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

8 de junho de 2026 atualizado por: 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.

Visão geral do estudo

Status

Concluído

Condições

Tipo de estudo

Observacional

Inscrição (Real)

37

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Austrália
    • New South Wales
      • Sydney, New South Wales, Austrália, 2145
        • Novartis Investigative Site
  • Canadá
      • Montreal, Canadá, H1T 2M4
        • Novartis Investigative Site
  • China
      • Hong Kong, China
        • Novartis Investigative Site
  • Espanha
      • Madrid, Espanha, 28006
        • Novartis Investigative Site
  • Estados Unidos
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02115
        • Novartis Investigative Site
  • França
      • Le Chesnay, França, 78150
        • Novartis Investigative Site
  • Holanda
      • Rotterdam, Holanda, 3015
        • Novartis Investigative Site
  • Israel
      • Ramat Gan, Israel, 52621
        • Novartis Investigative Site
  • Itália
      • Ascoli Piceno, Itália, 63100
        • Novartis Investigative Site
      • Bari, Itália, 70124
        • Novartis Investigative Site
      • Catania, Itália, 95123
        • Novartis Investigative Site
      • Cuneo, Itália, 12100
        • Novartis Investigative Site
      • Pescara, Itália, 65124
        • Novartis Investigative Site
      • Torino, Itália, 10126
        • Novartis Investigative Site
  • Paquistão
      • Islamabad, Paquistão, 44000
        • Novartis Investigative Site
      • Rawalpindi, Paquistão, 46000
        • Novartis Investigative Site
  • Polônia
      • Bialystok, Polônia, 15-276
        • Novartis Investigative Site
  • Reino Unido
      • London, Reino Unido, SE5 9RS
        • Novartis Investigative Site

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Método de amostragem

Amostra Não Probabilística

População do estudo

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

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

Coortes e Intervenções

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

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment
Prazo: 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

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: Up to 5 years and 4 months
Up to 5 years and 4 months

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Novartis Pharmaceuticals

Investigadores

  • Diretor de estudo: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

16 de dezembro de 2024

Conclusão Primária (Real)

17 de setembro de 2025

Conclusão do estudo (Real)

17 de setembro de 2025

Datas de inscrição no estudo

Enviado pela primeira vez

8 de junho de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

8 de junho de 2026

Primeira postagem (Real)

12 de junho de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

12 de junho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

8 de junho de 2026

Última verificação

1 de junho de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • CABL001A2007

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

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