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

8 juin 2026 mis à jour par: 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.

Aperçu de l'étude

Statut

Complété

Les conditions

Type d'étude

Observationnel

Inscription (Réel)

37

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

  • Australie
    • New South Wales
      • Sydney, New South Wales, Australie, 2145
        • Novartis Investigative Site
  • Canada
      • Montreal, Canada, H1T 2M4
        • Novartis Investigative Site
  • Chine
      • Hong Kong, Chine
        • Novartis Investigative Site
  • Espagne
      • Madrid, Espagne, 28006
        • Novartis Investigative Site
  • France
      • Le Chesnay, France, 78150
        • Novartis Investigative Site
  • Israël
      • Ramat Gan, Israël, 52621
        • Novartis Investigative Site
  • Italie
      • Ascoli Piceno, Italie, 63100
        • Novartis Investigative Site
      • Bari, Italie, 70124
        • Novartis Investigative Site
      • Catania, Italie, 95123
        • Novartis Investigative Site
      • Cuneo, Italie, 12100
        • Novartis Investigative Site
      • Pescara, Italie, 65124
        • Novartis Investigative Site
      • Torino, Italie, 10126
        • Novartis Investigative Site
  • Pakistan
      • Islamabad, Pakistan, 44000
        • Novartis Investigative Site
      • Rawalpindi, Pakistan, 46000
        • Novartis Investigative Site
  • Pays-Bas
      • Rotterdam, Pays-Bas, 3015
        • Novartis Investigative Site
  • Pologne
      • Bialystok, Pologne, 15-276
        • Novartis Investigative Site
  • Royaume-Uni
      • London, Royaume-Uni, SE5 9RS
        • Novartis Investigative Site
  • États-Unis
    • Massachusetts
      • Boston, Massachusetts, États-Unis, 02115
        • Novartis Investigative Site

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

Méthode d'échantillonnage

Échantillon non probabiliste

Population étudiée

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

La 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.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

Cohortes et interventions

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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Proportion of Patients Achieving Hematological Complete Remission (CR) or Hematological Complete Remission With Incomplete Count Recovery (CRi) in the First 3 Months of Treatment
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Proportion of Patients With Minimal Residual Disease (MRD) Evaluated in Peripheral Blood at Best Response
Délai: 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
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: Up to 5 years and 4 months
Up to 5 years and 4 months

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Novartis Pharmaceuticals

Les enquêteurs

  • Directeur d'études: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

16 décembre 2024

Achèvement primaire (Réel)

17 septembre 2025

Achèvement de l'étude (Réel)

17 septembre 2025

Dates d'inscription aux études

Première soumission

8 juin 2026

Première soumission répondant aux critères de contrôle qualité

8 juin 2026

Première publication (Réel)

12 juin 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

12 juin 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

8 juin 2026

Dernière vérification

1 juin 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • CABL001A2007

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