A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

PHASE3CompletedINTERVENTIONAL
Enrollment

80

Participants

Timeline

Start Date

August 9, 2018

Primary Completion Date

October 9, 2020

Study Completion Date

October 9, 2020

Conditions
Pyruvate Kinase DeficiencyAnemia, Hemolytic
Interventions
DRUG

Placebo

Placebo matching AG-348 tablets, administered to maintain the blind.

DRUG

AG-348

AG-348 tablets.

Trial Locations (46)

1011

Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne

2730

University of Copenhagen, Herlev Hospital, Herlev

3584

Universitair Medisch Centrum Utrecht, Utrecht

10700

Department of Paediatrics and Thalassaemia Center, Faculty of Medicine Siriraj, Bangkok

13385

Hôpital de la Timone, Marseille

16128

Ospedale Galliera, Genova

20122

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan

27705

Duke University, Durham

27858

East Carolina University, Greenville

28046

Hospital Universitario La Paz, Madrid

30120

Hospital Clinico Universitario Virgen de la Arricaxa, El Palmar

30322

Emory University, Atlanta

42415

Yeungnam University Hospital, Daegu 705-703

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

46260

Indiana Hemophilia and Thrombosis Center, Indianapolis

48304

Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit

72205

University of Arkansas for Medical Sciences, Little Rock

77030

Houston Methodist Research Institute, Houston

80054

CHU Amiens Picardie, Amiens

80138

"AOU Policlinico, Università della Campania Luigi Vanvitelli", Napoli

84112

Primary Children's Hospital Univ. of Utah, Salt Lake City

94010

Hôpital Henri-Mondor, Créteil

94609

Addenbrooke's Hospital, Cambridge

97080

Universitätsklinikum Würzburg, Würzburg

98109

Seattle Cancer Care Alliance, Seattle

02114

Massachusetts General Hospital, Boston

02115

The Children's Hospital Corporation d/b/a Boston's Children Hospital, Boston

Unknown

Hospital Central da Faculdade de Medicina USP Cidade Universitaria, São Paulo

Institute of Hematology and Blood Transfusion, Prague

Hopital Saint-Andre, Bordeaux

Institut Claudius Regaud, Toulouse

Charite University Medicine, Berlin

Tohoku University Hospital, Sendai

Kyoto Katsura Hospital, Kyoto

Agios Investigative Site, Mie

Kansai Medical University, Department of Pediatrics, Hirakata Hospital, Osaka

Toho University Omori Medical Center, Tokyo

Hacettepe University, Ankara

L8S 4LB

McMaster University - Health Sciences Centre, Hamilton

M5G 2C4

Toronto General Hospital, University Health Network, Toronto

534-0021

Osaka City General Hospital, Osaka

08035

Hospital U. Vall d'Hebron Servicio de Hematología Clínica, Barcelona

L7 8XP

The Royal Liverpool and Broadgreen University, Liverpool

W12 0NN

Imperial College Healthcare NHS Trust, Hammersmith Hospital, London

University College London, London

M13 9WL

Manchester Royal Infirmary, Manchester

Sponsors
All Listed Sponsors
lead

Agios Pharmaceuticals, Inc.

INDUSTRY