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

8 juni 2026 uppdaterad av: 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.

Studieöversikt

Status

Avslutad

Betingelser

Studietyp

Observationell

Inskrivning (Faktisk)

37

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • Australien
    • New South Wales
      • Sydney, New South Wales, Australien, 2145
        • Novartis Investigative Site
  • Frankrike
      • Le Chesnay, Frankrike, 78150
        • Novartis Investigative Site
  • Förenta staterna
    • Massachusetts
      • Boston, Massachusetts, Förenta staterna, 02115
        • Novartis Investigative Site
  • Israel
      • Ramat Gan, Israel, 52621
        • Novartis Investigative Site
  • Italien
      • Ascoli Piceno, Italien, 63100
        • Novartis Investigative Site
      • Bari, Italien, 70124
        • Novartis Investigative Site
      • Catania, Italien, 95123
        • Novartis Investigative Site
      • Cuneo, Italien, 12100
        • Novartis Investigative Site
      • Pescara, Italien, 65124
        • Novartis Investigative Site
      • Torino, Italien, 10126
        • Novartis Investigative Site
  • Kanada
      • Montreal, Kanada, H1T 2M4
        • Novartis Investigative Site
  • Kina
      • Hong Kong, Kina
        • Novartis Investigative Site
  • Nederländerna
      • Rotterdam, Nederländerna, 3015
        • Novartis Investigative Site
  • Pakistan
      • Islamabad, Pakistan, 44000
        • Novartis Investigative Site
      • Rawalpindi, Pakistan, 46000
        • Novartis Investigative Site
  • Polen
      • Bialystok, Polen, 15-276
        • Novartis Investigative Site
  • Spanien
      • Madrid, Spanien, 28006
        • Novartis Investigative Site
  • Storbritannien
      • London, Storbritannien, SE5 9RS
        • Novartis Investigative Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Testmetod

Icke-sannolikhetsprov

Studera befolkning

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

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Kohorter och interventioner

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

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: Up to 5 years and 4 months
Up to 5 years and 4 months

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Novartis Pharmaceuticals

Utredare

  • Studierektor: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

16 december 2024

Primärt slutförande (Faktisk)

17 september 2025

Avslutad studie (Faktisk)

17 september 2025

Studieregistreringsdatum

Först inskickad

8 juni 2026

Först inskickad som uppfyllde QC-kriterierna

8 juni 2026

Första postat (Faktisk)

12 juni 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

12 juni 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

8 juni 2026

Senast verifierad

1 juni 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • CABL001A2007

Plan för individuella deltagardata (IPD)

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