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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 (ACTIVATE-KidsT)

Primary Purpose

Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Mitapivat
Mitapivat-matching placebo
Sponsored by
Agios Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pediatric Pyruvate Kinase Deficiency focused on measuring Anemia, Hematologic Diseases, Metabolic Diseases, Mitapivat, AG-348, ACTIVATE-KidsT, PK Deficiency

Eligibility Criteria

1 Year - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Written informed consent from the participant, or the participant's legally authorized representative, parent(s), or legal guardian, and the participant's assent, where applicable (informed consent/assent) must be obtained before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study;
  • Aged 1 to <18 years. Participants between 12 and 24 months of age must weigh a minimum of 7 kilograms (kg);
  • Clinical laboratory confirmation of pyruvate kinase deficiency (PKD), defined as documented presence of at least 2 mutant alleles in the pyruvate kinase L/R (PKLR) gene, of which at least 1 is a missense mutation, as determined per the genotyping performed by the study central genotyping laboratory;
  • Six to 26 transfusion episodes in the 52-week period before providing informed consent/assent;
  • Have complete records of transfusion history for the 52 weeks before providing informed consent/assent, defined as having all the following available: (1) all the transfusion dates, (2) the RBC transfusion volume (milliliters and/or number of units) for all the transfusions, and (3) hemoglobin concentrations within 1 week before transfusion for at least 80% of the transfusions;
  • Receiving folic acid supplementation as part of routine clinical care for at least 21 days before administration of the first dose of study drug, to be continued during study participation;
  • Female participants who have attained menarche and/or breast development in Tanner Stage 2, as well as male participants with partners who have attained menarche, must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent/assent, throughout the study, and for 28 days after the last dose of study drug (including the time required to dose taper) for female participants who have attained menarche and 90 days after the last dose of study drug (including the time required to dose taper) for male participants. The second form of contraception can include an acceptable barrier method.

Exclusion Criteria:

  • Pregnant or breastfeeding;
  • Homozygous for the R479H mutation or have 2 nonmissense mutations, without the presence of another missense mutation, in the PKLR gene as determined per the genotyping performed by the study central genotyping laboratory;
  • History of malignancy;
  • History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent;
  • Hepatobiliary disorders including, but not limited to:

    • Liver disease with histopathological evidence of cirrhosis or severe fibrosis;
    • Clinically symptomatic cholelithiasis or cholecystitis (participants with prior cholecystectomy are eligible);
    • History of drug-induced cholestatic hepatitis;
    • Aspartate aminotransferase >2.5×upper limit of normal (ULN) (unless due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase >2.5×ULN (unless due to hepatic iron deposition);
  • Renal dysfunction as defined by an estimated glomerular filtration rate <60 milliliters per minute (mL/min)/1.73 m^2;
  • Nonfasting triglycerides >440 milligrams per deciliter (mg/dL) (5 millimoles per liter [mmol/L]);
  • Active uncontrolled infection requiring systemic antimicrobial therapy;
  • Participants with a high likelihood of exposure to, or parental history of, hepatitis B or hepatitis C who subsequently test positive for hepatitis B antigen or hepatitis C virus antibody with signs of active hepatitis B or hepatitis C virus infection;
  • Participants with a high likelihood of exposure to, or parental history of, human immunodeficiency virus (HIV) who subsequently test positive for HIV-1 or -2 antibodies;
  • History of major surgery (including splenectomy) ≤6 months before providing informed consent/assent and/or planning on undergoing a major surgical procedure during the screening or double-blind period;
  • Current enrollment or past participation (within 90 days before the first dose of study drug or a time frame equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational study drug or device;
  • Prior bone marrow or stem cell transplantation;
  • Currently receiving hematopoietic stimulating agents; the last dose must have been administered at least 28 days or a time frame equivalent to 5 half-lives (whichever is longer) before randomization;
  • Receiving products that are strong inhibitors of CYP3A4/5 that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of CYP3A4 that have not been stopped for ≥28 days or a time frame equivalent to 5 half-lives (whichever is longer), before randomization;
  • Receiving anabolic steroids, including testosterone preparations, that have not been stopped for at least 28 days before randomization;
  • Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate);
  • Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data.

Sites / Locations

  • Phoenix Children's HospitalRecruiting
  • Stanford MedicineRecruiting
  • Children's Healthcare of Atlanta - EmoryRecruiting
  • Boston Children's HospitalRecruiting
  • Cure 4 the Kids Foundation, A Division of Roseman University of Health SciencesRecruiting
  • Weill Cornell Medical CollegeRecruiting
  • Children's Hospital of PhiladelphiaRecruiting
  • UT Southwestern Medical CenterRecruiting
  • Texas Children's HospitalRecruiting
  • Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario)Recruiting
  • Fakultní Nemocnice OlomoucRecruiting
  • Aarhus University HospitalRecruiting
  • RigshospitaletRecruiting
  • Charite - UB - CVK - Medizinische KlinikRecruiting
  • Universitair Medisch Centrum UtrechtRecruiting
  • Hospital Universitario Vall d'HebronRecruiting
  • Hospital Clinico Universitario Virgen de la ArrixacaRecruiting
  • CHUV University Hospital of LausanneRecruiting
  • Ege University Faculty of MedicineRecruiting
  • Hacettepe UniversityRecruiting
  • İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health]Recruiting
  • Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation TrustRecruiting
  • King's College Hospital NHSRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

Mitapivat

Placebo

Mitapivat (OLE period)

Arm Description

Double-Blind Period: Participants will receive mitapivat orally, at doses based on age and weight, for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.

Double-Blind Period: Participants will receive mitapivat-matching placebo orally for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.

Participants who have completed the double-blind period will be eligible to receive mitapivat for up to 5 years in the OLE period. Participants entering the OLE period will receive both mitapivat and placebo for 8 weeks to maintain the double-blind treatment assignment.

Outcomes

Primary Outcome Measures

Percentage of Participants Achieving Transfusion Reduction Response (TRR)
TRR is defined as ≥33% reduction in total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the double-blind period, normalized by weight and actual study drug duration compared with the historical transfusion volume, standardized by weight, and to 24 weeks.

Secondary Outcome Measures

Percentage of Participants With Transfusion-free Response
Transfusion-free response is defined as achievement of 0 transfusions administered from Week 9 through Week 32 of the double-blind period.
Change in the Number of Transfusion Episodes
The change in the number of transfusion episodes from Week 9 through Week 32 of the double-blind period compared with the historical number of transfusion episodes standardized to 24 weeks.
Percentage Change in Weight-normalized and Study Treatment Duration-normalized Total Transfusion Volume
The percentage change in weight-normalized and study treatment duration-normalized total transfusion volume from Week 9 through Week 32 of the double-blind period will be compared with the historical transfusion volume standardized by weight and to 24 weeks.
Percentage of Participants With Normal Hemoglobin (Hb) Response
Normal Hb response is defined as achievement of Hb concentrations in the normal range at least once, 8 weeks or more after a transfusion during Week 9 through Week 32 of the double-blind period.
Change From Baseline in Estradiol Concentration
Change From Baseline in Estrone Concentration
Change From Baseline in Total Testosterone Concentration
Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First)
Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age
Change From Baseline in Sexual Maturity Rating with Tanner Stage
Tanner Stage 1 corresponds to the prepubertal form, with progression to Tanner Stage 5, the final adult form.
Percentage Number of Female Participants With Development of Ovarian Cysts
Change From Baseline in the Size of Ovarian Cysts in Female Participants
Change From Baseline in Height-for-age Z-score
Change From Baseline in Weight-for-age Z-score
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Change From Baseline in Bone Mineral Density (BMD) Z-score
Change from Baseline in Serum Iron Concentration
Change from Baseline in Serum Ferritin Concentration
Change from Baseline in Total Iron-binding Capacity
Change from Baseline in Transferrin/Transferrin Saturation
Change from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score
The PKDD is a 7-item measure of the core signs and symptoms of pyruvate kinase deficiency (PK deficiency). The PKDD has been validated in adults with PK deficiency; the scoring algorithm for the pediatric version of the PKDD will be developed as part of the in-trial psychometric validation of the instrument.
Change from Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score
The PKDIA for pediatric participants is a 4-item patient-reported outcome measure of the common impacts of PK deficiency on activities of daily living. The PKDIA has been validated in adults with PK deficiency; the scoring algorithm for the pediatric version of the PKDIA will be developed as part of the in-trial psychometric validation of the instrument.
Population Pharmacokinetic (PK) Model Parameter Estimate: Maximum Plasma Concentration (Cmax) of Mitapivat
Population PK Model Parameter Estimate: Area Under the Concentration-time Curve (AUC) Derived From Plasma Concentrations of Mitapivat
Concentration at Steady State (Css) of Mitapivat
Trough Concentration (Ctrough) of Mitapivat

Full Information

First Posted
November 22, 2021
Last Updated
October 19, 2023
Sponsor
Agios Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05144256
Brief Title
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
Acronym
ACTIVATE-KidsT
Official Title
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 8, 2022 (Actual)
Primary Completion Date
February 2025 (Anticipated)
Study Completion Date
February 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Agios Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to < 6 years, 6 to < 12 years, 12 to < 18 years) and splenectomy status. Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 24-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Keywords
Anemia, Hematologic Diseases, Metabolic Diseases, Mitapivat, AG-348, ACTIVATE-KidsT, PK Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
45 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Mitapivat
Arm Type
Experimental
Arm Description
Double-Blind Period: Participants will receive mitapivat orally, at doses based on age and weight, for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Double-Blind Period: Participants will receive mitapivat-matching placebo orally for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.
Arm Title
Mitapivat (OLE period)
Arm Type
Experimental
Arm Description
Participants who have completed the double-blind period will be eligible to receive mitapivat for up to 5 years in the OLE period. Participants entering the OLE period will receive both mitapivat and placebo for 8 weeks to maintain the double-blind treatment assignment.
Intervention Type
Drug
Intervention Name(s)
Mitapivat
Other Intervention Name(s)
AG-348, AG-348 sulfate hydrate, Mitapivat sulfate
Intervention Description
Tablets or granules
Intervention Type
Drug
Intervention Name(s)
Mitapivat-matching placebo
Intervention Description
Tablets or granules
Primary Outcome Measure Information:
Title
Percentage of Participants Achieving Transfusion Reduction Response (TRR)
Description
TRR is defined as ≥33% reduction in total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the double-blind period, normalized by weight and actual study drug duration compared with the historical transfusion volume, standardized by weight, and to 24 weeks.
Time Frame
Week 9 to Week 32
Secondary Outcome Measure Information:
Title
Percentage of Participants With Transfusion-free Response
Description
Transfusion-free response is defined as achievement of 0 transfusions administered from Week 9 through Week 32 of the double-blind period.
Time Frame
Week 9 to Week 32
Title
Change in the Number of Transfusion Episodes
Description
The change in the number of transfusion episodes from Week 9 through Week 32 of the double-blind period compared with the historical number of transfusion episodes standardized to 24 weeks.
Time Frame
Week 9 to Week 32
Title
Percentage Change in Weight-normalized and Study Treatment Duration-normalized Total Transfusion Volume
Description
The percentage change in weight-normalized and study treatment duration-normalized total transfusion volume from Week 9 through Week 32 of the double-blind period will be compared with the historical transfusion volume standardized by weight and to 24 weeks.
Time Frame
Week 9 to Week 32
Title
Percentage of Participants With Normal Hemoglobin (Hb) Response
Description
Normal Hb response is defined as achievement of Hb concentrations in the normal range at least once, 8 weeks or more after a transfusion during Week 9 through Week 32 of the double-blind period.
Time Frame
Week 9 to Week 32
Title
Change From Baseline in Estradiol Concentration
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Estrone Concentration
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Total Testosterone Concentration
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First)
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Sexual Maturity Rating with Tanner Stage
Description
Tanner Stage 1 corresponds to the prepubertal form, with progression to Tanner Stage 5, the final adult form.
Time Frame
Baseline up to Week 298
Title
Percentage Number of Female Participants With Development of Ovarian Cysts
Time Frame
Baseline up to Week 298
Title
Change From Baseline in the Size of Ovarian Cysts in Female Participants
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Height-for-age Z-score
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Weight-for-age Z-score
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Time Frame
Baseline up to Week 298
Title
Change From Baseline in Bone Mineral Density (BMD) Z-score
Time Frame
Baseline up to Week 298
Title
Change from Baseline in Serum Iron Concentration
Time Frame
Baseline up to Week 292
Title
Change from Baseline in Serum Ferritin Concentration
Time Frame
Baseline up to Week 292
Title
Change from Baseline in Total Iron-binding Capacity
Time Frame
Baseline up to Week 292
Title
Change from Baseline in Transferrin/Transferrin Saturation
Time Frame
Baseline up to Week 292
Title
Change from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score
Description
The PKDD is a 7-item measure of the core signs and symptoms of pyruvate kinase deficiency (PK deficiency). The PKDD has been validated in adults with PK deficiency; the scoring algorithm for the pediatric version of the PKDD will be developed as part of the in-trial psychometric validation of the instrument.
Time Frame
Baseline up to Week 292
Title
Change from Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score
Description
The PKDIA for pediatric participants is a 4-item patient-reported outcome measure of the common impacts of PK deficiency on activities of daily living. The PKDIA has been validated in adults with PK deficiency; the scoring algorithm for the pediatric version of the PKDIA will be developed as part of the in-trial psychometric validation of the instrument.
Time Frame
Baseline up to Week 292
Title
Population Pharmacokinetic (PK) Model Parameter Estimate: Maximum Plasma Concentration (Cmax) of Mitapivat
Time Frame
Weeks 2, 8, 12, and 16
Title
Population PK Model Parameter Estimate: Area Under the Concentration-time Curve (AUC) Derived From Plasma Concentrations of Mitapivat
Time Frame
Weeks 2, 8, 12, and 16
Title
Concentration at Steady State (Css) of Mitapivat
Time Frame
Week 16: 6 and 8 hours postdose
Title
Trough Concentration (Ctrough) of Mitapivat
Time Frame
Week 8: ≤30 minutes predose; Week 12: ≤30 minutes predose

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Written informed consent from the participant, or the participant's legally authorized representative, parent(s), or legal guardian, and the participant's assent, where applicable (informed consent/assent) must be obtained before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study; Aged 1 to <18 years. Participants between 12 and 24 months of age must weigh a minimum of 7 kilograms (kg); Clinical laboratory confirmation of pyruvate kinase deficiency (PKD), defined as documented presence of at least 2 mutant alleles in the pyruvate kinase L/R (PKLR) gene, of which at least 1 is a missense mutation, as determined per the genotyping performed by the study central genotyping laboratory; Six to 26 transfusion episodes in the 52-week period before providing informed consent/assent; Have complete records of transfusion history for the 52 weeks before providing informed consent/assent, defined as having all the following available: (1) all the transfusion dates, (2) the RBC transfusion volume (milliliters and/or number of units) for all the transfusions, and (3) hemoglobin concentrations within 1 week before transfusion for at least 80% of the transfusions; Receiving folic acid supplementation as part of routine clinical care for at least 21 days before administration of the first dose of study drug, to be continued during study participation; Female participants who have attained menarche and/or breast development in Tanner Stage 2, as well as male participants with partners who have attained menarche, must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent/assent, throughout the study, and for 28 days after the last dose of study drug (including the time required to dose taper) for female participants who have attained menarche and 90 days after the last dose of study drug (including the time required to dose taper) for male participants. The second form of contraception can include an acceptable barrier method. Exclusion Criteria: Pregnant or breastfeeding; Homozygous for the R479H mutation or have 2 nonmissense mutations, without the presence of another missense mutation, in the PKLR gene as determined per the genotyping performed by the study central genotyping laboratory; History of malignancy; History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent; Hepatobiliary disorders including, but not limited to: Liver disease with histopathological evidence of cirrhosis or severe fibrosis; Clinically symptomatic cholelithiasis or cholecystitis (participants with prior cholecystectomy are eligible); History of drug-induced cholestatic hepatitis; Aspartate aminotransferase >2.5×upper limit of normal (ULN) (unless due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase >2.5×ULN (unless due to hepatic iron deposition); Renal dysfunction as defined by an estimated glomerular filtration rate <60 milliliters per minute (mL/min)/1.73 m^2; Nonfasting triglycerides >440 milligrams per deciliter (mg/dL) (5 millimoles per liter [mmol/L]); Active uncontrolled infection requiring systemic antimicrobial therapy; Participants with a high likelihood of exposure to, or parental history of, hepatitis B or hepatitis C who subsequently test positive for hepatitis B antigen or hepatitis C virus antibody with signs of active hepatitis B or hepatitis C virus infection; Participants with a high likelihood of exposure to, or parental history of, human immunodeficiency virus (HIV) who subsequently test positive for HIV-1 or -2 antibodies; History of major surgery (including splenectomy) ≤6 months before providing informed consent/assent and/or planning on undergoing a major surgical procedure during the screening or double-blind period; Current enrollment or past participation (within 90 days before the first dose of study drug or a time frame equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational study drug or device; Prior bone marrow or stem cell transplantation; Currently receiving hematopoietic stimulating agents; the last dose must have been administered at least 28 days or a time frame equivalent to 5 half-lives (whichever is longer) before randomization; Receiving products that are strong inhibitors of CYP3A4/5 that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of CYP3A4 that have not been stopped for ≥28 days or a time frame equivalent to 5 half-lives (whichever is longer), before randomization; Receiving anabolic steroids, including testosterone preparations, that have not been stopped for at least 28 days before randomization; Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate); Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Agios Medical Affairs
Phone
833-228-8474
Email
medinfo@agios.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Affairs
Organizational Affiliation
Agios Pharmaceuticals, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Name
Stanford Medicine
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Name
Children's Healthcare of Atlanta - Emory
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Name
Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89135
Country
United States
Individual Site Status
Recruiting
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Individual Site Status
Recruiting
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario)
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 5B2
Country
Canada
Individual Site Status
Recruiting
Facility Name
Fakultní Nemocnice Olomouc
City
Olomouc
ZIP/Postal Code
779 00
Country
Czechia
Individual Site Status
Recruiting
Facility Name
Aarhus University Hospital
City
Aarhus
State/Province
Midtjylland
ZIP/Postal Code
8200
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Rigshospitalet
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Charite - UB - CVK - Medizinische Klinik
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Individual Site Status
Recruiting
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Clinico Universitario Virgen de la Arrixaca
City
Madrid
ZIP/Postal Code
30120
Country
Spain
Individual Site Status
Recruiting
Facility Name
CHUV University Hospital of Lausanne
City
Lausanne
State/Province
Bern
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Ege University Faculty of Medicine
City
Izmir
State/Province
Adana
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Hacettepe University
City
Ankara
Country
Turkey
Individual Site Status
Recruiting
Facility Name
İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health]
City
İstanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation Trust
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
King's College Hospital NHS
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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