Asciminib and Inotuzumab Ozogamicin for Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (R/R Ph+ B-ALL)
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.
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Descrizione dettagliata
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 di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Fase 1
Contatti e Sedi
Contatto studio
Contatto studio
- Nome: Research Nurse Navigator
- Numero di telefono: 212-342-5162
- Email: cancerclinicaltrials@cumc.columbia.edu
Luoghi di studio
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Florida
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Miami, Florida, Stati Uniti, 33136
- Non ancora reclutamento
- University of Miami Sylvester Comprehensive Cancer Center
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Contatto:
- Terrence Bradley, MD
- Numero di telefono: (305) 243-5302
- Email: tbradley@med.miami.edu
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Illinois
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Chicago, Illinois, Stati Uniti, 60805
- Non ancora reclutamento
- University of Chicago Comprehensive Cancer Center
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Contatto:
- Anand Ashwin Patel, MD
- Numero di telefono: (708) 229-6001
- Email: Anand.patel@bsd.uchicago.edu
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New York
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New York, New York, Stati Uniti, 10032
- Reclutamento
- Columbia University Irving Medical Center
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Contatto:
- Research Nurse Navigator
- Numero di telefono: 646-317-5077
- Email: cancerclinicaltrials@cumc.columbia.edu
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Washington
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Seattle, Washington, Stati Uniti, 98109
- Non ancora reclutamento
- University of Washington Fred Hutchinson Comprehensive Cancer Center
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Contatto:
- Ryan Cassaday, MD
- Numero di telefono: (855) 557-0555
- Email: rcassada@fredhutch.edu
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Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
Inclusion Criteria:
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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
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Disease must be positive for Ph (p190 or p210 transcript) by cytogenetics or PCR
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Disease must be positive for CD22, defined as any percentage of leukemic blasts expressing CD22
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Disease must be positive for t(9;22) by Cytogenetics and FISH. Subjects with complex translocations resulting in BCR::ABL1 fusion protein are eligible.
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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
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Subject must have recovered (no worse than Grade 1 severity) from effects of all previous treatments.
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Able to provide informed consent
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Age ≥18 years of age
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Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 2 or Karnofsky ≥60%
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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
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Female patients of childbearing potential must not be nursing or planning to become pregnant
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Female patients of childbearing potential require a negative urine or serum pregnancy test within 30 days of study therapy.
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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.
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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
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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:
- 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)
- 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
- 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
- Prior allogeneic stem cell transplant < 90 days prior to anticipated treatment start
- Patients with active GVHD (graft versus host disease) and/or receiving immunosuppressive drugs for GVHD
- Patients receiving calcineurin inhibitors < 4 weeks prior to anticipated treatment start
- Ph-directed TKI within 5 days of treatment start
- Prior exposure to INO
- Prior exposure to asciminib
- The presence of mixed phenotype acute leukemia or CML in lymphoid blast phase
- 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
- Patients with T315I mutation as detected by ABL1 kinase mutational panel are excluded from asciminib dose levels lower than 200mg BID
- Clinical suspicion for active CNS involvement by ALL; previously treated CNS involvement is allowed
- Second malignancy requiring treatment within 6 months of study enrollment, with the exception of non-melanoma skin cancers or cancers requiring hormonal therapy only
- Active, uncontrolled infection
- Human immunodeficiency virus (HIV) not controlled by standard therapy
- Active hepatitis B or C infection. Participants whose infections are controlled with antiviral therapy are allowed.
- Patients with end stage renal disease requiring dialysis
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione sequenziale
- Mascheramento: Nessuno (etichetta aperta)
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
|---|---|
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Sperimentale: 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:
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.
Altri nomi:
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.
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of adverse events
Lasso di tempo: 36 Months
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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
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Maximum Tolerated Dose (MTD) of asciminib
Lasso di tempo: 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
|
Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Composite Complete Response (CRc) rate
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 36 Months
|
OS is defined as the time from study entry until death from any cause |
36 Months
|
|
Event-free survival (EFS)
Lasso di tempo: 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)
Lasso di tempo: 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
|
Collaboratori e investigatori
Sponsor
Sponsor
Collaboratori
Collaboratori
Investigatori
Investigatori
- Investigatore principale: Sunil Iyer, MD, Columbia University
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Inizio studio
Completamento primario (Stimato)
Completamento primario
Completamento dello studio (Stimato)
Completamento dello studio
Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Primo Inserito
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento pubblicato
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Processi patologici
- Neoplasie
- Attributi della malattia
- Malattie del sistema immunitario
- Infezioni
- Malattie virali
- Neoplasie per tipo istologico
- Infezioni da virus del DNA
- Malattie linfatiche
- Malattie linfoproliferative
- Disturbi immunoproliferativi
- Linfoma non Hodgkin
- Linfoma, cellule B
- Linfoma
- Infezioni da virus di Epstein-Barr
- Infezioni da Herpesviridae
- Infezioni da virus tumorali
- Condizioni patologiche, segni e sintomi
- Malattie emiche e linfatiche
- Ricorrenza
- Linfoma di Burkitt
- Aminoacidi, peptidi e proteine
- Proteine
- Carboidrati
- Glicosidi
- Anticorpi, monoclonali, umanizzati
- Anticorpi, monoclonali
- Anticorpi
- Immunoglobuline
- Immunoproteine
- Proteine del sangue
- Globuline sieriche
- Globuline
- Aminoglicosidi
- Calicheamicins
- Inotuzumab ozogamicina
- asciminib
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- ACYY0048
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Informazioni su farmaci e dispositivi, documenti di studio
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