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A Real-world Study of Asciminib Effectiveness in Philadelphia Positive Acute Lymphoblastic Leukemia Patients (ASCERTAIN)

8 июня 2026 г. обновлено: Novartis Pharmaceuticals

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)

The aim of this study was to collect existing information from the medical charts of patients enrolled in the ongoing asciminib Managed Access Program (MAP) to better understand the effectiveness and safety of asciminib when used to treat adult patients with Ph+ ALL who are refractory, resistant or intolerant to available treatments.

Обзор исследования

Статус

Завершенный

Условия

Тип исследования

Наблюдательный

Регистрация (Действительный)

37

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Места учебы

  • Австралия
    • New South Wales
      • Sydney, New South Wales, Австралия, 2145
        • Novartis Investigative Site
  • Израиль
      • Ramat Gan, Израиль, 52621
        • Novartis Investigative Site
  • Испания
      • Madrid, Испания, 28006
        • Novartis Investigative Site
  • Италия
      • Ascoli Piceno, Италия, 63100
        • Novartis Investigative Site
      • Bari, Италия, 70124
        • Novartis Investigative Site
      • Catania, Италия, 95123
        • Novartis Investigative Site
      • Cuneo, Италия, 12100
        • Novartis Investigative Site
      • Pescara, Италия, 65124
        • Novartis Investigative Site
      • Torino, Италия, 10126
        • Novartis Investigative Site
  • Канада
      • Montreal, Канада, H1T 2M4
        • Novartis Investigative Site
  • Китай
      • Hong Kong, Китай
        • Novartis Investigative Site
  • Нидерланды
      • Rotterdam, Нидерланды, 3015
        • Novartis Investigative Site
  • Пакистан
      • Islamabad, Пакистан, 44000
        • Novartis Investigative Site
      • Rawalpindi, Пакистан, 46000
        • Novartis Investigative Site
  • Польша
      • Bialystok, Польша, 15-276
        • Novartis Investigative Site
  • Соединенное Королевство
      • London, Соединенное Королевство, SE5 9RS
        • Novartis Investigative Site
  • Соединенные Штаты
    • Massachusetts
      • Boston, Massachusetts, Соединенные Штаты, 02115
        • Novartis Investigative Site
  • Франция
      • Le Chesnay, Франция, 78150
        • Novartis Investigative Site

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

  • Взрослый
  • Пожилой взрослый

Принимает здоровых добровольцев

Нет

Метод выборки

Невероятностная выборка

Исследуемая популяция

Patients with Ph+ ALL who were enrolled in the asciminib MAP and received at least one dose of asciminib.

Описание

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.

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

Когорты и вмешательства

Группа / когорта
Asciminib Cohort
Ph+ ALL patients who received at least one dose of asciminib through the asciminib MAP.

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment
Временное ограничение: 3 months

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

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Временное ограничение: From Day 1 to 120, and at Baseline, Day 28, 60, 90, 180, 270, 360

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
Proportion of Patients Who Showed MRD Positivity and MRD Negativity
Временное ограничение: Baseline, Day 28, 60, 90, 180, 270, 360
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).
Baseline, Day 28, 60, 90, 180, 270, 360
Duration of Response (DoR)
Временное ограничение: Up to 5 years and 4 months

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
Proportion of Patients in Hematological CR or CRi Within 13 Months From Start of Treatment
Временное ограничение: 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)

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)
Proportion of Patients in Complete Remission
Временное ограничение: 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)

Proportion of patients in complete remission (as per peripheral blood and bone marrow, when both available).

Complete Remission was defined as:

  • No circulating lymphoblasts or extramedullary disease
  • No lymphadenopathy, splenomegaly, skin/gum infiltration, testicular mass, central nervous system (CNS) involvement.
  • Trilineage hematopoiesis (TLH) and <5% blasts.
  • ANC ≥1000/μL.
  • Platelets ≥100,000/μL.
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)
Proportion of Patients in Complete Remission With Incomplete Recovery
Временное ограничение: 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)

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:

  • Platelets <100,000/μL and ANC ≥1000/μL or
  • Platelets ≥100,000/μL and ANC <1000/μL
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)
Proportion of Patients who Proceed to Stem Cell Transplantation (SCT)
Временное ограничение: Up to 5 years and 4 months
Proportion of patients who proceed to SCT by the cutoff date.
Up to 5 years and 4 months
Proportion of Patients who Continued Treatment With Asciminib After Last SCT
Временное ограничение: Up to 5 years and 4 months
Proportion of patients who proceed to SCT and continue treatment with asciminib by the cutoff date.
Up to 5 years and 4 months
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
Временное ограничение: Up to 5 years and 4 months

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
Overall Survival (OS)
Временное ограничение: Up to 5 years and 4 months
OS was defined as the time from start of treatment with asciminib to death due to any cause.
Up to 5 years and 4 months
Relapse-free Survival (RFS)
Временное ограничение: Up to 5 years and 4 months

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
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
Временное ограничение: During the first 3 months of treatment and at any time point until Day 360 ± 30

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
Among Patients Intolerant to Prior Therapy With MRD Negativity at Baseline, Duration of MRD
Временное ограничение: Up to 5 years and 4 months
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)
Up to 5 years and 4 months
Proportion of Patients by Reasons for Starting Asciminib
Временное ограничение: Up to 5 years and 4 months
Up to 5 years and 4 months
Proportion of Patients Who Achieved CR or CRi by Patient Characteristics
Временное ограничение: Up to 5 years and 4 months
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
Proportion of Patients by DoR and Patient Characteristics
Временное ограничение: Up to 5 years and 4 months

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
Proportion of Patients With Adverse Events
Временное ограничение: Up to 5 years and 4 months
Up to 5 years and 4 months

Соавторы и исследователи

Здесь вы найдете людей и организации, участвующие в этом исследовании.

Спонсор

Novartis Pharmaceuticals

Следователи

  • Директор по исследованиям: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

16 декабря 2024 г.

Первичное завершение (Действительный)

17 сентября 2025 г.

Завершение исследования (Действительный)

17 сентября 2025 г.

Даты регистрации исследования

Первый отправленный

8 июня 2026 г.

Впервые представлено, что соответствует критериям контроля качества

8 июня 2026 г.

Первый опубликованный (Действительный)

12 июня 2026 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

12 июня 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

8 июня 2026 г.

Последняя проверка

1 июня 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Ключевые слова

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • CABL001A2007

Планирование данных отдельных участников (IPD)

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НЕТ

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

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