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저위험 골수이형성 증후군(LR-MDS)으로 인한 빈혈 환자의 AG-946에 대한 연구

2026년 9월 25일 업데이트: Agios Pharmaceuticals, Inc.

2a/2b상, 오픈 라벨, 개념 증명(2a상) 및 이중 맹검, 무작위, 위약 대조(2b상), 다기관, 효능 및 안전성 연구 -위험 골수이형성 증후군

This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect of tebapivat on transfusion independence (TI) in participants with LR-MDS in phase 2b.

연구 개요

상태

모집하지 않고 적극적으로

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

87

단계

  • 2 단계

연락처 및 위치

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

연구 장소

  • 그리스
      • Alexandroupoli, 그리스
        • University Hospital of Alexandroupolis
      • Athens, 그리스
        • Attikon University General Hospital
      • Pátrai, 그리스
        • University General Hospital of Patras
      • Thessaloniki, 그리스
        • Hippokration Hospital
  • 대한민국
      • Daegu, 대한민국, 41944
        • Kyungpook National University Hospital
      • Seoul, 대한민국, 05505
        • Asan Medical Center - PPDS
      • Seoul, 대한민국
        • The Catholic University of Korea, Seoul St. Mary's Hospital
  • 독일
    • Lower Saxony
      • Hanover, Lower Saxony, 독일, 30625
        • Medizinische Hochschule Hannover
    • North Rhine-Westphalia
      • Düsseldorf, North Rhine-Westphalia, 독일, 40225
        • Universitatsklinikum Dusseldorf
    • Saxony
      • Leipzig, Saxony, 독일, 04103
        • Universitatsklinikum Leipzig
  • 미국
    • California
      • Lakewood, California, 미국, 90805
        • Innovative Clinical Research Institute Whittier
      • Los Angeles, California, 미국, 90024
        • David Geffen School of Medicine at UCLA
      • Redlands, California, 미국, 92373
        • Emad Ibrahim, MD, Inc.
    • Connecticut
      • New Haven, Connecticut, 미국, 06510
        • Smilow Cancer Hospital at Yale New Haven
    • Florida
      • Jacksonville, Florida, 미국, 32224
        • Mayo Clinic Jacksonville - PPDS
    • Illinois
      • Skokie, Illinois, 미국, 60076
        • Edward H. Kaplan MD & Associates
    • Missouri
      • St Louis, Missouri, 미국, 63110
        • Washington University School of Medicine
    • New York
      • Long Island City, New York, 미국, 11101
        • Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Durham, North Carolina, 미국, 27705
        • Duke Adult Blood and Marrow Clinic
  • 스페인
      • Córdoba, 스페인, 14004
        • C.H. Regional Reina Sofia - PPDS
      • Madrid, 스페인, 28046
        • Hospital Universitario La Paz - PPDS
      • Madrid, 스페인, 28050
        • Hospital Universitario HM Sanchinarro - CIOCC
      • Salamanca, 스페인, 37007
        • Complejo Asistencial Universitario de Salamanca - H. Clinico
      • Seville, 스페인, 41013
        • Hospital Universitario Virgen del Rocio - PPDS
  • 영국
      • Edinburgh, 영국, EH24 2XU
        • Western General Hospital Edinburgh - PPDS
      • London, 영국, SE5 9RS
        • Kings College Hospital
      • Oxford, 영국, OX3 7LE
        • Churchill Hospital-NHS Oxford
    • Aberdeen City
      • Aberdeen, Aberdeen City, 영국, AB25 2ZN
        • Aberdeen Royal Infirmary - PPDS
  • 오스트리아
    • Upper Austria
      • Linz, Upper Austria, 오스트리아, 4020
        • Ordensklinikum Linz GmbH Elisabethinen
  • 이스라엘
      • Jerusalem, 이스라엘, 9103102
        • Shaare Zedek Medical Center
      • Tel Aviv, 이스라엘
        • Tel Aviv Sourasky Medical Center PPDS
  • 이탈리아
      • Roma, 이탈리아
        • Fondazione PTV Policlinico Tor Vergata
    • Lombardy
      • Milan, Lombardy, 이탈리아
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
      • Pavia, Lombardy, 이탈리아
        • Fondazione IRCCS Policlinico San Matteo di Pavia
      • Rozzano, Lombardy, 이탈리아
        • Istituto Clinico Humanitas
    • Piedmont
      • Turin, Piedmont, 이탈리아
        • Azienda Ospedaliera Ordine Mauriziano di Torino
  • 체코
      • Ostrava, 체코, 708 52
        • Fakultni nemocnice Ostrava
  • 폴란드
    • Masovian Voivodeship
      • Warsaw, Masovian Voivodeship, 폴란드
        • MTZ Clinical Research Powered by PRATIA - PPDS
    • Silesian Voivodeship
      • Katowice, Silesian Voivodeship, 폴란드
        • Pratia Onkologia Katowice - PRATIA - PPDS
    • Warmian-Masurian Voivodeship
      • Olsztyn, Warmian-Masurian Voivodeship, 폴란드
        • SPZOZ MiSWiA z Warminsko-Mazurskim Centrum Onkologii w Olsztynie
  • 프랑스
      • Lille, 프랑스, 59037
        • CHRU Lille
      • Paris, 프랑스, 75475
        • Hôpital Saint Louis
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, 프랑스, 13010
        • Hôpital de La Conception
    • Maine-et-Loire
      • Angers, Maine-et-Loire, 프랑스, 49933
        • CHU Angers
  • 호주
    • Victoria
      • Clayton, Victoria, 호주, 3168
        • Monash Health, Monash Medical Centre

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:

Phase 2a

  1. At least 18 years of age at the time of providing informed consent;

  2. Documented diagnosis of myelodysplastic syndromes (MDS) according to World Health Organization (WHO) classification (Arber et al, 2016), that meets Revised International Prognostic Scoring System (IPSS-R) classification of lower-risk disease (risk score: ≤3.5) and <5% blasts as determined by the participant's bone marrow biopsy/aspirate during the Screening Period;

  3. Nontransfused or with low transfusion burden (LTB), based on transfusion history from the participant's medical record, according to revised International Working Group (IWG) 2018 criteria:

    • Nontransfused (NTD): <3 red blood cell (RBC) units in the 16-week period before administration of the first dose of study drug and no transfusions in the 8-week period before administration of the first dose of study drug, or
    • LTB: 3 to 7 RBC units in the 16-week period before administration of the first dose of study drug and <4 RBC units in the 8-week period before administration of the first dose of study drug;

  4. A hemoglobin (Hb) concentration <11.0 grams per deciliter (g/dL) during the 4-week Screening Period;

  5. Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0, 1, or 2;

  6. If taking iron chelation therapy, the iron chelation therapy dose must have been stable and started ≥56 days before administration of the first dose of study drug;

  7. Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a highly effective method of contraception from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug; if the highly effective method of contraception is hormonal contraception, then an acceptable barrier method must also be used. Men with partners who are WOCBP must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a condom from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug;

  8. Written informed consent from the participant before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study.

Phase 2b

  1. At least 18 years of age at the time of providing informed consent;

  2. Documented diagnosis of MDS according to WHO classification (Arber et al, 2016), that meets IPSS-R classification of lower-risk disease (risk score: ≤3.5) and <5% blasts as determined by the participant's bone marrow biopsy/aspirate during the Screening Period;

  3. With LTB, or high transfusion burden (HTB), based on transfusion history from the participant's medical record, according to revised IWG 2018 criteria:

    1. LTB: 3 to 7 RBC units from at least 2 transfusion episodes in the 16-week period before administration of the first dose of study drug AND <4 RBC units in the 8-week period before administration of the first dose of study drug, or
    2. HTB: ≥8 RBC units in the 16-week period before administration of the first dose of study drug AND ≥4 RBC units in the 8-week period before administration of the first dose of study drug

    If a participant's transfusion burden does not fall into either the LTB or HTB category, as defined per IWG 2018 criteria, then the transfusion burden will be categorized based on their transfusion history in the 16-week period before administration of the first dose of study drug.

