Un estudio que evalúa la eficacia y la seguridad de mitapivat en participantes con alfa o beta talasemia no dependientes de transfusiones (α o β-NTDT)
Un estudio de fase 3, doble ciego, aleatorizado, controlado con placebo, multicéntrico que evalúa la eficacia y la seguridad de mitapivat en sujetos con talasemia alfa o beta no dependiente de transfusiones (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).
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
The mitapivat group included 130 participants whereas the placebo group had 64 participants.
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 3
Contactos y Ubicaciones
Ubicaciones de estudio
-
-
-
Al Mubarraz, Arabia Saudita, 36428
- King Abdulaziz Hospital - Al Ahsa
-
Riyadh, Arabia Saudita, 14611
- King Abdullah International Medical Research Center
-
-
-
-
-
Caxias do Sul, Brasil, 95070-560
- Universidade de Caxias do Sul
-
Ribeirão Preto, Brasil, 14051-260
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
-
Rio de Janeiro, Brasil, 20211-030
- HEMORIO Instituto Nacional de Hematologia
-
Santo André, Brasil, 09090-790
- Praxis Pesquisa Medica
-
São Paulo, Brasil, 04006-002
- GSH Banco de Sangue de São Paulo
-
São Paulo, Brasil, 05403-000
- Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
-
-
-
-
-
Kyustendil, Bulgaria, 2500
- MHAT "Dr. Nikola Vasiliev" AD
-
Sofia, Bulgaria, 1756
- SHATHD Sofia
-
-
-
-
-
Toronto, Canadá, M5G 2C4
- Toronto General Hospital, University Health Network
-
-
-
-
-
Copenhagen, Dinamarca, 2100
- Rigshospitalet
-
-
-
-
-
Abu Dhabi, Emiratos Árabes Unidos
- Burjeel Medical City
-
Dubai, Emiratos Árabes Unidos, 4545
- Thalassemia Centre Dubai
-
-
-
-
-
Barcelona, España, 08035
- Hospital Universitario Vall d'Hebron
-
Madrid, España, 28046
- Hospital Universitario La Paz
-
Murcia, España, 30120
- Hospital Universitario Virgen Arrixaca
-
Seville, España, 41013
- Hospital Universitario Virgen del Rocío
-
-
-
-
California
-
La Jolla, California, Estados Unidos, 92093
- San Diego Hospital, UC San Diego Health
-
Palo Alto, California, Estados Unidos, 94304-1601
- Stanford Medicine
-
-
Massachusetts
-
Boston, Massachusetts, Estados Unidos, 02114-2696
- Massachusetts General Hospital
-
-
New York
-
New York, New York, Estados Unidos, 10065-4870
- Weill Cornell Medical Center
-
-
North Carolina
-
Durham, North Carolina, Estados Unidos, 27710-3038
- Duke University Medical Center
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Estados Unidos, 19104
- Penn Medicine - University of Pennsylvania Health System
-
-
-
-
-
Créteil, Francia, 94010
- CHU Hôpital Henri Mondor
-
Lyon, Francia, 69003
- Hopital Edouard Herriot, CHU de Lyon
-
-
-
-
-
Athens, Grecia, 115 27
- Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School
-
Athens, Grecia, 115 26
- Laiko General Hospital
-
Rio, Grecia, 26504
- University General Hospital of Patras
-
Thessaloniki, Grecia, 546 42
- Ippokrateio General Hospital
-
-
-
-
-
Brindisi, Italia, 72100
- Ospedale "A. Perrino" - Brindisi
-
Cagliari, Italia, 09121
- Ospedale Pediatrico Microcitemico
-
Ferrara, Italia, 44124
- Ospedale Sant'Anna
-
Genova, Italia, 16128
- Ente Ospedaliero Ospedali Galliera
-
Milan, Italia, 20122
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
-
Modena, Italia, 41124
- A.O.U Di Modena
-
Naples, Italia, 80138
- AOU L. Vanvitelli Universita degli Studi della Campania Luigi Vanvitelli
-
Naples, Italia, 80131
- A.O.R.N. "A. Cardarelli"
-
Orbassano, Italia, 10043
- A.O.U. San Luigi Gonzaga
-
-
-
-
-
Beirut, Líbano, 9999
- Chronic Care Center
-
-
-
-
-
Alor Star, Malasia, 05460
- Hospital Sultanah Bahiyah
-
Ampang, Malasia, 68000
- Hospital Ampang
-
Johor Bahru, Malasia, 80100
- Hospital Sultanah Aminah Johor Bahru
-
Kota Kinabalu, Malasia, 88586
- Hospital Queen Elizabeth, Kota Kinabalu
-
Kuala Lumpur, Malasia, 50300
- Hospital Tunku Azizah
-
Kuantan, Malasia, 25100
- Hospital Tengku Ampuan Afzan
-
Kuching, Malasia, 93586
- Hospital Umum Sarawak
-
Pulau Pinang, Malasia, 10990
- Hospital Pulau Pinang
-
-
-
-
-
Rotterdam, Países Bajos, 3015 GD
- Erasmus MC
-
Utrecht, Países Bajos, 3584 CX
- Universitair Medisch Centrum Utrecht
-
-
-
-
-
London, Reino Unido, NW1 2PG
- University College London
-
London, Reino Unido, W12 OHS
- Imperial College Healthcare NHS Trust - Hammersmith Hospital
-
-
CAM
-
Cambridge, CAM, Reino Unido, CB2 0QQ
- Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
-
-
LAN
-
Manchester, LAN, Reino Unido, M13 9WL
- Manchester Royal Infirmary, Manchester University NHS Foundation Trust
-
-
-
-
-
Bangkok, Tailandia, 10400
- Phramongkutklao Hospital
-
Bangkok, Tailandia, 10400
- Ramathibodi Hospital
-
Bangkok, Tailandia, 10700
- Faculty of Medicine Siriraj Hospital
-
Chiang Mai, Tailandia, 50200
- Maharaj Nakorn Chiang Mai Hospital
-
Khon Kaen, Tailandia, 40002
- Srinagarind Hospital, Khon Kaen University
-
Mueang Phitsanulok, Tailandia, 65000
- Naresuan University Hospital
-
Pathum Wan, Tailandia, 10330
- King Chulalongkorn Memorial Hospital
-
-
-
-
-
Taichung, Taiwán, 40447
- China Medical University, Taiwan
-
-
-
-
-
Adana, Turquía (Türkiye), 01130
- Acibadem Adana Hospital
-
Antalya, Turquía (Türkiye), 07059
- Akdeniz University Faculty of Medicine
-
Balcalı, Turquía (Türkiye), 01330
- Çukurova University
-
Bornova, Turquía (Türkiye), 35040
- Ege University Faculty of Medicine
-
Fatih, Turquía (Türkiye), 34093
- Istanbul University Faculty of Medicine
-
Mersin, Turquía (Türkiye), 06230
- Hacettepe University
-
-
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
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)
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Cuadruplicar
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Experimental: Mitapivat
Mitapivat 100 milligrams (mg), orally, twice daily (BID) for 24 weeks in double blind (DB) period and for up to 5 years in open label extension (OLE) period. |
Tablets
Otros nombres:
|
|
Comparador de placebos: 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
Otros nombres:
Tablets |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
Periodo de tiempo: 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
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Período doble ciego: concentración plasmática de Mitapivat
Periodo de tiempo: Período doble ciego: pre-dosis en la semana 12; Previa, 0.5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: pre-dosis en la semana 12; Previa, 0.5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: tiempo de la última concentración cuantificable (TLAST) de Mitapivat
Periodo de tiempo: Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: concentración plasmática máxima observada (Cmax) de Mitapivat
Periodo de tiempo: Período doble ciego: pre-dosis, 0.5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: pre-dosis, 0.5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: tiempo para alcanzar la concentración plasmática máxima observada (Tmax) de Mitapivat
Periodo de tiempo: Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: última concentración de plasma cuantificable (CLAST) de Mitapivat
Periodo de tiempo: Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: concentración sanguínea de trifosfato de adenosina (ATP)
Periodo de tiempo: Período doble ciego: dosis previa el día 1; predosis en la semana 12; antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: dosis previa el día 1; predosis en la semana 12; antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Período doble ciego: concentración sanguínea de 2,3-difosfoglicerato (2,3-DPG)
Periodo de tiempo: Período doble ciego: dosis previa el día 1; predosis en la semana 12; antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
Período doble ciego: dosis previa el día 1; predosis en la semana 12; antes de la dosis, 0,5, 1, 3, 5, 7 horas después de la dosis en la semana 20
|
|
|
Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Silla de estudio: Medical Affairs, Agios Pharmaceuticals, Inc.
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- AG348-C-017
- 2021-000211-23 (Número EudraCT)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .