Italy
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Saint Josephs Healthcare System

Hamilton, Ontario, L8N 4A6, Canada
Siriraj Hospital Mahidol University

Bangkok, Thailand
St James's Hospital

Dublin, D08 NHY1, Ireland
St Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
St. Justine Hospital

Montreal, Quebec, H3T 1C5, Canada
Stanford University Medical Center

Palo Alto, California, 94304, United States
The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, South Korea
The Newcastle Upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, NE1 4LP, United Kingdom
Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada
UMass Memorial Medical Center

Worcester, Massachusetts, 01655-0002, United States
Universitair Medisch Centrum Utrecht

Utrecht, 3508 GA, Netherlands
Universitatsklinikum Heidelberg

Heidelberg, 69120, Germany
Universitatsklinikum Wurzburg

Würzburg, 97080, Germany
University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States
University of Vermont Medical Center

Burlington, Vermont, 05401, United States
Ustav hematologie a krevni transfuze

Prague, 128 20, Czechia

Conditions

Explore the condition pages connected to this study.

PYRUVATE KINASE DEFICIENCY