A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

June 6, 2022

Primary Completion Date

December 13, 2024

Study Completion Date

January 31, 2030

Conditions
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Interventions
DRUG

Mitapivat

Tablets or granules

DRUG

Mitapivat-matching placebo

Tablets or granules

Trial Locations (19)

13353

Charite - UB - CVK - Medizinische Klinik, Berlin

19104

Children's Hospital of Philadelphia, Philadelphia

27710

Duke University Medical Center, Durham

28009

Hospital Universitario Infantil Nino Jesus, Madrid

30322

Children's Healthcare of Atlanta - Emory, Atlanta

33000

Hôpital Pellegrin, Bordeaux

38105

St Jude's Children's Research Hospital, Memphis

48304

Children's Hospital of Michigan, Detroit

60637

UChicago Medicine, Chicago

75390

UT Southwestern Medical Center, Dallas

77030

Texas Children's Hospital, Houston

80045

Children's Hospital Colorado, Aurora

94304

Stanford Medicine, Palo Alto

97080

Universitatsklinikum Wurzburg, Würzburg

02115

Boston Children's Hospital, Boston

QC H3T 1C5

Centre hospitalier Universitaire de Sainte-Justine, Montreal

3584 CX

Universitair Medisch Centrum Utrecht, Utrecht

08035

Hospital Universitario Vall d'Hebron, Barcelona

Unknown

CHUV University Hospital of Lausanne, Lausanne

Sponsors
All Listed Sponsors
lead

Agios Pharmaceuticals, Inc.

INDUSTRY