A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (ENERGIZE)

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)

The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

Study Overview

• Status: Active, not recruiting
• Conditions: Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
• Intervention / Treatment: Drug: Mitapivat; Drug: Placebo Matching Mitapivat

Detailed Description

The mitapivat group included 130 participants whereas the placebo group had 64 participants.

• Study Type: Interventional
• Enrollment (Actual): 194
• Phase: Phase 3

Contacts and Locations

Study Locations

• Brazil
  • Caxias do Sul, Brazil, 95070-560
    • Universidade de Caxias do Sul
  • Ribeirão Preto, Brazil, 14051-260
    • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
  • Rio de Janeiro, Brazil, 20211-030
    • HEMORIO Instituto Nacional de Hematologia
  • Santo André, Brazil, 09090-790
    • Praxis Pesquisa Medica
  • São Paulo, Brazil, 04006-002
    • GSH Banco de Sangue de São Paulo
  • São Paulo, Brazil, 05403-000
    • Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
• Bulgaria
  • Kyustendil, Bulgaria, 2500
    • MHAT "Dr. Nikola Vasiliev" AD
  • Sofia, Bulgaria, 1756
    • SHATHD Sofia
• Canada
  • Toronto, Canada, M5G 2C4
    • Toronto General Hospital, University Health Network
• Denmark
  • Copenhagen, Denmark, 2100
    • Rigshospitalet
• France
  • Créteil, France, 94010
    • CHU Hôpital Henri Mondor
  • Lyon, France, 69003
    • Hopital Edouard Herriot, CHU de Lyon
• Greece
  • Athens, Greece, 115 27
    • Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School
  • Athens, Greece, 115 26
    • Laiko General Hospital
  • Rio, Greece, 26504
    • University General Hospital of Patras
  • Thessaloniki, Greece, 546 42
    • Ippokrateio General Hospital
• Italy
  • Brindisi, Italy, 72100
    • Ospedale "A. Perrino" - Brindisi
  • Cagliari, Italy, 09121
    • Ospedale Pediatrico Microcitemico
  • Ferrara, Italy, 44124
    • Ospedale Sant'Anna
  • Genova, Italy, 16128
    • Ente Ospedaliero Ospedali Galliera
  • Milan, Italy, 20122
    • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
  • Modena, Italy, 41124
    • A.O.U Di Modena
  • Naples, Italy, 80138
    • AOU L. Vanvitelli Universita degli Studi della Campania Luigi Vanvitelli
  • Naples, Italy, 80131
    • A.O.R.N. "A. Cardarelli"
  • Orbassano, Italy, 10043
    • A.O.U. San Luigi Gonzaga
• Lebanon
  • Beirut, Lebanon, 9999
    • Chronic Care Center
• Malaysia
  • Alor Star, Malaysia, 05460
    • Hospital Sultanah Bahiyah
  • Ampang, Malaysia, 68000
    • Hospital Ampang
  • Johor Bahru, Malaysia, 80100
    • Hospital Sultanah Aminah Johor Bahru
  • Kota Kinabalu, Malaysia, 88586
    • Hospital Queen Elizabeth, Kota Kinabalu
  • Kuala Lumpur, Malaysia, 50300
    • Hospital Tunku Azizah
  • Kuantan, Malaysia, 25100
    • Hospital Tengku Ampuan Afzan
  • Kuching, Malaysia, 93586
    • Hospital Umum Sarawak
  • Pulau Pinang, Malaysia, 10990
    • Hospital Pulau Pinang
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Erasmus MC
  • Utrecht, Netherlands, 3584 CX
    • Universitair Medisch Centrum Utrecht
• Saudi Arabia
  • Al Mubarraz, Saudi Arabia, 36428
    • King Abdulaziz Hospital - Al Ahsa
  • Riyadh, Saudi Arabia, 14611
    • King Abdullah International Medical Research Center
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitario Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Murcia, Spain, 30120
    • Hospital Universitario Virgen Arrixaca
  • Seville, Spain, 41013
    • Hospital Universitario Virgen del Rocio
• Taiwan
  • Taichung, Taiwan, 40447
    • China Medical University, Taiwan
• Thailand
  • Bangkok, Thailand, 10400
    • Phramongkutklao Hospital
  • Bangkok, Thailand, 10400
    • Ramathibodi Hospital
  • Bangkok, Thailand, 10700
    • Faculty of Medicine Siriraj Hospital
  • Chiang Mai, Thailand, 50200
    • Maharaj Nakorn Chiang Mai Hospital
  • Khon Kaen, Thailand, 40002
    • Srinagarind Hospital, Khon Kaen University
  • Mueang Phitsanulok, Thailand, 65000
    • Naresuan University Hospital
  • Pathum Wan, Thailand, 10330
    • King Chulalongkorn Memorial Hospital
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01130
    • Acibadem Adana Hospital
  • Antalya, Turkey (Türkiye), 07059
    • Akdeniz University Faculty of Medicine
  • Balcalı, Turkey (Türkiye), 01330
    • Çukurova University
  • Bornova, Turkey (Türkiye), 35040
    • Ege University Faculty of Medicine
  • Fatih, Turkey (Türkiye), 34093
    • Istanbul University Faculty of Medicine
  • Mersin, Turkey (Türkiye), 06230
    • Hacettepe University
• United Arab Emirates
  • Abu Dhabi, United Arab Emirates
    • Burjeel Medical City
  • Dubai, United Arab Emirates, 4545
    • Thalassemia Centre Dubai
• United Kingdom
  • London, United Kingdom, NW1 2PG
    • University College London
  • London, United Kingdom, W12 OHS
    • Imperial College Healthcare NHS Trust - Hammersmith Hospital
  • CAM
    • Cambridge, CAM, United Kingdom, CB2 0QQ
      • Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
  • LAN
    • Manchester, LAN, United Kingdom, M13 9WL
      • Manchester Royal Infirmary, Manchester University NHS Foundation Trust
• United States
  • California
    • La Jolla, California, United States, 92093
      • San Diego Hospital, UC San Diego Health
    • Palo Alto, California, United States, 94304-1601
      • Stanford Medicine
  • Massachusetts
    • Boston, Massachusetts, United States, 02114-2696
      • Massachusetts General Hospital
  • New York
    • New York, New York, United States, 10065-4870
      • Weill Cornell Medical Center
  • North Carolina
    • Durham, North Carolina, United States, 27710-3038
      • Duke University Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Penn Medicine - University of Pennsylvania Health System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H [HbH] disease) based on Hb electrophoresis, Hb high-performance liquid chromatography (HPLC)), and/or deoxyribonucleic acid (DNA) analysis;
• Hb concentration ≤10.0 grams per deciliter (g/dL) (100.0 grams per liter [g/L]), based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period;
• Non-transfusion-dependent, defined as ≤5 red blood cell (RBC) units during the 24-week period before randomization; and no RBC transfusions ≤8 weeks before providing informed consent and no RBC transfusions during the Screening Period;
• If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization;
• Women of child-bearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, one of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method;
• Written informed consent before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study.

Exclusion Criteria:

• Pregnant, breastfeeding, or parturient
• Documented history of homozygous or heterozygous sickle hemoglobin (HbS) or hemoglobin C (HbC);
• Prior exposure to gene therapy or prior bone marrow or stem cell transplantation;
• Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization;
• Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization;
• History of malignancy, (active or treated) ≤5 years before providing informed consent;
• History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ;
• Hepatobiliary disorders;
• Estimated glomerular filtration rate <45 milliliters per minute (mL/min)/1.73 m^2 by Chronic Kidney Disease Epidemiology Collaboration creatinine equation;
• Nonfasting triglycerides >440 milligrams per deciliter (mg/dL) (5 millimoles per liter [mmol/L]);
• Active infection requiring systemic antimicrobial therapy at the time of providing informed consent;
• Positive test for hepatitis C virus antibody (HCVAb) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg);
• Positive test for human immunodeficiency virus (HIV)-1 antibody (Ab) or HIV-2 Ab;
• History of major surgery (including splenectomy) ≤16 weeks before providing informed consent and/or a major surgical procedure planned during the study;
• Current enrollment or past participation (≤12 weeks before administration of the first dose of study drug or a timeframe equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational treatment or device;
• Receiving strong CYP3A4/5 inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer); or strong CYP3A4 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization;
• Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed. The testosterone dose and preparation must be stable for ≥10 weeks before randomization;
• Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate, Opadry® II Blue [hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD&C Blue #2]);

