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수혈 비의존성 알파 또는 베타 지중해빈혈(α- 또는 β-NTDT) 환자를 대상으로 Mitapivat의 효능 및 안전성을 평가하는 연구

2026년 10월 29일 업데이트: Agios Pharmaceuticals, Inc.

비수혈 의존성 알파 또는 베타 지중해빈혈(ENERGIZE) 대상자에서 Mitapivat의 효능 및 안전성을 평가하는 3상, 이중 맹검, 무작위, 위약 대조, 다기관 연구

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).

연구 개요

상태

모집하지 않고 적극적으로

정황

개입 / 치료

상세 설명

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

연구 유형

중재적

등록 (실제)

194

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

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

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

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)

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 네 배로

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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.
의약품: Mitapivat

Tablets

다른 이름들:
  • AG-348
  • AG-348 황산염 수화물
  • 미타피밧 설페이트
위약 비교기: 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.
의약품: Mitapivat

Tablets

다른 이름들:
  • AG-348
  • AG-348 황산염 수화물
  • 미타피밧 설페이트
의약품: Placebo Matching Mitapivat

Tablets

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
기간: 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

2차 결과 측정

결과 측정
측정값 설명
기간
이중 맹검 기간: Mitapivat의 혈장 농도
기간: 이중 맹검 기간 : 12 주차의 사전 복용; 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간 : 12 주차의 사전 복용; 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간: Mitapivat의 정량화 가능한 마지막 농도(Tlast) 시간
기간: 이중 맹검 기간: 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: Mitapivat의 관찰된 최대 혈장 농도(Cmax)
기간: 이중 맹검 기간 : 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간 : 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간 : Mitapivat의 최대 관찰 된 혈장 농도 (TMAX)에 도달하는 시간
기간: 이중 맹검 기간 : 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간 : 사전 복용량, 0.5, 1, 3, 5, 7 시간 20 주차에 복용량 후 7 시간
이중 맹검 기간: Mitapivat의 정량 가능한 마지막 혈장 농도(Clast)
기간: 이중 맹검 기간: 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 아데노신 삼인산(ATP)의 혈액 농도
기간: 이중 맹검 기간: 투여 전 1일차; 12주차에 사전 투여; 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 투여 전 1일차; 12주차에 사전 투여; 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 2,3 - 디포스포글리세레이트(2,3-DPG)의 혈액 농도
기간: 이중 맹검 기간: 투여 전 1일차; 12주차에 사전 투여; 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
이중 맹검 기간: 투여 전 1일차; 12주차에 사전 투여; 투여 전, 투여 후 20주차, 0.5, 1, 3, 5, 7시간
Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Agios Pharmaceuticals, Inc.

수사관

  • 연구 의자: Medical Affairs, Agios Pharmaceuticals, Inc.

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

일반 간행물

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2021년 12월 20일

기본 완료 (실제)

2023년 11월 13일

연구 완료 (추정된)

2028년 12월 1일

연구 등록 날짜

최초 제출

2021년 2월 18일

QC 기준을 충족하는 최초 제출

2021년 2월 22일

처음 게시됨 (실제)

2021년 2월 25일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 10월 30일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 10월 29일

마지막으로 확인됨

2026년 10월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

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

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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