A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

49

Participants

Timeline

Start Date

June 8, 2022

Primary Completion Date

May 3, 2024

Study Completion Date

June 30, 2029

Conditions
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Interventions
DRUG

Mitapivat

Tablets or granules

DRUG

Mitapivat-matching placebo

Tablets or granules

Trial Locations (22)

2100

Rigshospitalet, Copenhagen

8200

Aarhus University Hospital, Aarhus

10065

Weill Cornell Medical College, New York

13353

Charite - UB - CVK - Medizinische Klinik, Berlin

19104

Children's Hospital of Philadelphia, Philadelphia

30120

Hospital Clinico Universitario Virgen de la Arrixaca, Madrid

30322

Children's Healthcare of Atlanta - Emory, Atlanta

34093

İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health], Istanbul

75390

UT Southwestern Medical Center, Dallas

77030

Texas Children's Hospital, Houston

85016

Phoenix Children's Hospital, Phoenix

89135

Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences, Las Vegas

94304

Stanford Medicine, Palo Alto

02115

Boston Children's Hospital, Boston

K1H 5B2

Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario), Ottawa

779 00

Fakultní Nemocnice Olomouc, Olomouc

3584 CX

Universitair Medisch Centrum Utrecht, Utrecht

08035

Hospital Universitario Vall d'Hebron, Barcelona

Unknown

CHUV University Hospital of Lausanne, Lausanne

Ege University Faculty of Medicine, Izmir

Hacettepe University, Ankara

SE5 9RS

King's College Hospital NHS, London

Sponsors
All Listed Sponsors
lead

Agios Pharmaceuticals, Inc.

INDUSTRY