  4. Pretransfusion Hb concentration available for a minimum of 2 and at least half (50%) of the transfusions received in the 16-week period before administration of the first dose of study drug

  5. A Hb concentration <10.0 g/dL during the 4-week Screening Period;

  6. Up to 2 prior therapies including erythropoiesis-stimulating agents (ESAs) (eg, erythropoietin [EPO], EPO + granulocyte colony-stimulating factor [G-CSF]) and/or luspatercept;

  7. ECOG Performance Status score of 0, 1, or 2;

  8. If taking iron chelation therapy, the iron chelation therapy dose must have been stable and started ≥56 days before administration of the first dose of study drug;

  9. WOCBP must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a highly effective, from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug; if the highly effective method of contraception is hormonal contraception, then an acceptable barrier method must be used. Men with partners who are WOCBP must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a condom from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug;

  10. Written informed consent from the participant before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study.

Exclusion Criteria:

Phase 2a

  1. Known history of acute myeloid leukemia (AML);

  2. Secondary MDS, defined as MDS that is known to have arisen as a result of chemical injury or treatment with chemotherapy and/or radiation for other diseases;

  3. Prior exposure to a pyruvate kinase activator and/or disease-modifying agents for underlying MDS:

    • Immunomodulatory drugs (IMiDs) such as lenalidomide; at the Investigator's discretion and in consultation with the Medical Monitor, participants who received ≤1 week of treatment with IMiDs may not be excluded, provided their last dose was ≥8 weeks before administration of the first dose of study drug
    • Hypomethylating agents (HMAs); at the Investigator's discretion and in consultation with the Medical Monitor, participants who received ≤2 doses of HMAs may not be excluded, provided that their last dose was ≥8 weeks before administration of the first dose of study drug
    • Isocitrate dehydrogenase (IDH) inhibitors
    • Immunosuppressive therapy (IST)
    • Allogeneic or autologous stem cell transplant;

  4. Currently receiving treatment with ESAs±G-CSF and/or luspatercept. Treatment with ESAs±G-CSF must have been stopped for ≥28 days before administration of the first dose of study drug; treatment with luspatercept must have been stopped for ≥65 days before administration of the first dose of study drug;

  5. History of active and/or uncontrolled cardiac or pulmonary disease within 6 months before providing informed consent, including but not limited to:

    • New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia
    • Myocardial infarction, unstable angina pectoris, or unstable hypertension; high risk thrombosis; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism
    • Heart rate-corrected QT interval using Fridericia's method of ≥470 milliseconds for female participants and ≥450 milliseconds for male participants, except for right or left bundle branch block
    • Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis >50%
    • Severe pulmonary hypertension as defined by severe symptoms associated with hypoxia, right-sided heart failure, and oxygen indicated;

  6. History of hepatobiliary disorders, as defined by:

    • Serum aspartate aminotransferase (AST) >2.5 × upper limit of normal (ULN) (unless due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase (ALT) >2.5 × ULN (unless due to hepatic iron deposition)
    • Serum bilirubin >ULN, if the elevation is associated with clinically symptomatic choledocholithiasis, cholecystitis, biliary obstruction, or hepatocellular disease;

  7. Renal dysfunction, as defined by an estimated glomerular filtration rate (eGFR) <45 milliliters per minute (mL/min)/1.73 m^2;

  8. Active infection requiring systemic antimicrobial therapy at the time of providing informed consent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before administration of the first dose of study drug;

  9. Major surgery within 12 weeks before administration of the first dose of study drug. Participants must have completely recovered from any previous surgery before administration of the first dose of study drug;

  10. For any malignancy except MDS: History of malignancy (active or treated) ≤5 years before providing informed consent for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ;

  11. Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg);

  12. Positive test for HIV-1 Ab or HIV-2 Ab;

  13. Absolute neutrophil count (ANC) <500/microliter (μL) (0.5 × 10^9/L);

  14. Platelet count ≤75,000/μL during Screening (75 × 10^9/L) platelet transfusions within 28 days before Screening or during Screening;

  15. Nonfasting triglyceride concentration >500 mg/dL;

  16. Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug;

  17. Current enrollment or past participation (within 4 weeks or a time frame equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug or, whichever is longer) in any other clinical study involving an investigational treatment or device;

  18. Known allergy to tebapivat or its excipients;

  19. Pregnant or breastfeeding;

  20. 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 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).

