Chronic Myelogenous LeukemiaPhase 3NCT03106779
Study of Efficacy of CML-CP Patients Treated With ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIs
A Phase 3 study testing Asciminib and Bosutinib in people with Chronic Myelogenous Leukemia, run by Novartis Pharmaceuticals at 88 sites in 25 countries.
Trial summary
The purpose of this pivotal study was to compare the efficacy of asciminib (ABL001) with that of bosutinib in the treatment of participants with chronic myeloid leukemia - chronic phase (CML-CP) having previously been treated with a minimum of two prior ATP-binding site tyrosine kinase inhibitors (TKIs). Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Chronic Myelogenous Leukemia
- What is being tested?
- Asciminib and Bosutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 233 participants
- Where
- 88 sitesin 25 countries
- Study dates
- 26 Oct 2017to 4 Dec 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients with a diagnosis of CML-CP ≥ 18 years of age
- Patients must meet all of the following laboratory values at the screening visit:
- < 15% blasts in peripheral blood and bone marrow
- < 30% blasts plus promyelocytes in peripheral blood and bone marrow
- < 20% basophils in the peripheral blood
- ≥ 50 x 109/L (≥ 50,000/mm3) platelets
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- Transient prior therapy related thrombocytopenia (< 50,000/mm3 for ≤ 30 days prior to screening) is acceptable
- No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly
- BCR-ABL1 ratio > 0.1% IS according to central laboratory at the screening examination for patients intolerant to the most recent TKI therapy
- Prior treatment with a minimum of 2 prior ATP-binding site TKIs (i.e. imatinib, nilotinib, dasatinib, radotinib or ponatinib)
- Failure (adapted from the 2013 ELN Guidelines Bacarrani 2013) or intolerance to the most recent TKI therapy at the time of screening
- Failure is defined for CML-CP patients (CP at the time of initiation of last therapy) as follows. Patients must meet at least 1 of the following criteria.
- Three months after the initiation of therapy: No CHR or > 95% Ph+ metaphases
- Six months after the initiation of therapy: BCR-ABL1 ratio > 10% IS and/or > 65% Ph+ metaphases
- Twelve months after initiation of therapy: BCR-ABL1 ratio > 10% IS and/or > 35% Ph+ metaphases
- At any time after the initiation of therapy, loss of CHR, CCyR or PCyR
- At any time after the initiation of therapy, the development of new BCR-ABL1 mutations which potentially cause resistance to study treatment
- At any time after the initiation of therapy, confirmed loss of MMR in 2 consecutive tests, of which one must have a BCR-ABL1 ratio ≥ 1% IS
- At any time after the initiation of therapy, new clonal chromosome abnormalities in Ph+ cells: CCA/Ph+
- Intolerance is defined as:
- Non-hematologic intolerance: Patients with grade 3 or 4 toxicity while on therapy, or with persistent grade 2 toxicity, unresponsive to optimal management, including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal)
- Hematologic intolerance: Patients with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses recommended by manufacturer
Who cannot join
- Known presence of the T315I or V299L mutation at any time prior to study entry Known second chronic phase of CML after previous progression to AP/BC Previous treatment with a hematopoietic stem-cell transplantation Patient planning to undergo allogeneic hematopoietic stem cell transplantation
- Cardiac or cardiac repolarization abnormality, including any of the following:
- History within 6 months prior to starting study treatment of myocardial infarction (MI), angina pectoris, coronary artery bypass graft (CABG)
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block)
- QTcF at screening ≥450 msec (male patients), ≥460 msec (female patients)
- Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
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- Risk factors for Torsades de Pointes (TdP) including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/symptomatic bradycardia
- Concomitant medication(s) with a known risk of Torsades de Pointes per www.crediblemeds.org that cannot be discontinued or replaced 7 days prior to starting study drug by safe alternative medication.
- Inability to determine the QTcF interval
- Severe and/or uncontrolled 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, pulmonary hypertension)
- History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
- History of acute or chronic liver disease
- Treatment with medications that meet one of the following criteria and that cannot be discontinued at least one week prior to the start of treatment with study treatment
- Moderate or strong inducers of CYP3A
- Moderate or strong inhibitors of CYP3A
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 3 days after last dose of ABL001 and one month after last dose of bosutinib. Highly effective contraception methods include:
- Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
- Female sterilization (have had surgical bilateral oophorectomy (with or without hysterectomy) total hysterectomy or bilateral tubal ligation at least six weeks before taking study treatment). In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
- Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject.
- Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
- In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks before taking study medication. In the case of oophorectomy alone, women are considered post-menopausal and not of child bearing potential only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
Trial locations and contacts
88 sites in 25 countries.
88 sites in 25 countries
United States10 sites
University of Michigan Clinical Trials Office
Ann Arbor, Michigan, 48109
Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, 21205
Indiana Blood and Marrow Institute
Beech Grove, Indiana, 46107
Dana Farber Cancer Center
Boston, Massachusetts, 02215
Roswell Park Cancer Institute
Buffalo, New York, 14263
University of Chicago Hospital
Chicago, Illinois, 60637
Uni Of TX MD Anderson Cancer Cntr
Houston, Texas, 77030
Weill Cornell Medicine NY-Presb
New York, New York, 10021
Memorial Sloan Kettering Cancer Ctr
New York, New York, 10065
Utah Huntsman Cancer Center
Salt Lake City, Utah, 84112
Japan10 sites
Novartis Investigative Site
Akita, 0108543
Novartis Investigative Site
Aomori, 0308553
Novartis Investigative Site
Bunkyo Ku, Tokyo, 1138677
Novartis Investigative Site
Chūō, Yamanashi, 409-3898
Novartis Investigative Site
Kashiwa, Chiba, 277-8577
Novartis Investigative Site
Kobe, Hyōgo, 650-0017
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511
Novartis Investigative Site
Sakai, Osaka, 590-0197
Novartis Investigative Site
Suita, Osaka, 5650871
Novartis Investigative Site
Toyoake, Aichi-ken, 4701192
Germany7 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Düsseldorf, North Rhine-Westphalia, 40225
Novartis Investigative Site
Frankfurt am Main, Hesse, 60590
Novartis Investigative Site
Heidelberg, 69120
Novartis Investigative Site
Jena, Thuringia, 07740
Novartis Investigative Site
Kiel, 24116
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, 68305
France5 sites
Novartis Investigative Site
Bordeaux, 33076
Novartis Investigative Site
Lyon, 69373
Novartis Investigative Site
Marseille, 13273
Novartis Investigative Site
Paris, 75475
Novartis Investigative Site
Vandœuvre-lès-Nancy, 54511
Spain5 sites
Novartis Investigative Site
Barcelona, 08036
Novartis Investigative Site
Bilbao, Bizkaia, 48013
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, 08907
Novartis Investigative Site
Madrid, 28034
Novartis Investigative Site
Toledo, Castille-La Mancha, 45071
Turkey (Türkiye)5 sites
Novartis Investigative Site
Adana, Saricam, 01330
Novartis Investigative Site
Istanbul, Fatih, 34093
Novartis Investigative Site
Istanbul, Fatih, 34098
Novartis Investigative Site
Izmir, 35100
Novartis Investigative Site
Samsun, Atakum, 55200
United Kingdom5 sites
Novartis Investigative Site
Cardiff, CF14 4XW
Novartis Investigative Site
Glasgow, G12 0YN
Novartis Investigative Site
London, W12 0HS
Novartis Investigative Site
Metropolitan Borough of Wirral, Merseyside, CH63 4JY
Novartis Investigative Site
Oxford, OX3 7LE
Brazil4 sites
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, 90035-003
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, 20211-030
Novartis Investigative Site
São Paulo, São Paulo, 05403 000
Novartis Investigative Site
São Paulo, São Paulo, 08270-070
Russia4 sites
Novartis Investigative Site
Moscow, 125167
Novartis Investigative Site
Moscow, 125284
Novartis Investigative Site
Saint Petersburg, 191024
Novartis Investigative Site
Saint Petersburg, 197341
South Korea4 sites
Novartis Investigative Site
Busan, 49201
Novartis Investigative Site
Jeollanam, 519763
Novartis Investigative Site
Seoul, 06591
Novartis Investigative Site
Uijeongbu-si, Gyeonggi-do, 11759
Argentina3 sites
Novartis Investigative Site
CABA, Buenos Aires, C1221ADH
Novartis Investigative Site
Capital Federal, C1114AAN
Novartis Investigative Site
Córdoba, X5016KEH
Australia3 sites
Novartis Investigative Site
Adelaide, South Australia, 5000
Novartis Investigative Site
Melbourne, Victoria, 3000
Novartis Investigative Site
Murdoch, Western Australia, 6150
Italy3 sites
Novartis Investigative Site
Bari, BA, 70124
Novartis Investigative Site
Milan, MI, 20122
Novartis Investigative Site
Naples, 80131
Bulgaria2 sites
Novartis Investigative Site
Plovdiv, 4002
Novartis Investigative Site
Varna, 9000
Czechia2 sites
Novartis Investigative Site
Brno, 625 00
Novartis Investigative Site
Ostrava, Poruba, 708 52
Hungary2 sites
Novartis Investigative Site
Budapest, H-1097
Novartis Investigative Site
Debrecen, Hajdu Bihar Megye, 4032
Israel2 sites
Novartis Investigative Site
Jerusalem, 9112001
Novartis Investigative Site
Tsrifin, 7030000
Lebanon2 sites
Novartis Investigative Site
Beirut, 1100 2070
Novartis Investigative Site
Beirut, 113-0236
Netherlands2 sites
Novartis Investigative Site
Amsterdam, North Holland, 1081 HV
Novartis Investigative Site
Dordrecht, South Holland, 3318 AT
Romania2 sites
Novartis Investigative Site
Cluj-Napoca, Cluj, 400015
Novartis Investigative Site
Timișoara, 300079
Serbia2 sites
Novartis Investigative Site
Belgrade, 11000
Novartis Investigative Site
Novi Sad, 400107
About this study
Official title: A Phase 3, Multi-center, Open-label, Randomized Study of Oral ABL001 Versus Bosutinib in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors
The purpose of this pivotal study was to compare the efficacy of asciminib (ABL001) with that of bosutinib in the treatment of participants with chronic myeloid leukemia - chronic phase (CML-CP) having previously been treated with a minimum of two prior ATP-binding site tyrosine kinase inhibitors (TKIs).
Read the detailed description
Participants with a diagnosis of CML-CP who had received prior treatment with 2 or more ATP binding site TKIs and were treatment failure (as per guidelines adapted from the 2013 ELN recommendations) or intolerant to the most recent TKI.
Participants were randomized in a 2:1 ratio to asciminib 40 mg BID or bosutinib 500 mg QD. Randomization was stratified by major cytogenetic response (MCyR) at screening.
Participants with documented treatment failure (as per the 2013 ELN recommendations) while on bosutinib treatment were offered the option to switch to asciminib treatment within 96 weeks after the last patient was randomized to the study.
- Conditions
- Chronic Myelogenous Leukemia
- Being tested
- Asciminib, Bosutinib
- Keywords
- Phase 3, Chronic Myelogenous Leukemia, CML, bosutinib, ABL001, tyrosine kinase inhibitor, Chronic myelogenous leukemia (CML), chronic myeloid leukemia (CML), chronic myelocytic leukemia (CML), chronic granulocytic leukemia (CGL), cancer of the white blood cells, clonal bone marrow stem cell disorder, proliferation of mature granulocytes
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AsciminibDrug
Also called ABL001
40 mg tablets was taken orally twice a day (BID)
BosutinibDrug
500 mg tablets was taken orally once daily (QD)
| Study group | Role | What they receive |
|---|---|---|
| Asciminib | Experimental | Patients were randomized to asciminib 40mg BID Receives: Drug: Asciminib |
| Bosutinib | Active comparator | Patients were randomized to bosutinib 500mg QD Receives: Drug: Bosutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Major Molecular Response (MMR) Rate at 24 Weeks
MMR was defined as a ≥ 3.0 log reduction in BCR-ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1% BCR-ABL1/ABL% by international scale (IS) as measured by RQ-PCR.
Measured: 24 weeks
Secondary outcomes (17)
Major Molecular Response (MMR) Rate at 96 Weeks (Key Secondary Endpoint)
MMR at 96 weeks was defined as the percentage of participants with MMR at 96 weeks. MMR was defined as a ≥ 3.0 log reduction in BCR-ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1% BCR-ABL1/ABL% by international scale (IS) as measured by RQ-PCR.
Measured: 96 Weeks
Complete Cytogenetic Response (CCyR) Rate at Scheduled Time Points
Cytogenic response included Complete, Partial, Major, Minor, Minimal and no response. Cytogenetic response was assessed as the percentage of Ph+ metaphases in the bone marrow and is defined as the following: Complete (CCyR) - 0% Ph+ metaphases; Partial (PCyR) - >0 to 35% Ph+ metaphases; Major (MCyR) - 0 to 35% Ph+ metaphases; Minor (mCyR) - >35 to 65% Ph+ metaphases; Minimal - >65 to 95% Ph+ metaphases; None - >95 to 100% Ph+ metaphases.
Measured: at 24, 48, 72, 96, 120 and 144 weeks
Complete Cytogenetic Response Rate by Scheduled Time Points
Cytogenic response included Complete, Partial, Major, Minor, Minimal and no response. Cytogenetic response was assessed as the percentage of Ph+ metaphases in the bone marrow and is defined as the following: Complete (CCyR) - 0% Ph+ metaphases; Partial (PCyR) - >0 to 35% Ph+ metaphases; Major (MCyR) - 0 to 35% Ph+ metaphases; Minor (mCyR) - >35 to 65% Ph+ metaphases; Minimal - >65 to 95% Ph+ metaphases; None - >95 to 100% Ph+ metaphases.
Measured: by 24, 48, 72, 96, 120 and 144 weeks
Major Molecular Response (MMR) Rate at All Scheduled Data Collection Time Points Except Weeks 24 & 96
MMR was defined as a ≥ 3.0 log reduction in BCR-ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1% BCR-ABL1/ABL% by international scale (IS) as measured by RQ-PCR.
Measured: at Weeks 36, 48, 60, 72, 84, 108, 120, 132, 144 & 156
Major Molecular Response (MMR) Rate by All Scheduled Data Collection Time Points
MMR was defined as a ≥ 3.0 log reduction in BCR-ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1% BCR-ABL1/ABL% by international scale (IS) as measured by RQ-PCR. Overall time point has the count indicating participants achieving MMR at any time during the study.
Measured: by Weeks 4, 8, 12, 16, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144 & 156, Overall
Time to Major Molecular Response (MMR)
Time to MMR was defined as the time from the date of randomization to the date of the first documented MMR.
Measured: 216 Weeks
Duration of Major Molecular Response (MMR)
Duration of MMR was defined as the time from the date of first documented MMR to the earliest date of loss of MMR, progression to Accelerated phase (AP) or Blast crisis (BC), or Chronic myeloid leukemia (CML)-related death.
Measured: 216 Weeks
Time to Complete Cytogenetic Response Rate (CCyR) Among Participants Who Achieved CCyR
Time to CCyR was defined as the time from the date of randomization to the date of the first documented CCyR.
Measured: 216 Weeks
Duration of Complete Cytogenetic Response (CCyR)
Duration of CCyR was defined as the time between date of first documented CCyR and the earliest date of loss of CCyR, progression to AP/BC, or CML-related death for participants in the Cytogenetic Responder Set. The time was censored at the last cytogenetic assessment date on treatment for participants for whom none of the events was reported or last PCR evaluation on treatment indicating MMR.
Measured: 144 weeks
Time to Treatment Failure (TTF)
TTF was defined as the time from date of randomization to an event of treatment failure. Treatment failure was defined as meeting a lack of efficacy criterion or discontinuing treatment due to any reason.
Measured: From the first dose of treatment up to 5 years, through treatment completion, an average of 2.3 years, approximately
Progression Free Survival Per Percentage Event-free Kaplan-Meier Estimates
Progression-free survival was defined as the time from the date of randomization to the earliest occurrence of documented disease progression to AP/BC or the date of death from any cause (including progressions and deaths observed during the survival follow-up period), per Kaplan-Meier.
Measured: From the first dose of treatment up to study completion, up to 5 years
Overall Survival (OS) Per Event Free Percentage Per Kaplan-Meier Estimates
Overall survival is defined as the time from the date of randomization to the date of death (including the survival follow-up period) per Kaplan-Meier (KM).
Measured: From the first dose of treatment to study completion, up to 5 years
Trough Plasma Concentrations for Asciminib
Measured concentration at the end of a dosing interval at steady state (taken directly before next administration)
Measured: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: Cmax for Asciminib
Maximum (peak) observed plasma concentration after dose administration (mass x volume-1).
Measured: Week 2 Day 1 at pre-dose, 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: Tmax for Asciminib
Time to reach maximum (peak) plasma concentration after dose administration (time). Actual sampling times were taken into consideration for the pharmacokinetic analysis.
Measured: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: AUC0-12h for Asciminib
Area under the plasma concentration-time curve from time zero to 12 hours (mass x time x volume-1)
Measured: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: CL/F for Asciminib
Total apparent body clearance of drug from the plasma after oral administration (volume x time-1).
Measured: Week 2 day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2017
- Study start26 Oct 2017
- Primary completion25 May 2020
- Study completion4 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Chronic Myelogenous LeukemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 233 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03106779
- Sponsor's protocol ID
- CABL001A2301
- Other IDs
- 2016-002461-66, 2016
- Status last verified
- Jul 2026
- First submitted
- 9 Mar 2017
- Last updated
- 18 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com
Publications
Mauro MJ, Minami Y, Hochhaus A, Lomaia E, Voloshin S, Turkina A, Kim DW, Apperley JF, Cortes JE, Abdo A, Fogliatto LM, Kim DDH, le Coutre P, Saussele S, Annunziata M, Hughes TP, Chaudhri N, Chee L, Garcia-Gutierrez V, Sasaki K, Boquimpani C, Kapoor S, Espurz N, Dhamal V, Rea D. Asciminib remained superior vs bosutinib in late-line CML-CP after nearly 4 years of follow-up in ASCEMBL. Blood Adv. 2025 Aug 26;9(16):4248-4259. doi: 10.1182/bloodadvances.2025016042.
PubMed 40334072Cortes JE, Rea D, Mauro MJ, Tran D, Wang P, Jadhav K, Yocolly A, Sasaki K. Health care resource utilization in 3L + patients with chronic phase chronic myeloid leukemia receiving asciminib or bosutinib. J Med Econ. 2023 Jan-Dec;26(1):915-923. doi: 10.1080/13696998.2023.2234776.
PubMed 37431294Yuda J, Doki N, Matsuoka H, Yokota T, Tomita A, Takahashi N, Matsumura I, Kubo K, Goto T, Kirito K, Maki A, Aoki M, Allepuz A, Minami Y. Asciminib vs bosutinib in CML patients pretreated with >/=2 tyrosine kinase inhibitors: Results from the Japanese subgroup analysis of ASCEMBL study. Cancer Med. 2023 Feb;12(3):2990-2998. doi: 10.1002/cam4.5212. Epub 2022 Sep 27.
PubMed 36168187Li YF, Combes FP, Hoch M, Lorenzo S, Sy SKB, Ho YY. Population Pharmacokinetics of Asciminib in Tyrosine Kinase Inhibitor-Treated Patients with Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic and Acute Phases. Clin Pharmacokinet. 2022 Oct;61(10):1393-1403. doi: 10.1007/s40262-022-01148-9. Epub 2022 Jun 28.
PubMed 35764773Rea D, Mauro MJ, Boquimpani C, Minami Y, Lomaia E, Voloshin S, Turkina A, Kim DW, Apperley JF, Abdo A, Fogliatto LM, Kim DDH, le Coutre P, Saussele S, Annunziata M, Hughes TP, Chaudhri N, Sasaki K, Chee L, Garcia-Gutierrez V, Cortes JE, Aimone P, Allepuz A, Quenet S, Bedoucha V, Hochhaus A. A phase 3, open-label, randomized study of asciminib, a STAMP inhibitor, vs bosutinib in CML after 2 or more prior TKIs. Blood. 2021 Nov 25;138(21):2031-2041. doi: 10.1182/blood.2020009984.
PubMed 34407542
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multi-center, Open-label, Randomized Study of Oral ABL001 Versus Bosutinib in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors
- ClinicalTrials.gov ID
- NCT03106779
- Acronym / sponsor ID
- CABL001A2301
- Conditions
- Chronic Myelogenous Leukemia
- Keywords
- Phase 3, Chronic Myelogenous Leukemia, CML, bosutinib, ABL001, tyrosine kinase inhibitor, Chronic myelogenous leukemia (CML), chronic myeloid leukemia (CML)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 233 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 26 Oct 2017 (actual)
- Primary completion
- 25 May 2020 (actual)
- Study completion
- 4 Dec 2024 (actual)
- First posted
- 10 Apr 2017 (actual)
- Results
- Posted 15 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this pivotal study was to compare the efficacy of asciminib (ABL001) with that of bosutinib in the treatment of participants with chronic myeloid leukemia - chronic phase (CML-CP) having previously been treated with a minimum of two prior ATP-binding site tyrosine kinase inhibitors (TKIs).
Detailed description
Participants with a diagnosis of CML-CP who had received prior treatment with 2 or more ATP binding site TKIs and were treatment failure (as per guidelines adapted from the 2013 ELN recommendations) or intolerant to the most recent TKI.
Participants were randomized in a 2:1 ratio to asciminib 40 mg BID or bosutinib 500 mg QD. Randomization was stratified by major cytogenetic response (MCyR) at screening.
Participants with documented treatment failure (as per the 2013 ELN recommendations) while on bosutinib treatment were offered the option to switch to asciminib treatment within 96 weeks after the last patient was randomized to the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Asciminib | Experimental | Drug: AsciminibPatients were randomized to asciminib 40mg BID |
| Bosutinib | Active comparator | Drug: BosutinibPatients were randomized to bosutinib 500mg QD |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Male or female patients with a diagnosis of CML-CP ≥ 18 years of age
- < 15% blasts in peripheral blood and bone marrow
- < 30% blasts plus promyelocytes in peripheral blood and bone marrow
- < 20% basophils in the peripheral blood
- ≥ 50 x 109/L (≥ 50,000/mm3) platelets
Key exclusion criteria (22 listed)
- Known presence of the T315I or V299L mutation at any time prior to study entry Known second chronic phase of CML after previous progression to AP/BC Previous treatment with a hematopoietic stem-cell transplantation Patient planning to undergo allogeneic hematopoietic stem cell transplantation
- History within 6 months prior to starting study treatment of myocardial infarction (MI), angina pectoris, coronary artery bypass graft (CABG)
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block)
- QTcF at screening ≥450 msec (male patients), ≥460 msec (female patients)
- Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AsciminibAlso: ABL00140 mg tablets was taken orally twice a day (BID) | Drug | Asciminib |
| Bosutinib500 mg tablets was taken orally once daily (QD) | Drug | Bosutinib |
Primary outcome
Major Molecular Response (MMR) Rate at 24 Weeks
Time frame: 24 weeks
MMR was defined as a ≥ 3.0 log reduction in BCR-ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1% BCR-ABL1/ABL% by international scale (IS) as measured by RQ-PCR.
17 secondary outcomes
Major Molecular Response (MMR) Rate at 96 Weeks (Key Secondary Endpoint)
Time frame: 96 Weeks
Complete Cytogenetic Response (CCyR) Rate at Scheduled Time Points
Time frame: at 24, 48, 72, 96, 120 and 144 weeks
Complete Cytogenetic Response Rate by Scheduled Time Points
Time frame: by 24, 48, 72, 96, 120 and 144 weeks
Major Molecular Response (MMR) Rate at All Scheduled Data Collection Time Points Except Weeks 24 & 96
Time frame: at Weeks 36, 48, 60, 72, 84, 108, 120, 132, 144 & 156
Major Molecular Response (MMR) Rate by All Scheduled Data Collection Time Points
Time frame: by Weeks 4, 8, 12, 16, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144 & 156, Overall
Time to Major Molecular Response (MMR)
Time frame: 216 Weeks
Duration of Major Molecular Response (MMR)
Time frame: 216 Weeks
Time to Complete Cytogenetic Response Rate (CCyR) Among Participants Who Achieved CCyR
Time frame: 216 Weeks
Duration of Complete Cytogenetic Response (CCyR)
Time frame: 144 weeks
Time to Treatment Failure (TTF)
Time frame: From the first dose of treatment up to 5 years, through treatment completion, an average of 2.3 years, approximately
Progression Free Survival Per Percentage Event-free Kaplan-Meier Estimates
Time frame: From the first dose of treatment up to study completion, up to 5 years
Overall Survival (OS) Per Event Free Percentage Per Kaplan-Meier Estimates
Time frame: From the first dose of treatment to study completion, up to 5 years
Trough Plasma Concentrations for Asciminib
Time frame: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: Cmax for Asciminib
Time frame: Week 2 Day 1 at pre-dose, 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: Tmax for Asciminib
Time frame: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: AUC0-12h for Asciminib
Time frame: Week 2 Day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
PK Parameter: CL/F for Asciminib
Time frame: Week 2 day 1 at: 0hr (pre-dose), 0.5hr, 1hr, 2hr, 3hr, 4hr, 6hr, 8hr & 12hr post-dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40334072Mauro MJ, Minami Y, Hochhaus A, Lomaia E, Voloshin S, Turkina A, Kim DW, Apperley JF, Cortes JE, Abdo A, Fogliatto LM, Kim DDH, le Coutre P, Saussele S, Annunziata M, Hughes TP, Chaudhri N, Chee L, Garcia-Gutierrez V, Sasaki K, Boquimpani C, Kapoor S, Espurz N, Dhamal V, Rea D. Asciminib remained superior vs bosutinib in late-line CML-CP after nearly 4 years of follow-up in ASCEMBL. Blood Adv. 2025 Aug 26;9(16):4248-4259. doi: 10.1182/bloodadvances.2025016042.
- Derived from this study: PubMed 37431294Cortes JE, Rea D, Mauro MJ, Tran D, Wang P, Jadhav K, Yocolly A, Sasaki K. Health care resource utilization in 3L + patients with chronic phase chronic myeloid leukemia receiving asciminib or bosutinib. J Med Econ. 2023 Jan-Dec;26(1):915-923. doi: 10.1080/13696998.2023.2234776.
- Derived from this study: PubMed 36168187Yuda J, Doki N, Matsuoka H, Yokota T, Tomita A, Takahashi N, Matsumura I, Kubo K, Goto T, Kirito K, Maki A, Aoki M, Allepuz A, Minami Y. Asciminib vs bosutinib in CML patients pretreated with >/=2 tyrosine kinase inhibitors: Results from the Japanese subgroup analysis of ASCEMBL study. Cancer Med. 2023 Feb;12(3):2990-2998. doi: 10.1002/cam4.5212. Epub 2022 Sep 27.
- Derived from this study: PubMed 35764773Li YF, Combes FP, Hoch M, Lorenzo S, Sy SKB, Ho YY. Population Pharmacokinetics of Asciminib in Tyrosine Kinase Inhibitor-Treated Patients with Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic and Acute Phases. Clin Pharmacokinet. 2022 Oct;61(10):1393-1403. doi: 10.1007/s40262-022-01148-9. Epub 2022 Jun 28.
- Derived from this study: PubMed 34407542Rea D, Mauro MJ, Boquimpani C, Minami Y, Lomaia E, Voloshin S, Turkina A, Kim DW, Apperley JF, Abdo A, Fogliatto LM, Kim DDH, le Coutre P, Saussele S, Annunziata M, Hughes TP, Chaudhri N, Sasaki K, Chee L, Garcia-Gutierrez V, Cortes JE, Aimone P, Allepuz A, Quenet S, Bedoucha V, Hochhaus A. A phase 3, open-label, randomized study of asciminib, a STAMP inhibitor, vs bosutinib in CML after 2 or more prior TKIs. Blood. 2021 Nov 25;138(21):2031-2041. doi: 10.1182/blood.2020009984.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
88 sites in 25 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Asciminib and Bosutinib in people with Chronic Myelogenous Leukemia, run by Novartis Pharmaceuticals at 88 sites in 25 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 88 sites in United States, Japan, Germany, France, Spain and 20 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Major Molecular Response (MMR) Rate at 24 Weeks (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03106779, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Aug 2026.