A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

PHASE3CompletedINTERVENTIONAL
Enrollment

27

Participants

Timeline

Start Date

June 26, 2018

Primary Completion Date

November 12, 2020

Study Completion Date

November 12, 2020

Conditions
Pyruvate Kinase DeficiencyAnemia, Hemolytic
Interventions
DRUG

AG-348

"Part 1 (Dose Optimization Period): Participants began by receiving 5 mg orally, BID. Each participant's dose of AG-348 was sequentially increased to 20 mg BID followed by 50 mg BID depending on their response to AG-348 and their tolerance.~Part 2 (Fixed Dose Period): Optimized dose determined in Part 1."

Trial Locations (19)

2730

University of Copenhagen, Herlev Hospital, Herlev

3584

Universitair Medisch Centrum Utrecht, Utrecht

10700

Department of Paediatrics and Thalassaemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok

13385

Hôpital de la Timone, Marseille

20122

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan

30322

Emory University, Atlanta

80131

AORN Cardarelli, Napoli

Ospedale Galliera, Napoli

80138

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

94010

Hopital Universitaire Henri Mondor, Créteil

94609

UCSF Benioff Children's Hospital, Oakland

98109

Seattle Cancer Care Alliance, Seattle

02115

Massachusetts General Hospital, Boston

M5G 2C4

Toronto General Hospital, University Health Network, Toronto

Unknown

St James's Hospital Department of Haematology, Dublin

CB2 0QQ

Addenbrooke's Hospital, Cambridge

W12 0NN

Imperial College Healthcare NHS Trust, Hammersmith Hospital, London

WC1E 6BT

University College London, London

M13 9WL

Manchester Royal Infirmary, Manchester

Sponsors
All Listed Sponsors
lead

Agios Pharmaceuticals, Inc.

INDUSTRY