A Real-world Study of Asciminib Effectiveness in Philadelphia Positive Acute Lymphoblastic Leukemia Patients (ASCERTAIN)
Asciminib Effectiveness in Real World Setting of Philadelphia Positive Acute Lymphoblastic Leukemia (Ph+ALL); a Retrospective Review Study of Patients From the Asciminib Managed Access Program (ASCERTAIN)
Descripción general del estudio
Estado
Condiciones
Tipo de estudio
Inscripción (Actual)
Contactos y Ubicaciones
Ubicaciones de estudio
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New South Wales
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Sydney, New South Wales, Australia, 2145
- Novartis Investigative Site
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Montreal, Canadá, H1T 2M4
- Novartis Investigative Site
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Madrid, España, 28006
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02115
- Novartis Investigative Site
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Le Chesnay, Francia, 78150
- Novartis Investigative Site
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Ramat Gan, Israel, 52621
- Novartis Investigative Site
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Ascoli Piceno, Italia, 63100
- Novartis Investigative Site
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Bari, Italia, 70124
- Novartis Investigative Site
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Catania, Italia, 95123
- Novartis Investigative Site
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Cuneo, Italia, 12100
- Novartis Investigative Site
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Pescara, Italia, 65124
- Novartis Investigative Site
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Torino, Italia, 10126
- Novartis Investigative Site
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Islamabad, Pakistán, 44000
- Novartis Investigative Site
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Rawalpindi, Pakistán, 46000
- Novartis Investigative Site
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Rotterdam, Países Bajos, 3015
- Novartis Investigative Site
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Bialystok, Polonia, 15-276
- Novartis Investigative Site
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Hong Kong, Porcelana
- Novartis Investigative Site
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London, Reino Unido, SE5 9RS
- Novartis Investigative Site
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Método de muestreo
Población de estudio
Descripción
Inclusion criteria
- Adult patients enrolled in the asciminib MAP.
- Diagnosis of Ph+ ALL.
- Patients received at least one dose of asciminib through the asciminib MAP.
Appropriate approval obtained for the use of patient data including:
- Signed informed consent form (ICF), or
- ICF waiver granted by an Institutional review board/Independent Ethics Committee (IRB/IEC).
Exclusion criteria
• Age < 18 years at the time of initiating asciminib treatment.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
Cohortes e Intervenciones
Grupo / Cohorte |
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Asciminib Cohort
Ph+ ALL patients who received at least one dose of asciminib through the asciminib MAP.
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment
Periodo de tiempo: 3 months
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Hematological CR was defined as no circulating lymphoblasts, absolute neutrophil count (ANC) ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
3 months
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Periodo de tiempo: From Day 1 to 120, and at Baseline, Day 28, 60, 90, 180, 270, 360
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MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, polymerase chain reaction (PCR), and/or next generation sequencing (NGS) techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%. NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells). Best response: CR or CRi achievement. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
From Day 1 to 120, and at Baseline, Day 28, 60, 90, 180, 270, 360
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Proportion of Patients Who Showed MRD Positivity and MRD Negativity
Periodo de tiempo: Baseline, Day 28, 60, 90, 180, 270, 360
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MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, PCR, and/or NGS techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%.
NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells).
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Baseline, Day 28, 60, 90, 180, 270, 360
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Duration of Response (DoR)
Periodo de tiempo: Up to 5 years and 4 months
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DoR measured as the time from achievement of CR or CRi, whichever occurs first, to relapse or death due to acute lymphoblastic leukemia (ALL) at the time of data cut-off. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
Up to 5 years and 4 months
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Proportion of Patients in Hematological CR or CRi Within 13 Months From Start of Treatment
Periodo de tiempo: By Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
By Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Proportion of Patients in Complete Remission
Periodo de tiempo: Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Proportion of patients in complete remission (as per peripheral blood and bone marrow, when both available). Complete Remission was defined as:
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Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Proportion of Patients in Complete Remission With Incomplete Recovery
Periodo de tiempo: Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Proportion of patients in complete remission with incomplete recovery (as per peripheral blood and bone marrow, when both available). Complete remission with incomplete recovery was defined as meeting all criteria for complete remission except without recovery of platelet count or without recovery of ANC:
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Day 28 ± 7 (Day 1 to Day 35), at Day 90 ± 30, by Day 90 ± 30 (Day 1 to Day 120), at Day 180 ± 30, by Day 180 ± 30 (Day 1 to Day 210), at Day 270 ± 30, by Day 270 ± 30 (Day 1 to Day 300), at Day 360 ± 30, by Day 360 ± 30 (Day 1 to Day 390)
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Proportion of Patients who Proceed to Stem Cell Transplantation (SCT)
Periodo de tiempo: Up to 5 years and 4 months
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Proportion of patients who proceed to SCT by the cutoff date.