• Any medical, hematological, 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 excluded 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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mitapivat; Mitapivat 100 milligrams (mg), orally, twice daily (BID) for 24 weeks in double blind (DB) period and for up to 5 years in open label extension (OLE) period.	Drug: Mitapivat; Tablets; Other Names: AG-348; AG-348 sulfate hydrate; Mitapivat sulfate
Placebo Comparator: Placebo; Placebo matching mitapivat, orally, BID for 24 weeks in double blind period followed by Mitapivat 100 mg, orally, BID for up to 5 years in open label extension period.	Drug: Mitapivat; Tablets; Other Names: AG-348; AG-348 sulfate hydrate; Mitapivat sulfate; Drug: Placebo Matching Mitapivat; Tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline	Hb response is defined as ≥10 grams/ liter (g/L) (1.0 gram per deciliter) (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline. Hb response was tested using the Mantel-Haenszel stratum weighted method adjusting for randomization stratification factors. Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline up to Week 12 through Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24	The FACIT-Fatigue subscale includes a 13-item self-reported fatigue subscale, which assesses the severity and impact of fatigue (including the impact on daily activities and functioning).The FACIT-Fatigue subscale is scored on a 5-point Likert scale: 0 (not at all) to 4 (very much). The total FACIT-Fatigue subscale score ranges from 0 to 52, with a higher score indicating better health-related quality of life (HRQOL). Baseline is defined as the last assessment before randomization for subjects randomized and not dosed or the last assessment before start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 12 through Week 24
Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 12 through Week 24
Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline	Hb 1.5+ response is defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline. Hb 1.5+ response will be summarized using the Mantel-Haenszel stratum weighted method adjusting for randomization stratification factors. Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline up to Week 12 through Week 24
Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Haptoglobin at Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Reticulocytes at Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Erythropoietin at Week 24	Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Percentage of Participants Who Achieved Patient Global Impression of Severity (PGIS)- Fatigue Response at Weeks 12, 16, 20, and 24	PGIS-Fatigue measured participants' perception of their fatigue severity (7-day recall) on a 4-point scale ranging from '1=none' to '4=severe'. A participant was considered to have achieved the PGIS-Fatigue response at Weeks 12, 16, 20, or 24, if their baseline to postbaseline score met one of the following conditions: 'none' at baseline to 'none' postbaseline; 'mild' to 'mild' or 'none'; 'moderate' to 'mild' or 'none'; or 'severe' to 'moderate', 'mild', or 'none'.	Double-Blind Period: At Weeks 12, 16, 20, and 24
Double-Blind Period: Percentage of Participants Who Achieved the Patient Global Impression of Change (PGIC)- Fatigue Response at Weeks 12, 16, 20, and 24	The PGIC-Fatigue assesses change over time compared with baseline on a 5-point scale ranging from 0 to 4 where 0 indicates Much better and 4 as Much worse. A participant was considered to have achieved the PGIC-Fatigue response at Weeks 12, 16, 20, or 24 if their baseline PGIS and corresponding PGIC met one of the following conditions: if the PGIS at baseline was 'none' or 'mild' and PGIC at the visit was 'no change', 'a little better', or 'much better'; or if the PGIS at baseline was 'moderate' or 'severe' and PGIC at the visit was 'a little better' or 'much better'.	Double-Blind Period: At Weeks 12, 16, 20, and 24
Double-Blind Period: Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 24	The 6MWT is a well-established performance outcome (PerfO) measure that is widely used to evaluate physical activity in terms of distance walked in patients with a variety of conditions. The test measures the distance an individual can walk on a hard, flat surface in 6 minutes.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Serum Ferritin at Week 24	Iron metabolism was assessed based on serum ferritin levels.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Change From Baseline in Transferrin Saturation (TSAT) at Week 24	Iron metabolism was assessed based on TSAT levels. Transferrin saturation is reported by dividing value of serum iron by total iron binding capacity.	Double-Blind Period: Baseline, Week 24
Double-Blind Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3	AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) is any untoward medical occurrence that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and includes both serious & non-serious TEAEs. Severity of AEs was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE; Version 4.03): grade 1: mild; grade 2: moderate; grade 3: severe or medically significant but not immediately life-threatening; grade 4: life threatening or disabling; grade 5: death related to AE.	Double-Blind Period: From the time of signing informed consent to Week 24
Double-Blind Period: Plasma Concentration of Mitapivat		Double-Blind Period: Pre-dose at Week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mitapivat	Area under the concentration-time curve from time zero to Tlast on dosing day, calculated using the linear-log trapezoidal rule.	Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Time of Last Quantifiable Concentration (Tlast) of Mitapivat		Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Maximum Observed Plasma Concentration (Cmax) of Mitapivat		Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mitapivat		Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Last Quantifiable Plasma Concentration (Clast) of Mitapivat		Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Blood Concentration of Adenosine Triphosphate (ATP)		Double-Blind Period: Pre-dose at Day 1; pre-dose at Week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Double-Blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)		Double-Blind Period: Pre-dose at Day 1; pre-dose at Week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
Open-Label Extension Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity of Greater Than or Equal to Grade 3	AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) is any untoward medical occurrence that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and includes both serious & non-serious TEAEs. Severity of abnormalities was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE; Version 4.03): grade 1: mild; grade 2: moderate; grade 3: severe or medically significant but not immediately life-threatening; grade 4: life threatening or disabling; grade 5: death related to AE.	Open-Label Extension Period: From week 24 up to end of study (approximately 5 years)

Collaborators and Investigators

• Sponsor: Agios Pharmaceuticals, Inc.
• Investigators: Study Chair: Medical Affairs, Agios Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Taher AT, Al-Samkari H, Aydinok Y, Besser M, Boscoe AN, Dahlin JL, De Luna G, Estepp JH, Gheuens S, Gilroy KS, Glenthoj A, Sim Goh A, Iyer V, Kattamis A, Loggetto SR, Morris S, Musallam KM, Osman K, Ricchi P, Salido-Fierrez E, Sheth S, Tai F, Tevich H, Uhlig K, Urbstonaitis R, Viprakasit V, Cappellini MD, Kuo KHM; ENERGIZE investigators. Mitapivat in adults with non-transfusion-dependent alpha-thalassaemia or beta-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X. Epub 2025 Jun 19.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2021
• Primary Completion (Actual): November 13, 2023
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: February 18, 2021
• First Submitted That Met QC Criteria: February 22, 2021
• First Posted (Actual): February 25, 2021

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 28, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia; beta-Thalassemia; alpha-Thalassemia; Molecular Mechanisms of Pharmacological Action; Enzyme Activators; mitapivat
• Other Study ID Numbers: AG348-C-017; 2021-000211-23 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No