Badanie oceniające skuteczność i bezpieczeństwo Mitapivatu u uczestników z niezależną od transfuzji talasemią alfa lub beta (α- lub β-NTDT)
Podwójnie ślepe, randomizowane, wieloośrodkowe badanie fazy 3, oceniające skuteczność i bezpieczeństwo stosowania mitapivatu u osób z niezależną od transfuzji krwi talasemią alfa lub beta (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).
Przegląd badań
Status
Status
Warunki
Warunki
Interwencja / Leczenie
Interwencja / Leczenie
Szczegółowy opis
The mitapivat group included 130 participants whereas the placebo group had 64 participants.
Typ studiów
Typ studiów
Zapisy (Rzeczywisty)
Zapisy
Faza
Faza
- Faza 3
Kontakty i lokalizacje
Lokalizacje studiów
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Al Mubarraz, Arabia Saudyjska, 36428
- King Abdulaziz Hospital - Al Ahsa
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Riyadh, Arabia Saudyjska, 14611
- King Abdullah International Medical Research Center
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Caxias do Sul, Brazylia, 95070-560
- Universidade de Caxias do Sul
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Ribeirão Preto, Brazylia, 14051-260
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
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Rio de Janeiro, Brazylia, 20211-030
- HEMORIO Instituto Nacional de Hematologia
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Santo André, Brazylia, 09090-790
- Praxis Pesquisa Medica
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São Paulo, Brazylia, 04006-002
- GSH Banco de Sangue de São Paulo
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São Paulo, Brazylia, 05403-000
- Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
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Kyustendil, Bułgaria, 2500
- MHAT "Dr. Nikola Vasiliev" AD
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Sofia, Bułgaria, 1756
- SHATHD Sofia
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Copenhagen, Dania, 2100
- Rigshospitalet
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Créteil, Francja, 94010
- CHU Hôpital Henri Mondor
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Lyon, Francja, 69003
- Hopital Edouard Herriot, CHU de Lyon
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Athens, Grecja, 115 27
- Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School
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Athens, Grecja, 115 26
- Laiko General Hospital
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Rio, Grecja, 26504
- University General Hospital of Patras
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Thessaloniki, Grecja, 546 42
- Ippokrateio General Hospital
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Barcelona, Hiszpania, 08035
- Hospital Universitario Vall d'Hebron
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Madrid, Hiszpania, 28046
- Hospital Universitario La Paz
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Murcia, Hiszpania, 30120
- Hospital Universitario Virgen Arrixaca
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Seville, Hiszpania, 41013
- Hospital Universitario Virgen del Rocío
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Rotterdam, Holandia, 3015 GD
- Erasmus MC
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Utrecht, Holandia, 3584 CX
- Universitair Medisch Centrum Utrecht
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Toronto, Kanada, M5G 2C4
- Toronto General Hospital, University Health Network
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Beirut, Liban, 9999
- Chronic Care Center
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Alor Star, Malezja, 05460
- Hospital Sultanah Bahiyah
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Ampang, Malezja, 68000
- Hospital Ampang
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Johor Bahru, Malezja, 80100
- Hospital Sultanah Aminah Johor Bahru
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Kota Kinabalu, Malezja, 88586
- Hospital Queen Elizabeth, Kota Kinabalu
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Kuala Lumpur, Malezja, 50300
- Hospital Tunku Azizah
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Kuantan, Malezja, 25100
- Hospital Tengku Ampuan Afzan
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Kuching, Malezja, 93586
- Hospital Umum Sarawak
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Pulau Pinang, Malezja, 10990
- Hospital Pulau Pinang
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California
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La Jolla, California, Stany Zjednoczone, 92093
- San Diego Hospital, UC San Diego Health
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Palo Alto, California, Stany Zjednoczone, 94304-1601
- Stanford Medicine
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Massachusetts
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Boston, Massachusetts, Stany Zjednoczone, 02114-2696
- Massachusetts General Hospital
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New York
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New York, New York, Stany Zjednoczone, 10065-4870
- Weill Cornell Medical Center
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North Carolina
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Durham, North Carolina, Stany Zjednoczone, 27710-3038
- Duke University Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, Stany Zjednoczone, 19104
- Penn Medicine - University of Pennsylvania Health System
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Bangkok, Tajlandia, 10400
- Phramongkutklao Hospital
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Bangkok, Tajlandia, 10400
- Ramathibodi Hospital
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Bangkok, Tajlandia, 10700
- Faculty of Medicine Siriraj Hospital
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Chiang Mai, Tajlandia, 50200
- Maharaj Nakorn Chiang Mai Hospital
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Khon Kaen, Tajlandia, 40002
- Srinagarind Hospital, Khon Kaen University
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Mueang Phitsanulok, Tajlandia, 65000
- Naresuan University Hospital
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Pathum Wan, Tajlandia, 10330
- King Chulalongkorn Memorial Hospital
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Taichung, Tajwan, 40447
- China Medical University, Taiwan
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Adana, Turcja (Türkiye), 01130
- Acibadem Adana Hospital
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Antalya, Turcja (Türkiye), 07059
- Akdeniz University Faculty of Medicine
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Balcalı, Turcja (Türkiye), 01330
- Çukurova University
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Bornova, Turcja (Türkiye), 35040
