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)
Přehled studie
Postavení
Podmínky
Typ studie
Zápis (Aktuální)
Kontakty a umístění
Studijní místa
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New South Wales
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Sydney, New South Wales, Austrálie, 2145
- Novartis Investigative Site
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Le Chesnay, Francie, 78150
- Novartis Investigative Site
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Rotterdam, Holandsko, 3015
- Novartis Investigative Site
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Ascoli Piceno, Itálie, 63100
- Novartis Investigative Site
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Bari, Itálie, 70124
- Novartis Investigative Site
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Catania, Itálie, 95123
- Novartis Investigative Site
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Cuneo, Itálie, 12100
- Novartis Investigative Site
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Pescara, Itálie, 65124
- Novartis Investigative Site
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Torino, Itálie, 10126
- Novartis Investigative Site
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Ramat Gan, Izrael, 52621
- Novartis Investigative Site
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Montreal, Kanada, H1T 2M4
- Novartis Investigative Site
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Bialystok, Polsko, 15-276
- Novartis Investigative Site
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Islamabad, Pákistán, 44000
- Novartis Investigative Site
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Rawalpindi, Pákistán, 46000
- Novartis Investigative Site
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London, Spojené království, SE5 9RS
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, Spojené státy, 02115
- Novartis Investigative Site
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Hong Kong, Čína
- Novartis Investigative Site
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Madrid, Španělsko, 28006
- Novartis Investigative Site
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Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Metoda odběru vzorků
Studijní populace
Popis
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.
Studijní plán
Jak je studie koncipována?
Detaily designu
Kohorty a intervence
Skupina / kohorta |
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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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Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
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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
Časové okno: 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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Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
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Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Časové okno: 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
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: Up to 5 years and 4 months
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Up to 5 years and 4 months
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Spolupracovníci a vyšetřovatelé
Sponzor
Vyšetřovatelé
- Ředitel studie: Novartis Pharmaceuticals, Novartis Pharmaceuticals
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Další relevantní podmínky MeSH
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