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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 listopada 2026 zaktualizowane przez: 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.

Przegląd badań

Status

Rekrutacyjny

Warunki

Interwencja / Leczenie

Szczegółowy opis

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.

Typ studiów

Interwencyjne

Zapisy (Szacowany)

45

Faza

  • Faza 1

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

Lokalizacje studiów

    • Florida
      • Miami, Florida, Stany Zjednoczone, 33136
        • Jeszcze nie rekrutacja
        • University of Miami Sylvester Comprehensive Cancer Center
        • Kontakt:
    • Illinois
      • Chicago, Illinois, Stany Zjednoczone, 60805
        • Jeszcze nie rekrutacja
        • University of Chicago Comprehensive Cancer Center
        • Kontakt:
    • New York
      • New York, New York, Stany Zjednoczone, 10032
    • Washington
      • Seattle, Washington, Stany Zjednoczone, 98109
        • Jeszcze nie rekrutacja
        • University of Washington Fred Hutchinson Comprehensive Cancer Center
        • Kontakt:

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

18 lat i starsze (Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

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 studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nie dotyczy
  • Model interwencyjny: Zadanie sekwencyjne
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: 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.

Inne nazwy:
  • 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.

Inne nazwy:
  • JA NIE
  • BESPONSA

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Number of adverse events
Ramy czasowe: 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
Ramy czasowe: 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

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Composite Complete Response (CRc) rate
Ramy czasowe: 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)
Ramy czasowe: 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)
Ramy czasowe: 36 Months

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

36 Months
Event-free survival (EFS)
Ramy czasowe: 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)
Ramy czasowe: 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

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Współpracownicy

Śledczy

  • Główny śledczy: Sunil Iyer, MD, Columbia University

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Szacowany)

31 marca 2027

Zakończenie podstawowe (Szacowany)

31 grudnia 2030

Ukończenie studiów (Szacowany)

31 grudnia 2030

Daty rejestracji na studia

Pierwszy przesłany

27 października 2026

Pierwszy przesłany, który spełnia kryteria kontroli jakości

9 listopada 2026

Pierwszy wysłany (Rzeczywisty)

13 listopada 2026

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

13 listopada 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

9 listopada 2026

Ostatnia weryfikacja

1 listopada 2026

Więcej informacji

Terminy związane z tym badaniem

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIEZDECYDOWANY

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Tak

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .