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Asciminib and Inotuzumab Ozogamicin for Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (R/R Ph+ B-ALL)

9 de noviembre de 2026 actualizado por: Sunil G. Iyer

A Phase I Open-label, Multicenter Trial of Asciminib (ABL001) Added to Inotuzumab Ozogamicin (Besponsa®) in Patients With Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia

This is a multi-center phase 1 open-label study to determine the safety of ABL001 and INO in patients with relapsed or refractory Philadelphia chromosome-positive b-cell acute lymphoblastic leukemia.

Descripción general del estudio

Descripción detallada

This study will test a combination of two drugs for people with Philadelphia chromosome-positive B-cell acute lymphoblastic leukemia (Ph-positive B-ALL), a fast-growing blood cancer. Asciminib is a targeted oral drug that is FDA-approved for chronic myeloid leukemia, but is not FDA-approved for Ph-positive B-ALL. Inotuzumab ozogamicin (also called Besponsa) is an FDA-approved treatment that uses an antibody (a protein) to deliver a small amount of chemotherapy directly to leukemia cells. It is commonly used when leukemia has come back after earlier treatment (relapsed) or has not improved with treatment (refractory). This study aims to find the safest dose of asciminib when it is given together with inotuzumab ozogamicin. The goal is to see whether this combination can provide a more effective and better tolerated treatment option for people whose leukemia has returned or has not responded to previous therapies.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

45

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Ubicaciones de estudio

    • Florida
      • Miami, Florida, Estados Unidos, 33136
        • Aún no reclutando
        • University of Miami Sylvester Comprehensive Cancer Center
        • Contacto:
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60805
        • Aún no reclutando
        • University of Chicago Comprehensive Cancer Center
        • Contacto:
    • New York
      • New York, New York, Estados Unidos, 10032
    • Washington
      • Seattle, Washington, Estados Unidos, 98109
        • Aún no reclutando
        • University of Washington Fred Hutchinson Comprehensive Cancer Center
        • Contacto:

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años y mayores (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Inclusion Criteria:

  1. Documented and confirmed CD22+ R/R Ph+ B-ALL according to the World Health Organization (WHO) classification

    • Relapsed ALL is defined as the detection of ≥ 5% blasts in the bone marrow with or without non-central nervous system (CNS) extramedullary disease
    • Refractory ALL is defined as the failure to obtain a CRc after at least two courses of intensive induction or immunotherapy such as blinatumomab, or to CAR-T cells. Persistent MRD without ≥ 5% blasts in the bone marrow will not be sufficient for inclusion
  2. Disease must be positive for Ph (p190 or p210 transcript) by cytogenetics or PCR

  3. Disease must be positive for CD22, defined as any percentage of leukemic blasts expressing CD22

  4. Disease must be positive for t(9;22) by Cytogenetics and FISH. Subjects with complex translocations resulting in BCR::ABL1 fusion protein are eligible.

  5. BCR-ABL mutational panel performed within 90 days prior to start of treatment, including the following 4 mutations: T315I and M244V, A337V, or F359C/I/V mutations. Specific panel may be determined per institution, however must include all aforementioned mutations. Note: T315I mutated patients are only eligible once Dose Level 3 is open to enrollment

  6. Subject must have recovered (no worse than Grade 1 severity) from effects of all previous treatments.

  7. Able to provide informed consent

  8. Age ≥18 years of age

  9. Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 2 or Karnofsky ≥60%

  10. Adequate organ function defined by the following parameters:

    • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5x the upper limit of normal (ULN)
    • Total bilirubin ≤1.5x the ULN unless due to known Gilbert's disease or hemolysis due to blood transfusion
    • For patients with Gilbert's disease: total bilirubin ≤ 3x the ULN
    • Calculated glomerular filtration rate (GFR) of ≥ 15 mL/min/1.73 m2
  11. Female patients of childbearing potential must not be nursing or planning to become pregnant

  12. Female patients of childbearing potential require a negative urine or serum pregnancy test within 30 days of study therapy.

  13. Female patients of childbearing potential must be willing to use at least 1 method of highly effective contraception during the study until 8 months after last dose of inotuzumab ozogamicin, or 1 week after the last dose of asciminib, whichever is later.

  14. Male patients who are sexually active must be willing to use condoms and refrain from sperm donation during the study until 5 months after last dose of inotuzumab ozogamicin

  15. Willing to provide pretreatment bone marrow aspirate and biopsies samples as well as subsequent bone marrow aspirate and biopsies samples during the study.

Exclusion Criteria:

  1. Presence of severe and/or uncontrollable concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection)
  2. All prior Philadelphia chromosome-directed TKI therapy must be discontinued at least five days prior to study entry; corticosteroids and hydroxyurea are permitted during this period
  3. Treatment with chemotherapy, radiotherapy, or biologic therapy within 3 weeks prior to treatment Note: Hydroxyurea or corticosteroids is permitted; Previous treatment with ≥1 BCR-ABL-directed TKI
  4. Prior allogeneic stem cell transplant < 90 days prior to anticipated treatment start
  5. Patients with active GVHD (graft versus host disease) and/or receiving immunosuppressive drugs for GVHD
  6. Patients receiving calcineurin inhibitors < 4 weeks prior to anticipated treatment start
  7. Ph-directed TKI within 5 days of treatment start
  8. Prior exposure to INO
  9. Prior exposure to asciminib
  10. The presence of mixed phenotype acute leukemia or CML in lymphoid blast phase
  11. Presence of BCR-ABL1 M244V, A337V, or F359C/I/V mutations other n-lobe known to confer clinical resistance to asciminib as detected by ABL1 kinase mutational panel
  12. Patients with T315I mutation as detected by ABL1 kinase mutational panel are excluded from asciminib dose levels lower than 200mg BID
  13. Clinical suspicion for active CNS involvement by ALL; previously treated CNS involvement is allowed
  14. Second malignancy requiring treatment within 6 months of study enrollment, with the exception of non-melanoma skin cancers or cancers requiring hormonal therapy only
  15. Active, uncontrolled infection
  16. Human immunodeficiency virus (HIV) not controlled by standard therapy
  17. Active hepatitis B or C infection. Participants whose infections are controlled with antiviral therapy are allowed.
  18. Patients with end stage renal disease requiring dialysis

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Single-Arm

All participants enrolled to the treatment arm of the study will receive Asciminib in combination with Inotuzumab Ozogamicin.

INO is to be dosed in a fractionated schedule as follows:

  • Prior to achieving compositive complete remission (CRc): 0.8 mg/m2 day 1, 0.5 mg/m2 days 8 and 15
  • Once CRc achieved (even if MR3/MR4 not achieved): 0.5 mg/m2 days 1, 8, 15

Asciminib will be administered at a dose of 40, 60, 80, 160, or 200 mg twice daily.

Asciminib is a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket), and is an approved TKI in newly-diagnosed and previously-treated chronic myeloid leukemia (CML). It is a non-chemotherapeutic, oral agent which specifically and potently targets the mutant Philadelphia chromosome driven by t(9;22). Due to its novel mechanism of action as a STAMP, asciminib is active in the setting of mutations in the BCR-ABL ATP-binding pocket including the T315I gatekeeper mutations, which confers resistance to first- and second-generation tyrosine kinase inhibitors (TKIs). Asciminib has not yet been studied in Philadelphia-chromosome positive B-ALL.

Otros nombres:
  • ABL001

Inotuzumab ozogamicin (INO) is an antibody-drug conjugate which targets CD22, an antigen present in >90% of B-ALL cases. INO is FDA approved and a standard-of-care treatment in relapsed/refractory B-ALL. The drug is approved and active in Ph-positive and -negative disease.

Otros nombres:
  • INO
  • BESPONSA

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of adverse events
Periodo de tiempo: 36 Months

The safety profile of asciminib added to inotuzumab ozogamicin (INO) will be evaluated by the rates of adverse events categorized by the NCI Common Terminology Criteria for Adverse Events [CTCAE] v5.0.

36 Months
Maximum Tolerated Dose (MTD) of asciminib
Periodo de tiempo: 36 Months

The MTD of asciminib will be determined using a dose escalation strategy. The MTD is defined as the highest dose that be administered without causing dose-limiting toxicities in 75% of participants.

36 Months

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Composite Complete Response (CRc) rate
Periodo de tiempo: 36 Months

Composite complete response (CRc) is defined as the rate of complete response (CR) + complete response with incomplete hematologic recovery (CRi), with complete metabolic response by PET/CT by the end of consolidation, according to the European LeukemiaNet (ELN) 2024 ALL criteria.

36 Months
Duration of remission (DOR)
Periodo de tiempo: 36 Months

DOR is defined as the time from CRc until relapse or death from any cause, whichever is first

36 Months
Overall survival (OS)
Periodo de tiempo: 36 Months

OS is defined as the time from study entry until death from any cause

36 Months
Event-free survival (EFS)
Periodo de tiempo: 36 Months

EFS is defined as the time from randomization to an event which may include disease progression, discontinuation of the treatment for any reason, or death

36 Months
Rate of molecular response (MR)
Periodo de tiempo: 36 Months

The rate of molecular response (MR) is assessed by clearance of measurable disease <0.1% (MR3) and <0.01% (MR4) as measured by BCR::ABL1 PCR in the bone marrow with complete metabolic response by PET/CT by the end of consolidation.

36 Months

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Colaboradores

Investigadores

  • Investigador principal: Sunil Iyer, MD, Columbia University

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

31 de marzo de 2027

Finalización primaria (Estimado)

31 de diciembre de 2030

Finalización del estudio (Estimado)

31 de diciembre de 2030

Fechas de registro del estudio

Enviado por primera vez

27 de octubre de 2026

Primero enviado que cumplió con los criterios de control de calidad

9 de noviembre de 2026

Publicado por primera vez (Actual)

13 de noviembre de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

13 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

9 de noviembre de 2026

Última verificación

1 de noviembre de 2026

Más información

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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