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Up to 5 years and 4 months
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Proportion of Patients who Continued Treatment With Asciminib After Last SCT
Periodo de tiempo: Up to 5 years and 4 months
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Proportion of patients who proceed to SCT and continue treatment with asciminib by the cutoff date.
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Up to 5 years and 4 months
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Proportion of Patients With BCR::ABL1 T315I Mutation at Baseline and Correlation With Hematological CR/CRi With Asciminib Monotherapy or as a Combination by the Cutoff Date
Periodo de tiempo: Up to 5 years and 4 months
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Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
Up to 5 years and 4 months
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Overall Survival (OS)
Periodo de tiempo: Up to 5 years and 4 months
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OS was defined as the time from start of treatment with asciminib to death due to any cause.
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Up to 5 years and 4 months
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Relapse-free Survival (RFS)
Periodo de tiempo: Up to 5 years and 4 months
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RFS was defined as the time from achievement of hematological CR or CRi, whichever occurs first, to relapse or death due to any cause. Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
Up to 5 years and 4 months
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Proportion of Patients Intolerant to Prior Therapy and not in Hematological CR or CRi at Baseline who Achieved CR or CRi as the Best Response
Periodo de tiempo: During the first 3 months of treatment and at any time point until Day 360 ± 30
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Hematological CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. Hematological CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. |
During the first 3 months of treatment and at any time point until Day 360 ± 30
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Among Patients Intolerant to Prior Therapy With MRD Negativity at Baseline, Duration of MRD
Periodo de tiempo: Up to 5 years and 4 months
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MRD, defined as the percentage of leukemia cells remaining after treatment with asciminib with the use of flow cytometry, PCR, and/or NGS techniques (flow or molecular based MRD is defined as non-detectable if less than 0.01%.
NGS MRD is defined as non-detected if less than 1 in 1 x 10^6 cells)
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Up to 5 years and 4 months
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Proportion of Patients by Reasons for Starting Asciminib
Periodo de tiempo: Up to 5 years and 4 months
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Up to 5 years and 4 months
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Proportion of Patients Who Achieved CR or CRi by Patient Characteristics
Periodo de tiempo: Up to 5 years and 4 months
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Patient characteristics included age group, gender, race and ethnicity, medical history, prior treatments, relapse/remission status, and treatment phase.
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Up to 5 years and 4 months
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Proportion of Patients by DoR and Patient Characteristics
Periodo de tiempo: Up to 5 years and 4 months
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DoR measured as the time from achievement of CR or CRi, whichever occurs first, to relapse or death due to ALL at the time of data cut-off. CR was defined as no circulating lymphoblasts, ANC ≥1,000/μL, platelets ≥100K/μL. CRi was defined as no circulating lymphoblasts, ANC ˂1,000/μL, platelets ˂100K/μL. Patient characteristics included age group, gender, race and ethnicity, medical history, prior treatments, relapse/remission status, and treatment phase. |
Up to 5 years and 4 months
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Proportion of Patients With Adverse Events
Periodo de tiempo: Up to 5 years and 4 months
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Up to 5 years and 4 months
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
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
Palabras clave
Términos MeSH relevantes adicionales
- Neoplasias
- Enfermedades del sistema inmunológico
- Neoplasias por tipo histológico
- Enfermedades hematológicas
- Enfermedades linfáticas
- Trastornos linfoproliferativos
- Trastornos inmunoproliferativos
- Leucemia Linfoide
- Leucemia
- Enfermedades hemic y linfáticas
- Leucemia-linfoma linfoblástico de células precursoras
Otros números de identificación del estudio
- CABL001A2007
Plan de datos de participantes individuales (IPD)
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