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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)

maanantai 9. marraskuuta 2026 päivittänyt: 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.

Tutkimuksen yleiskatsaus

Yksityiskohtainen kuvaus

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.

Opintotyyppi

Interventio

Ilmoittautuminen (Arvioitu)

45

Vaihe

  • Vaihe 1

Yhteystiedot ja paikat

Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.

Opiskeluyhteys

Opiskelupaikat

    • Florida
      • Miami, Florida, Yhdysvallat, 33136
        • Ei vielä rekrytointia
        • University of Miami Sylvester Comprehensive Cancer Center
        • Ottaa yhteyttä:
    • Illinois
      • Chicago, Illinois, Yhdysvallat, 60805
        • Ei vielä rekrytointia
        • University of Chicago Comprehensive Cancer Center
        • Ottaa yhteyttä:
    • New York
      • New York, New York, Yhdysvallat, 10032
    • Washington
      • Seattle, Washington, Yhdysvallat, 98109
        • Ei vielä rekrytointia
        • University of Washington Fred Hutchinson Comprehensive Cancer Center
        • Ottaa yhteyttä:

Osallistumiskriteerit

Tutkijat etsivät ihmisiä, jotka sopivat tiettyyn kuvaukseen, jota kutsutaan kelpoisuuskriteereiksi. Joitakin esimerkkejä näistä kriteereistä ovat henkilön yleinen terveydentila tai aiemmat hoidot.

Kelpoisuusvaatimukset

Opintokelpoiset iät

18 vuotta ja vanhemmat (Aikuinen, Vanhempi Aikuinen)

Hyväksyy terveitä vapaaehtoisia

Ei

Sukupuolet, jotka voivat opiskella

Kaikki

Kuvaus

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

Opintosuunnitelma

Tässä osiossa on tietoja tutkimussuunnitelmasta, mukaan lukien kuinka tutkimus on suunniteltu ja mitä tutkimuksella mitataan.

Miten tutkimus on suunniteltu?

Suunnittelun yksityiskohdat

  • Ensisijainen käyttötarkoitus: Hoito
  • Jako: Ei käytössä
  • Inventiomalli: Peräkkäinen tehtävä
  • Naamiointi: Ei mitään (avoin tarra)

Aseet ja interventiot

Osallistujaryhmä / Arm
Interventio / Hoito
Kokeellinen: 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.

Muut nimet:
  • 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.

Muut nimet:
  • INO
  • BESPONSA

Mitä tutkimuksessa mitataan?

Ensisijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Number of adverse events
Aikaikkuna: 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
Aikaikkuna: 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

Toissijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Composite Complete Response (CRc) rate
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 36 Months

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

36 Months
Event-free survival (EFS)
Aikaikkuna: 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)
Aikaikkuna: 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

Yhteistyökumppanit ja tutkijat

Täältä löydät tähän tutkimukseen osallistuvat ihmiset ja organisaatiot.

Sponsori

Yhteistyökumppanit

Tutkijat

  • Päätutkija: Sunil Iyer, MD, Columbia University

Opintojen ennätyspäivät

Nämä päivämäärät seuraavat ClinicalTrials.gov-sivustolle lähetettyjen tutkimustietueiden ja yhteenvetojen edistymistä. National Library of Medicine (NLM) tarkistaa tutkimustiedot ja raportoidut tulokset varmistaakseen, että ne täyttävät tietyt laadunvalvontastandardit, ennen kuin ne julkaistaan ​​julkisella verkkosivustolla.

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Tutkimustietojen päivitykset

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Lisää tietoa

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