Phase 2b

  1. Known history of AML;

  2. Secondary MDS, defined as MDS that is known to have arisen as a result of chemical injury or treatment with chemotherapy and/or radiation for other diseases;

  3. Prior exposure to a pyruvate kinase activator, including exposure to tebapivat in the Phase 2a part of this study, and/or disease-modifying agents for underlying MDS:

    • Imetelstat; at the Investigator's discretion and in consultation with the Medical Monitor, participants who received ≤2 doses of imetelstat may not be excluded, provided that their last dose was ≥8 weeks before administration of the first dose of study drug
    • IMiDs such as lenalidomide; at the Investigator's discretion and in consultation with the Medical Monitor, participants who received ≤1 week of treatment with IMiDs may not be excluded, provided their last dose was ≥8 weeks before administration of the first dose of study drug
    • HMAs; at the Investigator's discretion and in consultation with the Medical Monitor, participants who received ≤2 doses of HMAs may not be excluded, provided that their last dose was ≥8 weeks before administration of the first dose of study drug
    • IDH inhibitors
    • IST
    • Allogeneic or autologous stem cell transplant;

  4. Currently receiving treatment with ESAs±G-CSF and/or luspatercept. Treatment with ESAs±G-CSF must have been stopped for ≥28 days before administration of the first dose of study drug; treatment with luspatercept must have been stopped for ≥65 days before administration of the first dose of study drug;

  5. History of active and/or uncontrolled cardiac or pulmonary disease within 6 months before providing informed consent, including but not limited to:

    • New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia
    • Myocardial infarction, unstable angina pectoris, or unstable hypertension; high risk thrombosis; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism
    • Heart rate-corrected QT interval using Fridericia's method of ≥470 milliseconds for female participants and ≥450 milliseconds for male participants, except for right or left bundle branch block
    • Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis >50%
    • Severe pulmonary hypertension as defined by severe symptoms associated with hypoxia, right-sided heart failure, and oxygen indicated

  6. History of hepatobiliary disorders, as defined by:

    • Serum AST >2.5 × ULN (unless due to hemolysis and/or hepatic iron deposition) and ALT >2.5 × ULN (unless due to hepatic iron deposition)
    • Serum bilirubin >ULN, if the elevation is associated with clinically symptomatic choledocholithiasis, cholecystitis, biliary obstruction, or hepatocellular disease

  7. Renal dysfunction, as defined by an eGFR <45 mL/min/1.73 m^2;

  8. Active infection requiring systemic antimicrobial therapy at the time of providing informed consent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before administration of the first dose of study drug;

  9. Major surgery within 12 weeks before administration of the first dose of study drug. Participants must have completely recovered from any previous surgery before administration of the first dose of study drug;

  10. For any malignancy except MDS: History of malignancy (active or treated) ≤5 years before providing informed consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.;

  11. Positive test for HCV Ab with evidence of active HCV infection, or positive test for HBsAg;

  12. Positive test for HIV-1 Ab or HIV-2 Ab;

  13. ANC <500/μL (0.5 × 10^9/L);

  14. Platelet count < 75,000/μL (75 × 10^9 /L) during Screening; platelet transfusions within 28 days before Screening or during Screening;

  15. Nonfasting triglyceride concentration >500 mg/dL;

  16. Receiving inhibitors of P-gp that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer) beforeadministration of the first dose of study drug;

  17. Current enrollment or past participation (within 4 weeks or a time frame equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug or, whichever is longer) in any other clinical study involving an investigational treatment or device;

  18. Known allergy to tebapivat or its excipients;

  19. Pregnant or breastfeeding;

  20. 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 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).

  21. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, autoimmune or hereditary hemolytic anemia, hypothyroidism, or any type of known clinically significant bleeding.

