Chronic Myelogenous LeukemiaPhase 1NCT02081378
A Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL
A Phase 1 study testing Asciminib (ABL001), Nilotinib and Imatinib in people with Chronic Myelogenous Leukemia and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals at 18 sites in 10 countries.
Trial summary
The design of a phase I, open label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent ABL001 in Chronic myeloid leukemia (CML) and Philadelphia chromosome positive Acute lymphoblastic leukemia (Ph+ ALL) patients who are relapsed or refractory to or are intolerant of… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myelogenous Leukemia, Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia
- What is being tested?
- Asciminib (ABL001), Nilotinib and Imatinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 326 participants
- Where
- 18 sitesin 10 countries
- Study dates
- 24 Apr 2014to 14 Mar 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- For CML patients either:
- a. Patients with Ph+ CML in chronic or accelerated phase who were previously treated with at least two different tyrosine kinase inhibitors prior to study entry and are relapsed, refractory to or intolerant of TKIs as determined by investigators or
- b. Patients with CML in chronic or accelerated phase who exhibit relapsed disease associated with the presence of the T315I "gatekeeper mutation" after at least one TKI are also eligible provided that no other effective therapy exists
- For ALL and CML-BP patients:
- Patients with CML BP or Ph+ ALL who have a cytopathologically confirmed diagnosis and are relapsed or refractory to at least one prior TKI or intolerant of TKIs. TKI failure for Ph+ ALL and CML-BP patients is defined as at least the loss of Molecular Response (MR) 4.5 (BCR-ABL ≤ 0.0032%)
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Willingness and ability to comply with all study procedures
- Written informed consent obtained prior to any screening procedures
Who cannot join
- Wash-out period:
- Systemic antineoplastic therapy (including cytotoxic chemotherapy, alfa-interferon and toxin immunoconjugates) or any experimental therapy within 14 days or 5 half-lives, whichever is shorter, before the first dose of study treatment
- Therapy with TKIs as single agent within 5 half-lives before the first dose of study treatment
- Unconjugated monoclonal antibody therapies within 28 days or 5 half-lives, whichever is shorter, before the first dose of study treatment
- For patients receiving ABL001 in combination with either nilotinib or imatinib or dasatinib, intolerance to nilotinib, imatinib or dasatinib, respectively
- Radiotherapy with a wide field of radiation within 4 weeks or radiotherapy with a limited field of radiation for palliation within 1 week of the first dose of study treatment.
- CNS irradiation for meningeal leukemia, except if radiotherapy occurred > 3 months previously. At least four weeks must have elapsed since prophylactic CNS irradiation given as part of a front-line therapy regimen for ALL
- Major surgery within 2 weeks before the first dose of study treatment
Trial locations and contacts
18 sites in 10 countries.
18 sites in 10 countries
United States6 sites
University of Michigan Comprehensive Cancer Center SC
Ann Arbor, Michigan, 48109
Dana Farber Cancer Institute Hematology / Oncology
Boston, Massachusetts, 02215
University of Texas/MD Anderson Cancer Center UT MD Anderson
Houston, Texas, 77030-4009
Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering
New York, New York, 10065
Oregon Health Sciences University SC-6
Portland, Oregon, 97239
Huntsman Cancer Institute SC
Salt Lake City, Utah, 84112
Germany3 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Frankfurt, 60590
Novartis Investigative Site
Jena, 07740
France2 sites
Novartis Investigative Site
Bordeaux, 33076
Novartis Investigative Site
Paris, Cedex 10, 75475
About this study
Official title: A Phase I, Multicenter, Open-label Study of Oral ABL001 in Patients With Chronic Myelogenous Leukemia (CML) or Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL)
The design of a phase I, open label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent ABL001 in Chronic myeloid leukemia (CML) and Philadelphia chromosome positive Acute lymphoblastic leukemia (Ph+ ALL) patients who are relapsed or refractory to or are intolerant of Tyrosine kinase inhibitors (TKIs), and of ABL001+Nilotinib, ABL001+Imatinib and ABL001+Dasatinib in Ph positive CML patients who are relapsed or refractory to TKIs.
Read the detailed description
This first-in-human trial with ABL001 was a dose escalation study whose primary purpose was to estimate the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of single agent ABL001 in CML or Ph+ ALL patients, and in combination with either Nilotinib or Imatinib or Dasatinib in Ph positive CML patients. The safety, tolerability and pharmacokinetic (PK) profile of ABL001 and ABL001+Nilotinib, ABL001+Imatinib and ABL001+Dasatinib were assessed together with an evaluation of pharmacodynamic (PD) changes in peripheral blood mononuclear cells (PBMC) and bone marrow aspirates and all data could contribute to the assessment of the RDE.
An understanding of the MTD/RDE, safety profile, PK/PD relationship, and preliminary evidence of anti-CML and ALL activity wias used to inform future development in adults with CML and Ph+ ALL. By virtue of its distinct pharmacological profile and by preclinical pharmacological studies demonstrating an additive effect, a combination of ABL001 and a tyrosine-kinase inhibitor (TKI) has the potential to achieve a deeper molecular response in a higher proportion of CML patients as compared to single agent TKI therapy. Such a combination has the added advantage of targeting the ABL kinase domain at two distinct locations, theoretically preventing single point mutation-associated treatment resistance. The prediction is that a nilotinib+ABL001, imatinib+ABL001 and/or dasatinib+ABL001 combination will increase the percentage of patients who achieve a complete molecular response (CMR) and decrease the time to CMR, thereby increasing the possibility of achieving sustained treatment-free remissions in these patients. In addition, some patients could be intolerant of therapy with TKIs or could develop mutations that promote resistance to TKI therapy. In these patients, ABL001 could provide a novel therapeutic option.
- Conditions
- Chronic Myelogenous Leukemia, Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia
- Being tested
- Asciminib (ABL001), Nilotinib, Imatinib, Dasatinib
- Keywords
- CML, Ph+ ALL, asciminib, ABL001, Maximum Tolerated Dose (MTD), Recommended Dose for Expansion (RDE), relapsed or refractory, TKIs, Nilotinib, Imatinib, Dasatinib
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Asciminib (ABL001)Drug
Also called ABL001
Asciminib was be administered orally in a dose escalation schedule.
NilotinibDrug
Asciminib and Nilotinib was administered orally in CML patients
ImatinibDrug
Asciminib and imatinib was administered orally in CML patients
DasatinibDrug
Asciminib and dasatinib was administered orally in CML patients
| Study group | Role | What they receive |
|---|---|---|
| Asciminib in CML patients | Experimental | Dose escalation study estimated the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of asciminib in adult patients with chronic myeloid leukemia (CML). Receives: Drug: Asciminib (ABL001) |
| Asciminib+Nilotinib in CML patients | Experimental | Dose escalation study estimated the MTD and/or RDE of asciminib in combination with Nilotinib in adult CML patients Receives: Drug: Asciminib (ABL001); Drug: Nilotinib |
| Asciminib in Ph+ ALL patients | Experimental | Dose escalation study estimated the MTD and/or RDE of asciminib in adult patients with Ph positive ALL patients Receives: Drug: Asciminib (ABL001) |
| Asciminib+Imatinib in CML patients | Experimental | Dose escalation study to estimate the MTD and/or RDE of asciminib in combination with imatinib in adult CML patients Receives: Drug: Asciminib (ABL001); Drug: Imatinib |
| Asciminib+dasatinib in CML patients | Experimental | Dose escalation study estimated the MTD and/or RDE of asciminib in combination with dasatinib in adult CML patients Receives: Drug: Asciminib (ABL001); Drug: Dasatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose limiting toxicities (DLTs) during the first cycle of study treatment
Determine the MTD and/or RDE of ABL001 as single agent in CML and Ph+ ALL, and in combination with either nilotinib or imatinib or dasatinib in CML patients
Measured: First Cycle is 28 days
Secondary outcomes (10)
Hematologic Response
Measured: At screening and first day of cycle 2 and 3 and every 12 weeks afterwards
Cytogenetic response
Measured: at screening or when a patient's BCR-ABL ratio has risen to >1%
BCR-ABL transcript level
Measured: At screening and first day of cycle 2 and 3 and every 12 weeks afterwards
Cmax of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
Cmin of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUCinf of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUClast of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUCtau of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
T1/2 of ABL001 as measured in plasma
Measured: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
Adverse events
Measured: Collected from screening visit through post-treatment follow-up period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2014 (estimated)
- Study start24 Apr 2014
- Primary completion3 Jun 2021
- Study completion14 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 326 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
- NCT02081378
- Sponsor's protocol ID
- CABL001X2101
- Other IDs
- 2013-004491-36
- Status last verified
- Mar 2024
- First submitted
- 28 Feb 2014
- Last updated
- 18 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Luskin MR, Murakami MA, Keating J, Flamand Y, Winer ES, Garcia JS, Stahl M, Stone RM, Wadleigh M, Jaeckle SL, Hagopian E, Weinstock DM, Liegel J, McMasters M, Wang ES, Stock W, DeAngelo DJ. Asciminib plus dasatinib and prednisone for Philadelphia chromosome-positive acute leukemia. Blood. 2025 Feb 6;145(6):577-589. doi: 10.1182/blood.2024025800.
PubMed 39374521Cortes JE, Sasaki K, Kim DW, Hughes TP, Etienne G, Mauro MJ, Hochhaus A, Lang F, Heinrich MC, Breccia M, Deininger M, Goh YT, Janssen JJWM, Talpaz M, de Soria VGG, le Coutre P, DeAngelo DJ, Damon A, Cacciatore S, Polydoros F, Agrawal N, Rea D. Asciminib monotherapy in patients with chronic-phase chronic myeloid leukemia with the T315I mutation after >/=1 prior tyrosine kinase inhibitor: 2-year follow-up results. Leukemia. 2024 Jul;38(7):1522-1533. doi: 10.1038/s41375-024-02278-8. Epub 2024 May 16.
PubMed 38755421Li 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 35764773Hughes TP, Mauro MJ, Cortes JE, Minami H, Rea D, DeAngelo DJ, Breccia M, Goh YT, Talpaz M, Hochhaus A, le Coutre P, Ottmann O, Heinrich MC, Steegmann JL, Deininger MWN, Janssen JJWM, Mahon FX, Minami Y, Yeung D, Ross DM, Tallman MS, Park JH, Druker BJ, Hynds D, Duan Y, Meille C, Hourcade-Potelleret F, Vanasse KG, Lang F, Kim DW. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019 Dec 12;381(24):2315-2326. doi: 10.1056/NEJMoa1902328.
PubMed 31826340
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multicenter, Open-label Study of Oral ABL001 in Patients With Chronic Myelogenous Leukemia (CML) or Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL)
- ClinicalTrials.gov ID
- NCT02081378
- Acronym / sponsor ID
- CABL001X2101
- Conditions
- Chronic Myelogenous Leukemia, Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia
- Keywords
- CML, Ph+ ALL, asciminib, ABL001, Maximum Tolerated Dose (MTD), Recommended Dose for Expansion (RDE), relapsed or refractory, TKIs
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 326 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Apr 2014 (actual)
- Primary completion
- 3 Jun 2021 (actual)
- Study completion
- 14 Mar 2023 (actual)
- First posted
- 7 Mar 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The design of a phase I, open label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent ABL001 in Chronic myeloid leukemia (CML) and Philadelphia chromosome positive Acute lymphoblastic leukemia (Ph+ ALL) patients who are relapsed or refractory to or are intolerant of Tyrosine kinase inhibitors (TKIs), and of ABL001+Nilotinib, ABL001+Imatinib and ABL001+Dasatinib in Ph positive CML patients who are relapsed or refractory to TKIs.
Detailed description
This first-in-human trial with ABL001 was a dose escalation study whose primary purpose was to estimate the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of single agent ABL001 in CML or Ph+ ALL patients, and in combination with either Nilotinib or Imatinib or Dasatinib in Ph positive CML patients. The safety, tolerability and pharmacokinetic (PK) profile of ABL001 and ABL001+Nilotinib, ABL001+Imatinib and ABL001+Dasatinib were assessed together with an evaluation of pharmacodynamic (PD) changes in peripheral blood mononuclear cells (PBMC) and bone marrow aspirates and all data could contribute to the assessment of the RDE.
An understanding of the MTD/RDE, safety profile, PK/PD relationship, and preliminary evidence of anti-CML and ALL activity wias used to inform future development in adults with CML and Ph+ ALL. By virtue of its distinct pharmacological profile and by preclinical pharmacological studies demonstrating an additive effect, a combination of ABL001 and a tyrosine-kinase inhibitor (TKI) has the potential to achieve a deeper molecular response in a higher proportion of CML patients as compared to single agent TKI therapy. Such a combination has the added advantage of targeting the ABL kinase domain at two distinct locations, theoretically preventing single point mutation-associated treatment resistance. The prediction is that a nilotinib+ABL001, imatinib+ABL001 and/or dasatinib+ABL001 combination will increase the percentage of patients who achieve a complete molecular response (CMR) and decrease the time to CMR, thereby increasing the possibility of achieving sustained treatment-free remissions in these patients. In addition, some patients could be intolerant of therapy with TKIs or could develop mutations that promote resistance to TKI therapy. In these patients, ABL001 could provide a novel therapeutic option.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Asciminib in CML patients | Experimental | Drug: Asciminib (ABL001)Dose escalation study estimated the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of asciminib in adult patients with chronic myeloid leukemia (CML). |
| Asciminib+Nilotinib in CML patients | Experimental | Drug: Asciminib (ABL001), Drug: NilotinibDose escalation study estimated the MTD and/or RDE of asciminib in combination with Nilotinib in adult CML patients |
| Asciminib in Ph+ ALL patients | Experimental | Drug: Asciminib (ABL001)Dose escalation study estimated the MTD and/or RDE of asciminib in adult patients with Ph positive ALL patients |
| Asciminib+Imatinib in CML patients | Experimental | Drug: Asciminib (ABL001), Drug: ImatinibDose escalation study to estimate the MTD and/or RDE of asciminib in combination with imatinib in adult CML patients |
| Asciminib+dasatinib in CML patients | Experimental | Drug: Asciminib (ABL001), Drug: DasatinibDose escalation study estimated the MTD and/or RDE of asciminib in combination with dasatinib in adult CML patients |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- a. Patients with Ph+ CML in chronic or accelerated phase who were previously treated with at least two different tyrosine kinase inhibitors prior to study entry and are relapsed, refractory to or intolerant of TKIs as determined by investigators or
- b. Patients with CML in chronic or accelerated phase who exhibit relapsed disease associated with the presence of the T315I "gatekeeper mutation" after at least one TKI are also eligible provided that no other effective therapy exists
- Patients with CML BP or Ph+ ALL who have a cytopathologically confirmed diagnosis and are relapsed or refractory to at least one prior TKI or intolerant of TKIs. TKI failure for Ph+ ALL and CML-BP patients is defined as at least the loss of Molecular Response (MR) 4.5 (BCR-ABL ≤ 0.0032%)
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Willingness and ability to comply with all study procedures
Key exclusion criteria (8 listed)
- Systemic antineoplastic therapy (including cytotoxic chemotherapy, alfa-interferon and toxin immunoconjugates) or any experimental therapy within 14 days or 5 half-lives, whichever is shorter, before the first dose of study treatment
- Therapy with TKIs as single agent within 5 half-lives before the first dose of study treatment
- Unconjugated monoclonal antibody therapies within 28 days or 5 half-lives, whichever is shorter, before the first dose of study treatment
- For patients receiving ABL001 in combination with either nilotinib or imatinib or dasatinib, intolerance to nilotinib, imatinib or dasatinib, respectively
- Radiotherapy with a wide field of radiation within 4 weeks or radiotherapy with a limited field of radiation for palliation within 1 week of the first dose of study treatment.
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 |
|---|---|---|
| Asciminib (ABL001)Also: ABL001Asciminib was be administered orally in a dose escalation schedule. | Drug | Asciminib in CML patients, Asciminib in Ph+ ALL patients, Asciminib+Imatinib in CML patients, Asciminib+Nilotinib in CML patients, Asciminib+dasatinib in CML patients |
| NilotinibAsciminib and Nilotinib was administered orally in CML patients | Drug | Asciminib+Nilotinib in CML patients |
| ImatinibAsciminib and imatinib was administered orally in CML patients | Drug | Asciminib+Imatinib in CML patients |
| DasatinibAsciminib and dasatinib was administered orally in CML patients | Drug | Asciminib+dasatinib in CML patients |
Primary outcome
Incidence of dose limiting toxicities (DLTs) during the first cycle of study treatment
Time frame: First Cycle is 28 days
Determine the MTD and/or RDE of ABL001 as single agent in CML and Ph+ ALL, and in combination with either nilotinib or imatinib or dasatinib in CML patients
10 secondary outcomes
Hematologic Response
Time frame: At screening and first day of cycle 2 and 3 and every 12 weeks afterwards
Cytogenetic response
Time frame: at screening or when a patient's BCR-ABL ratio has risen to >1%
BCR-ABL transcript level
Time frame: At screening and first day of cycle 2 and 3 and every 12 weeks afterwards
Cmax of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
Cmin of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUCinf of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUClast of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
AUCtau of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
T1/2 of ABL001 as measured in plasma
Time frame: Cycle 1 days 1,2,8,15,16 and 22. Cycle 2 days 1 and 2, and subsequent cycles at the begining of each cycle up to cycle 6.
Adverse events
Time frame: Collected from screening visit through post-treatment follow-up period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2024.
- Derived from this study: PubMed 39374521Luskin MR, Murakami MA, Keating J, Flamand Y, Winer ES, Garcia JS, Stahl M, Stone RM, Wadleigh M, Jaeckle SL, Hagopian E, Weinstock DM, Liegel J, McMasters M, Wang ES, Stock W, DeAngelo DJ. Asciminib plus dasatinib and prednisone for Philadelphia chromosome-positive acute leukemia. Blood. 2025 Feb 6;145(6):577-589. doi: 10.1182/blood.2024025800.
- Derived from this study: PubMed 38755421Cortes JE, Sasaki K, Kim DW, Hughes TP, Etienne G, Mauro MJ, Hochhaus A, Lang F, Heinrich MC, Breccia M, Deininger M, Goh YT, Janssen JJWM, Talpaz M, de Soria VGG, le Coutre P, DeAngelo DJ, Damon A, Cacciatore S, Polydoros F, Agrawal N, Rea D. