Pediatric Pyruvate Kinase DeficiencyPhase 3ACTIVATE-KidsTNCT05144256
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
A Phase 3 study testing Mitapivat in people with Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, run by Agios Pharmaceuticals, Inc. at 22 sites in 10 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
- What is being tested?
- Mitapivat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 49 participants
- Where
- 22 sitesin 10 countries
- Study dates
- 8 Jun 2022to Jun 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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 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). The second form of contraception can include an acceptable barrier method.
Who cannot join
- 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;
Show all 25Show fewer
- 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 known active hepatitis B or hepatitis C virus infection;
- Participants with known human immunodeficiency virus (HIV) infection;
- 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 exposure to gene therapy, or 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, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, Opadry® II Blue [hypromellose, titanium dioxide, lactose monohydrate, triacetin, and Food, Drug, and Cosmetics blue dye number 2 (FD\&C Blue #2)], Opadry® II White [hypromellose, titanium dioxide, lactose monohydrate, and triacetin], 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; also included are:
- Participants who are institutionalized by regulatory or court order.
- Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).
- Receiving a pyruvate kinase activator that has not been stopped for ≥52 weeks before providing informed consent/assent.
Trial locations and contacts
22 sites in 10 countries.
22 sites in 10 countries
United States9 sites
Children's Healthcare of Atlanta - Emory
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
UT Southwestern Medical Center
Dallas, Texas, 75390
Texas Children's Hospital
Houston, Texas, 77030
Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences
Las Vegas, Nevada, 89135
Weill Cornell Medical College
New York, New York, 10065
Stanford Medicine
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Turkey (Türkiye)3 sites
Hacettepe University
Ankara
İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health]
Istanbul, 34093
Ege University Faculty of Medicine
Izmir, Adana
Denmark2 sites
Aarhus University Hospital
Aarhus, Central Jutland, 8200
Rigshospitalet
Copenhagen, 2100
Spain2 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Clinico Universitario Virgen de la Arrixaca
Madrid, 30120
Canada1 site
Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario)
Ottawa, Ontario, K1H 5B2
Switzerland1 site
CHUV University Hospital of Lausanne
Lausanne, Canton of Bern
About this study
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
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 in the open-label extension (OLE) period.
- Being tested
- Mitapivat, Mitapivat-matching placebo
- Keywords
- Anemia, Hematologic Diseases, Metabolic Diseases, Mitapivat, AG-348, ACTIVATE-KidsT, PK Deficiency
- Medical subject headings
- Anemia, Hematologic Diseases, Metabolic Diseases, Pyruvate Kinase Deficiency of Red Cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MitapivatDrug
Also called AG-348, AG-348 sulfate hydrate, Mitapivat sulfate
Tablets or granules
Mitapivat-matching placeboDrug
Tablets or granules
| Study group | Role | What they receive |
|---|---|---|
| Mitapivat | Experimental | For participants randomized to receive mitapivat, dosing occurs orally twice daily (BID), and is based on age and weight. Dosing is optimized through 2 potential sequential increases in dose levels (1-5 mg, 4-20 mg and 10-50 mg) at Week 4 and Week 8, and aims to achieve the adult-equivalent exposure at 5, 20, and 50 mg. Following titration, participants remain on their individually optimized dose during the remainder of the Double-blind (DB) Period for 24 weeks. After the DB period, participants will enter an Open-label Extension (OLE) Period. To preserve blinding of treatment allocation, participants will continue mitapivat at their optimized dose and undergo mock titration with placebo for 8 weeks. At the conclusion of 8 weeks, participants may continue receiving mitapivat in the OLE Period for up to 262 weeks (including up to 2 weeks of dose taper). Receives: Drug: Mitapivat; Drug: Mitapivat-matching placebo |
| Placebo | Placebo comparator | For participants randomized to receive matched placebo, dosing is identical to that described above for mitapivat. Following the initial dose titration period, participants remain on their individually optimized dose during the remainder of the DB period for 32 weeks. After the DB period, participants will enter an OLE period. To preserve blinding of treatment allocation, participants will continue placebo at their optimized dose and undergo mitapivat dose optimization through 2 potential sequential increases in dose levels (1-5 mg, 4-20 mg and 10-50 mg) at Week 4 and Week 8 of the OLE Period, and aims to achieve the adult-equivalent exposure at 5, 20, and 50 mg. At the conclusion of 8 weeks, participants may continue receiving mitapivat in the OLE Period for up to 262 weeks (including up to 2 weeks of dose taper). Receives: Drug: Mitapivat; Drug: Mitapivat-matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 9 to Week 32
Secondary outcomes (26)
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.
Measured: Week 9 to Week 32
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.
Measured: Week 9 to Week 32
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.
Measured: Week 9 to Week 32
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.
Measured: Week 9 to Week 32
Change From Baseline in Estradiol Concentration
Measured: Baseline up to Week 298
Change From Baseline in Estrone Concentration
Measured: Baseline up to Week 298
Change From Baseline in Total Testosterone Concentration
Measured: Baseline up to Week 298
Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First)
Measured: Baseline up to Week 298
Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age
Measured: Baseline up to Week 298
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.
Measured: Baseline up to Week 298
Percentage Number of Female Participants With Development of Ovarian Cysts
Measured: Baseline up to Week 298
Change From Baseline in the Size of Ovarian Cysts in Female Participants
Measured: Baseline up to Week 298
Change From Baseline in Height-for-age Z-score
Measured: Baseline up to Week 298
Change From Baseline in Weight-for-age Z-score
Measured: Baseline up to Week 298
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Measured: Baseline up to Week 298
Change From Baseline in Bone Mineral Density (BMD) Z-score
Measured: Baseline up to Week 298
Change from Baseline in Serum Iron Concentration
Measured: Baseline up to Week 292
Change from Baseline in Serum Ferritin Concentration
Measured: Baseline up to Week 292
Change from Baseline in Total Iron-binding Capacity
Measured: Baseline up to Week 292
Change from Baseline in Transferrin/Transferrin Saturation
Measured: Baseline up to Week 292
Change from Baseline in Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale
The PedsQL multidimensional fatigue scale yields a total score from age-appropriate validated questionnaire that asks for perceived fatigue within the domains of 'General', 'Sleep/Rest' and 'Cognitive'. Each domain consists of 6 questions that are rated from 0 to 4 (therefore total score can range from 0 to 72). A higher total score indicates greater fatigue (i.e., worse outcome).
Measured: Baseline up to Week 292
Change from Baseline in PedsQL Generic Core Scale (GCS)
PedsQL GCS is designed to measure health-related quality of life in pediatric participants and adolescents (2 to 18 years of age). It encompasses 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), and Teens (13-17 years). Depending on the participant's age, the questionnaire may be completed by parent/caregiver as appropriate. For the Toddler group, the PedsQL GCS consist of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consist of 23 items, with a 3-point Likert scale (0, 2, 4) for the young pediatric, and a 5-point Likert scale for the pediatric and teens groups. Scores are transformed on a scale from 0 to 100 where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Measured: Baseline up to Week 292
Population Pharmacokinetic (PK) Model Parameter Estimate: Maximum Plasma Concentration (Cmax) of Mitapivat
Measured: Weeks 2, 8, 12, and 16
Population PK Model Parameter Estimate: Area Under the Concentration-time Curve (AUC) Derived From Plasma Concentrations of Mitapivat
Measured: Weeks 2, 8, 12, and 16
Concentration at Steady State (Css) of Mitapivat
Measured: Week 16: 6 and 8 hours postdose
Trough Concentration (Ctrough) of Mitapivat
Measured: Week 8: ≤30 minutes predose; Week 12: ≤30 minutes predose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2021
- Study start8 Jun 2022
- Primary completion3 May 2024
- Study completionJun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Pyruvate Kinase DeficiencyStudies of the same condition, open and under way.
- More trials run by Agios Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Affairs, Study chair, Agios Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05144256
- Sponsor's protocol ID
- AG348-C-022
- Other IDs
- 2021-003265-36, 2024-515024-37-00
- Acronym
- ACTIVATE-KidsT
- Status last verified
- Oct 2026
- First submitted
- 22 Nov 2021
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05144256
- Acronym / sponsor ID
- ACTIVATE-KidsT / AG348-C-022
- Keywords
- Anemia, Hematologic Diseases, Metabolic Diseases, Mitapivat, AG-348, ACTIVATE-KidsT, PK Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Agios Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Jun 2022 (actual)
- Primary completion
- 3 May 2024 (actual)
- Study completion
- Jun 2029 (estimated)
- First posted
- 3 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 in the open-label extension (OLE) period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mitapivat | Experimental | Drug: Mitapivat, Drug: Mitapivat-matching placeboFor participants randomized to receive mitapivat, dosing occurs orally twice daily (BID), and is based on age and weight. Dosing is optimized through 2 potential sequential increases in dose levels (1-5 mg, 4-20 mg and 10-50 mg) at Week 4 and Week 8, and aims to achieve the adult-equivalent exposure at 5, 20, and 50 mg. Following titration, participants remain on their individually optimized dose during the remainder of the Double-blind (DB) Period for 24 weeks. After the DB period, participants will enter an Open-label Extension (OLE) Period. To preserve blinding of treatment allocation, participants will continue mitapivat at their optimized dose and undergo mock titration with placebo for 8 weeks. At the conclusion of 8 weeks, participants may continue receiving mitapivat in the OLE Period for up to 262 weeks (including up to 2 weeks of dose taper). |
| Placebo | Placebo comparator | Drug: Mitapivat, Drug: Mitapivat-matching placeboFor participants randomized to receive matched placebo, dosing is identical to that described above for mitapivat. Following the initial dose titration period, participants remain on their individually optimized dose during the remainder of the DB period for 32 weeks. After the DB period, participants will enter an OLE period. To preserve blinding of treatment allocation, participants will continue placebo at their optimized dose and undergo mitapivat dose optimization through 2 potential sequential increases in dose levels (1-5 mg, 4-20 mg and 10-50 mg) at Week 4 and Week 8 of the OLE Period, and aims to achieve the adult-equivalent exposure at 5, 20, and 50 mg. At the conclusion of 8 weeks, participants may continue receiving mitapivat in the OLE Period for up to 262 weeks (including up to 2 weeks of dose taper). |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (25 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MitapivatAlso: AG-348, AG-348 sulfate hydrate, Mitapivat sulfateTablets or granules | Drug | Mitapivat, Placebo |
| Mitapivat-matching placeboTablets or granules | Drug | Mitapivat, Placebo |
Primary outcome
Percentage of Participants Achieving Transfusion Reduction Response (TRR)
Time frame: Week 9 to Week 32
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.
26 secondary outcomes
Percentage of Participants With Transfusion-free Response
Time frame: Week 9 to Week 32
Change in the Number of Transfusion Episodes
Time frame: Week 9 to Week 32
Percentage Change in Weight-normalized and Study Treatment Duration-normalized Total Transfusion Volume
Time frame: Week 9 to Week 32
Percentage of Participants With Normal Hemoglobin (Hb) Response
Time frame: Week 9 to Week 32
Change From Baseline in Estradiol Concentration
Time frame: Baseline up to Week 298
Change From Baseline in Estrone Concentration
Time frame: Baseline up to Week 298
Change From Baseline in Total Testosterone Concentration
Time frame: Baseline up to Week 298
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
Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age
Time frame: Baseline up to Week 298
Change From Baseline in Sexual Maturity Rating with Tanner Stage
Time frame: Baseline up to Week 298
Percentage Number of Female Participants With Development of Ovarian Cysts
Time frame: Baseline up to Week 298
Change From Baseline in the Size of Ovarian Cysts in Female Participants
Time frame: Baseline up to Week 298
Change From Baseline in Height-for-age Z-score
Time frame: Baseline up to Week 298
Change From Baseline in Weight-for-age Z-score
Time frame: Baseline up to Week 298
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Time frame: Baseline up to Week 298
Change From Baseline in Bone Mineral Density (BMD) Z-score
Time frame: Baseline up to Week 298
Change from Baseline in Serum Iron Concentration
Time frame: Baseline up to Week 292
Change from Baseline in Serum Ferritin Concentration
Time frame: Baseline up to Week 292
Change from Baseline in Total Iron-binding Capacity
Time frame: Baseline up to Week 292
Change from Baseline in Transferrin/Transferrin Saturation
Time frame: Baseline up to Week 292
Change from Baseline in Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale
Time frame: Baseline up to Week 292
Change from Baseline in PedsQL Generic Core Scale (GCS)
Time frame: Baseline up to Week 292
Population Pharmacokinetic (PK) Model Parameter Estimate: Maximum Plasma Concentration (Cmax) of Mitapivat
Time frame: Weeks 2, 8, 12, and 16
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
Concentration at Steady State (Css) of Mitapivat
Time frame: Week 16: 6 and 8 hours postdose
Trough Concentration (Ctrough) of Mitapivat
Time frame: Week 8: ≤30 minutes predose; Week 12: ≤30 minutes predose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Pediatric Pyruvate Kinase Deficiency
Active, not recruitingPhase 3
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
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Affairs study chair
Agios Pharmaceuticals, Inc.
22 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mitapivat in people with Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, run by Agios Pharmaceuticals, Inc. at 22 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Turkey (Türkiye), Denmark, Spain, Canada and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Achieving Transfusion Reduction Response (TRR) (time frame: Week 9 to Week 32). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2026.