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)

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.

Study Overview

• Status: Completed
• Conditions: Acute Lymphoblastic Leukemia

• Study Type: Observational
• Enrollment (Actual): 37

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2145
      • Novartis Investigative Site
• Canada
  • Montreal, Canada, H1T 2M4
    • Novartis Investigative Site
• China
  • Hong Kong, China
    • Novartis Investigative Site
• France
  • Le Chesnay, France, 78150
    • Novartis Investigative Site
• Israel
  • Ramat Gan, Israel, 52621
    • Novartis Investigative Site
• Italy
  • Ascoli Piceno, Italy, 63100
    • Novartis Investigative Site
  • Bari, Italy, 70124
    • Novartis Investigative Site
  • Catania, Italy, 95123
    • Novartis Investigative Site
  • Cuneo, Italy, 12100
    • Novartis Investigative Site
  • Pescara, Italy, 65124
    • Novartis Investigative Site
  • Torino, Italy, 10126
    • Novartis Investigative Site
• Netherlands
  • Rotterdam, Netherlands, 3015
    • Novartis Investigative Site
• Pakistan
  • Islamabad, Pakistan, 44000
    • Novartis Investigative Site
  • Rawalpindi, Pakistan, 46000
    • Novartis Investigative Site
• Poland
  • Bialystok, Poland, 15-276
    • Novartis Investigative Site
• Spain
  • Madrid, Spain, 28006
    • Novartis Investigative Site
• United Kingdom
  • London, United Kingdom, SE5 9RS
    • Novartis Investigative Site
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment	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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response	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	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)	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	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	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	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)	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	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	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)	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)	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	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	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		Up to 5 years and 4 months
Proportion of Patients Who Achieved CR or CRi by Patient Characteristics	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	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		Up to 5 years and 4 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): December 16, 2024
• Primary Completion (Actual): September 17, 2025
• Study Completion (Actual): September 17, 2025

Study Registration Dates

• First Submitted: June 8, 2026
• First Submitted That Met QC Criteria: June 8, 2026
• First Posted (Actual): June 12, 2026

Study Record Updates

• Last Update Posted (Actual): June 12, 2026
• Last Update Submitted That Met QC Criteria: June 8, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Asciminib; Real world study; Ph+ ALL
• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia, Lymphoid; Leukemia; Hemic and Lymphatic Diseases; Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Other Study ID Numbers: CABL001A2007

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO