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非輸血依存性アルファまたはベータサラセミア（α-またはβ-NTDT）の参加者におけるミタピバットの有効性と安全性を評価する研究

2026年10月29日 更新者：Agios Pharmaceuticals, Inc.

非輸血依存性アルファサラセミアまたはベータサラセミア（ENERGIZE）の被験者におけるミタピバットの有効性と安全性を評価する第3相、二重盲検、無作為化、プラセボ対照、多施設試験

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).

調査の概要

状態

積極的、募集していない

条件

介入・治療

詳細な説明

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

研究の種類

介入

入学 (実際)

194

段階

  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

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

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

18年歳以上 (大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

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)

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：4倍

武器と介入

参加者グループ / アーム
介入・治療
実験的：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.
薬：Mitapivat

Tablets

他の名前：
  • AG-348
  • AG-348硫酸塩水和物
  • 硫酸ミタピバット
プラセボコンパレーター：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.
薬：Mitapivat

Tablets

他の名前：
  • AG-348
  • AG-348硫酸塩水和物
  • 硫酸ミタピバット
薬：Placebo Matching Mitapivat

Tablets

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
時間枠：Double-Blind Period: Baseline up to Week 12 through Week 24

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

二次結果の測定

結果測定
メジャーの説明
時間枠
二重盲検期間：Mitapivatの血漿濃度
時間枠：二重盲検期間: 12週目に投与前。投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間: 12週目に投与前。投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：Mitapivatの最後の定量化可能濃度（TLAST）の時間
時間枠：二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：Mitapivatの最大観測血漿濃度（CMAX）
時間枠：二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間: ミタピバットの観察された最大血漿濃度 (Tmax) に達するまでの時間
時間枠：二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：Mitapivatの最後の定量化可能な血漿濃度（CLAST）
時間枠：二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間：アデノシン三リン酸（ATP）の血液濃度
時間枠：二重盲検期間: 1日目に投与前。 12週目の投与前。投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間: 1日目に投与前。 12週目の投与前。投与前、投与後0.5、1、3、5、7時間、20週目
二重盲検期間: 2,3 - ジホスホグリセリン酸 (2,3-DPG) の血中濃度
時間枠：二重盲検期間：1日目の事前投与。 12週目の事前投与。 20週目の投与後、0.5、1、3、5、7時間
二重盲検期間：1日目の事前投与。 12週目の事前投与。 20週目の投与後、0.5、1、3、5、7時間
Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline up to Week 12 through Week 24

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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: 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
時間枠：Double-Blind Period: 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: Baseline, 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
時間枠：Double-Blind Period: From the time of signing informed consent to Week 24

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: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mitapivat
時間枠：Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20

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
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
時間枠：Open-Label Extension Period: From week 24 up to end of study (approximately 5 years)

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)

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Agios Pharmaceuticals, Inc.

捜査官

  • スタディチェア：Medical Affairs、Agios Pharmaceuticals, Inc.

出版物と役立つリンク

研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。

一般刊行物

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2021年12月20日

一次修了 (実際)

2023年11月13日

研究の完了 (推定)

2028年12月1日

試験登録日

最初に提出

2021年2月18日

QC基準を満たした最初の提出物

2021年2月22日

最初の投稿 (実際)

2021年2月25日

学習記録の更新

投稿された最後の更新 (実際)

2026年10月30日

QC基準を満たした最後の更新が送信されました

2026年10月29日

最終確認日

2026年10月1日

詳しくは

本研究に関する用語

追加の関連 MeSH 用語

その他の研究ID番号

  • AG348-C-017
  • 2021-000211-23 (EudraCT番号)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

いいえ

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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