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: 무작위화되지 않음
  • 중재 모델: 순차적 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: Core Period: Phase 2a - Tebapivat 5 mg

Participants will receive 5 milligrams (mg) tebapivat orally, once daily for up to 16 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
의약품: Tebapivat

Tebapivat Tablet

다른 이름들:
  • AG-946
실험적: Core Period: Phase 2b - Tebapivat 10 mg

Participants will receive 10 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
의약품: Tebapivat

Tebapivat Tablet

다른 이름들:
  • AG-946
실험적: Core Period: Phase 2b - Tebapivat 15 mg

Participants will receive 15 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
의약품: Tebapivat

Tebapivat Tablet

다른 이름들:
  • AG-946
실험적: Core Period: Phase 2b - Tebapivat 20 mg

Participants will receive 20 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
의약품: Tebapivat

Tebapivat Tablet

다른 이름들:
  • AG-946

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Phase 2a: Proportion of Participants With Hemoglobin (Hb) Response
기간: Baseline, Week 8 through Week 16

Hb response is defined as a ≥1.5-grams per deciliter (g/dL) increase from baseline in the average Hb concentration from Week 8 through Week 16.
Baseline, Week 8 through Week 16
Phase 2a: Proportion of Participants With Transfusion Independence During the Core Period
기간: Up to 16 weeks

Transfusion Independence is defined as transfusion-free for ≥8 consecutive weeks during the Core Period (participants With Low Transfusion Burden [LTB] only).
Up to 16 weeks
Phase 2b: Proportion of Participants With Transfusion Independence
기간: Up to 24 weeks

Transfusion independence, defined as transfusion-free for ≥8 consecutive weeks (TI8) during the Core Period.
Up to 24 weeks