- Ege University Faculty of Medicine
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Fatih, Turcja (Türkiye), 34093
- Istanbul University Faculty of Medicine
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Mersin, Turcja (Türkiye), 06230
- Hacettepe University
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Brindisi, Włochy, 72100
- Ospedale "A. Perrino" - Brindisi
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Cagliari, Włochy, 09121
- Ospedale Pediatrico Microcitemico
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Ferrara, Włochy, 44124
- Ospedale Sant'Anna
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Genova, Włochy, 16128
- Ente Ospedaliero Ospedali Galliera
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Milan, Włochy, 20122
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
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Modena, Włochy, 41124
- A.O.U Di Modena
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Naples, Włochy, 80138
- AOU L. Vanvitelli Universita degli Studi della Campania Luigi Vanvitelli
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Naples, Włochy, 80131
- A.O.R.N. "A. Cardarelli"
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Orbassano, Włochy, 10043
- A.O.U. San Luigi Gonzaga
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Abu Dhabi, Zjednoczone Emiraty Arabskie
- Burjeel Medical City
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Dubai, Zjednoczone Emiraty Arabskie, 4545
- Thalassemia Centre Dubai
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London, Zjednoczone Królestwo, NW1 2PG
- University College London
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London, Zjednoczone Królestwo, W12 OHS
- Imperial College Healthcare NHS Trust - Hammersmith Hospital
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CAM
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Cambridge, CAM, Zjednoczone Królestwo, CB2 0QQ
- Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
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LAN
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Manchester, LAN, Zjednoczone Królestwo, M13 9WL
- Manchester Royal Infirmary, Manchester University NHS Foundation Trust
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Kryteria uczestnictwa
Kryteria kwalifikacji
Kryteria kwalifikacji
Wiek uprawniający do nauki
Akceptuje zdrowych ochotników
Płeć kwalifikująca się do nauki
Opis
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:
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Pregnant, breastfeeding, or parturient
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Documented history of homozygous or heterozygous sickle hemoglobin (HbS) or hemoglobin C (HbC);
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Prior exposure to gene therapy or prior bone marrow or stem cell transplantation;
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Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization;
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Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization;
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History of malignancy, (active or treated) ≤5 years before providing informed consent;
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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;
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Hepatobiliary disorders;
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Estimated glomerular filtration rate <45 milliliters per minute (mL/min)/1.73 m^2 by Chronic Kidney Disease Epidemiology Collaboration creatinine equation;
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Nonfasting triglycerides >440 milligrams per deciliter (mg/dL) (5 millimoles per liter [mmol/L]);
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Active infection requiring systemic antimicrobial therapy at the time of providing informed consent;
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Positive test for hepatitis C virus antibody (HCVAb) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg);
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Positive test for human immunodeficiency virus (HIV)-1 antibody (Ab) or HIV-2 Ab;
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History of major surgery (including splenectomy) ≤16 weeks before providing informed consent and/or a major surgical procedure planned during the study;
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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;
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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;
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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;
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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]);
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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)
Plan studiów
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Randomizowane
- Model interwencyjny: Przydział równoległy
- Maskowanie: Poczwórny
Liczba ramion
Broń i interwencje
Grupa uczestników / ArmGrupa uczestników / Arm |
Interwencja / LeczenieInterwencja / Leczenie |
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Eksperymentalny: 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. |
Tablets
Inne nazwy:
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Komparator placebo: 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. |
Tablets
Inne nazwy:
Tablets |
Co mierzy badanie?
Podstawowe miary wyniku
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
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Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
Ramy czasowe: Double-Blind Period: Baseline up to Week 12 through Week 24
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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
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Miary wyników drugorzędnych
Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
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Okres podwójnie ślepej próby: Stężenie Mitapivatu w osoczu
Ramy czasowe: Okres podwójnie ślepa: dawka przed 12. tygodniem; Dawki przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepa: dawka przed 12. tygodniem; Dawki przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepej próby: czas ostatniego wymiernego stężenia (Tlast) Mitapivatu
Ramy czasowe: Okres podwójnie ślepej próby: przed podaniem dawki, 0,5, 1, 3, 5, 7 godzin po podaniu dawki w 20. tygodniu
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Okres podwójnie ślepej próby: przed podaniem dawki, 0,5, 1, 3, 5, 7 godzin po podaniu dawki w 20. tygodniu
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Okres podwójnie ślepej próby: Maksymalne zaobserwowane stężenie Mitapivatu w osoczu (Cmax).
Ramy czasowe: Okres podwójnie ślepa: dawka przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepa: dawka przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepa: czas do osiągnięcia maksymalnego zaobserwowanego stężenia w osoczu (TMAX) mitapivat
Ramy czasowe: Okres podwójnie ślepej próby: przed podaniem dawki, 0,5, 1, 3, 5, 7 godzin po podaniu dawki w 20. tygodniu
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Okres podwójnie ślepej próby: przed podaniem dawki, 0,5, 1, 3, 5, 7 godzin po podaniu dawki w 20. tygodniu
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Okres podwójnie ślepa: ostatnie kwantyfikowalne stężenie w osoczu (Clast) mitapivat
Ramy czasowe: Okres podwójnie ślepa: dawka przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepa: dawka przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepej próby: Stężenie adenozynotrifosforanu (ATP) we krwi
Ramy czasowe: Okres podwójnie ślepej próby: Dawka wstępna w dniu 1; dawka wstępna w 12. tygodniu; przed dawką, 0,5, 1, 3, 5, 7 godzin po podaniu w 20. tygodniu
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Okres podwójnie ślepej próby: Dawka wstępna w dniu 1; dawka wstępna w 12. tygodniu; przed dawką, 0,5, 1, 3, 5, 7 godzin po podaniu w 20. tygodniu
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Okres podwójnie ślepej próby: stężenie we krwi 2,3-difosfoglicerynianu (2,3-DPG)
Ramy czasowe: Okres podwójnie ślepej ślepej: dawka przed 1 dzień; Dawka przed 12. tygodniem; Dawki przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Okres podwójnie ślepej ślepej: dawka przed 1 dzień; Dawka przed 12. tygodniem; Dawki przed dawką, 0,5, 1, 3, 5, 7 godzin po dawce w tygodniu 20
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Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 12 through Week 24
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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
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Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 12 through Week 24
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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
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Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline
Ramy czasowe: Double-Blind Period: Baseline up to Week 12 through Week 24
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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
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Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Change From Baseline in Haptoglobin at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Change From Baseline in Reticulocytes at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Change From Baseline in Erythropoietin at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Percentage of Participants Who Achieved Patient Global Impression of Severity (PGIS)- Fatigue Response at Weeks 12, 16, 20, and 24
Ramy czasowe: Double-Blind Period: At Weeks 12, 16, 20, and 24
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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
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Double-Blind Period: Percentage of Participants Who Achieved the Patient Global Impression of Change (PGIC)- Fatigue Response at Weeks 12, 16, 20, and 24
Ramy czasowe: Double-Blind Period: At Weeks 12, 16, 20, and 24
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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
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Double-Blind Period: Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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Double-Blind Period: Change From Baseline in Serum Ferritin at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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Iron metabolism was assessed based on serum ferritin levels. |
Double-Blind Period: Baseline, Week 24
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Double-Blind Period: Change From Baseline in Transferrin Saturation (TSAT) at Week 24
Ramy czasowe: Double-Blind Period: Baseline, Week 24
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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
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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
Ramy czasowe: Double-Blind Period: From the time of signing informed consent to Week 24
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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
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Double-Blind Period: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mitapivat
Ramy czasowe: Double-Blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 20
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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
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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
Ramy czasowe: Open-Label Extension Period: From week 24 up to end of study (approximately 5 years)
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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)
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Współpracownicy i badacze
Sponsor
Sponsor
Śledczy
Śledczy
- Krzesło do nauki: Medical Affairs, Agios Pharmaceuticals, Inc.
Publikacje i pomocne linki
Daty zapisu na studia
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
Rozpoczęcie studiów
Zakończenie podstawowe (Rzeczywisty)
Zakończenie podstawowe
Ukończenie studiów (Szacowany)
Ukończenie studiów
Daty rejestracji na studia
Pierwszy przesłany
Pierwszy przesłany
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy wysłany (Rzeczywisty)
Pierwszy wysłany
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
Ostatnia wysłana aktualizacja
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia weryfikacja
Ostatnia weryfikacja
Więcej informacji
Terminy związane z tym badaniem
Dodatkowe istotne warunki MeSH
Inne numery identyfikacyjne badania
Inne numery identyfikacyjne badania
- AG348-C-017
- 2021-000211-23 (Numer EudraCT)
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Bada produkt urządzenia regulowany przez amerykańską FDA
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