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Completed

NCT Number: NCT07644351

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

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.

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Key information

Conditions

Acute Lymphoblastic Leukemia Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Lymphatic Diseases Lymphoproliferative Disorders Neoplasms Neoplasms by Histologic Type Precursor Cell Lymphoblastic Leukemia-Lymphoma

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment

    Time frame: 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.

Secondary outcomes

  1. Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response

    Time frame: 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.

  2. Proportion of Patients Who Showed MRD Positivity and MRD Negativity

    Time frame: 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).

  3. Duration of Response (DoR)

    Time frame: 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.

  4. Proportion of Patients in Hematological CR or CRi Within 13 Months From Start of Treatment

    Time frame: 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.

  5. Proportion of Patients in Complete Remission

    Time frame: 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.

  6. Proportion of Patients in Complete Remission With Incomplete Recovery

    Time frame: 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

  7. Proportion of Patients who Proceed to Stem Cell Transplantation (SCT)

    Time frame: Up to 5 years and 4 months

    Proportion of patients who proceed to SCT by the cutoff date.

  8. Proportion of Patients who Continued Treatment With Asciminib After Last SCT

    Time frame: Up to 5 years and 4 months

    Proportion of patients who proceed to SCT and continue treatment with asciminib by the cutoff date.

  9. 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

    Time frame: 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.

  10. Overall Survival (OS)

    Time frame: 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.

  11. Relapse-free Survival (RFS)

    Time frame: 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.

  12. 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

    Time frame: 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.

  13. Among Patients Intolerant to Prior Therapy With MRD Negativity at Baseline, Duration of MRD

    Time frame: 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)

  14. Proportion of Patients by Reasons for Starting Asciminib

    Time frame: Up to 5 years and 4 months

  15. Proportion of Patients Who Achieved CR or CRi by Patient Characteristics

    Time frame: 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.

  16. Proportion of Patients by DoR and Patient Characteristics

    Time frame: 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.

  17. Proportion of Patients With Adverse Events

    Time frame: Up to 5 years and 4 months

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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)

Acronym: ASCERTAIN

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jun 12, 2026
Registry last updated
Jun 12, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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