2차 결과 측정

결과 측정
측정값 설명
기간
2a 단계: 핵심 기간 동안 Hb 농도의 기준선에서 변경
기간: 기준선 최대 16주
기준선 최대 16주
2a단계: 핵심 기간 동안 총 수혈된 적혈구(RBC) 단위의 기준선에서 변경
기간: 기준선 최대 16주
기준선 최대 16주
2a상: 핵심 기간 동안 2,3-디포스포글리세르산(2,3-DPG)의 전혈 농도
기간: 1일 및 8주(투약 전 60분 이하 및 투약 후 0.5, 1, 2, 3, 4, 6 및 8시간) 및 2, 4, 12 및 16주(투약 전 60분 이하)
1일 및 8주(투약 전 60분 이하 및 투약 후 0.5, 1, 2, 3, 4, 6 및 8시간) 및 2, 4, 12 및 16주(투약 전 60분 이하)
2a상: 핵심 기간 동안 아데노신 삼인산(ATP)의 전혈 농도
기간: 1일 및 8주(투약 전 60분 이하 및 투약 후 0.5, 1, 2, 3, 4, 6 및 8시간) 및 2, 4, 12 및 16주(투약 전 60분 이하)
1일 및 8주(투약 전 60분 이하 및 투약 후 0.5, 1, 2, 3, 4, 6 및 8시간) 및 2, 4, 12 및 16주(투약 전 60분 이하)
2b상: 기준선에서 8주차부터 24주차까지 수혈된 총 RBC 단위의 변화
기간: 기준선, 8주차부터 24주차까지
기준선, 8주차부터 24주차까지
2a단계: 핵심 기간 동안 실험실에 이상이 있는 참가자의 비율
기간: 최대 16주
최대 16주
2a단계: 8주차부터 16주차까지 2회 연속 시점에서 기준선보다 Hb 농도가 1.5g/dL 이상 증가한 참가자의 비율
기간: 기준선, 8주차부터 16주차까지
기준선, 8주차부터 16주차까지
2a단계: 기준선과 비교하여 핵심 기간 동안 연속 8주 이상 동안 총 수혈된 적혈구 단위가 50% 이상 감소한 참가자의 비율
기간: 기준 최대 16주
기준 최대 16주
2a단계: 핵심 기간 동안 테바피바트의 혈장 농도
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2a단계: 핵심 기간 동안 테바피바트의 최대(피크) 농도(Cmax)
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2a단계: 핵심 기간 동안 테바피바트의 Cmax(tmax)까지 걸리는 시간
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2a단계: 핵심 기간 동안 테바피바트의 0~t시간(AUC0-t) 농도-시간 곡선 아래 면적
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2a 단계: 핵심 기간 중 0부터 테바피바트 투여 간격(AUC0-τ) 종료까지의 농도-시간 곡선 아래 영역
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2a단계: 핵심 기간 동안 테바피바트의 명백한 최종 제거 반감기(t½)
기간: 1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
1일차 및 8주차(투약 전 60분 이하, 투약 후 0.5, 1, 2, 3, 4, 6, 8시간) 및 2, 4, 12, 16주차(투약 전 60분 이하)
2b단계: 핵심 기간 동안 실험실에 이상이 있는 참가자의 비율
기간: 최대 24주
최대 24주
2b단계: 핵심 기간 동안 Hb 농도의 기준선 대비 변화
기간: 기준 최대 24주
기준 최대 24주
2b단계: 기준선과 비교하여 핵심 기간 동안 연속 8주 이상 동안 총 수혈된 적혈구 단위가 50% 이상 감소한 참가자의 비율
기간: 기준 최대 24주
기준 최대 24주
2b단계: 핵심 기간 동안 첫 번째 TI8까지의 시간
기간: 기준 최대 24주
기준 최대 24주
2b단계: 핵심 기간 동안 연속 12주 이상(TI12) 수혈을 받지 않은 참가자의 비율
기간: 최대 24주
최대 24주
2b단계: 기준선과 비교하여 핵심 기간 동안 연속 12주 이상 동안 총 수혈된 적혈구 단위가 50% 이상 감소한 참가자의 비율
기간: 기준 최대 24주
기준 최대 24주
2b단계: 핵심 기간 동안 첫 번째 TI12까지의 시간
기간: 기준 최대 24주
기준 최대 24주
2b단계: 핵심 기간 동안 테바피바트의 혈장 농도
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 테바피바트의 최대(피크) 농도(Cmax)
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 테바피바트의 Cmax(tmax)까지 걸리는 시간
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 테바피바트의 0~t시간(AUC0-t) 농도-시간 곡선 아래 면적
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b 단계: 핵심 기간 중 0부터 테바피바트 투여 간격(AUC0-τ) 종료까지의 농도-시간 곡선 아래 영역
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 테바피바트의 명백한 최종 제거 반감기(t½)
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 2,3-디포스포글리세레이트(2,3-DPG)의 전혈 농도
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
2b단계: 핵심 기간 동안 아데노신 삼인산(ATP)의 전혈 농도
기간: 1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
1일차부터 20주차까지 투여 전 및 투여 후 여러 시점
Phase 2a: Proportion of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Discontinuation During the Core Period
기간: Up to 16 weeks

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An SAE is any AE or suspected adverse reaction that results in death, is immediately life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly/birth defect, or is considered an important medical event.
Up to 16 weeks
Phase 2a: Proportion of Participants With Hb 1.0+ Response
기간: Baseline, Week 8 through Week 16

Hb 1.0+ response is defined as a ≥1.0-g/dL increase from baseline in the average Hb concentration from Week 8 through Week 16
Baseline, Week 8 through Week 16
Phase 2b: Proportion of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Discontinuation During the Core Period
기간: Up to 24 weeks

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An SAE is any AE or suspected adverse reaction that results in death, is immediately life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly/birth defect, or is considered an important medical event.
Up to 24 weeks
Phase 2b: Duration of Transfusion Independence (TI)
기간: Baseline up to 24 weeks

The duration of TI will be calculated as the number of days in the longest transfusion-free period starting on or after the start of study treatment through the end of the Core Period.
Baseline up to 24 weeks

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Agios Pharmaceuticals, Inc.

수사관

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

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)

2022년 11월 7일

기본 완료 (실제)

2026년 3월 17일

연구 완료 (추정된)

2029년 3월 1일

연구 등록 날짜

최초 제출

2022년 7월 26일

QC 기준을 충족하는 최초 제출

2022년 8월 4일

처음 게시됨 (실제)

2022년 8월 5일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 9월 28일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 25일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • AG946-C-002
  • 2022-500609-42-00 (기타 식별자: EU-CTR)

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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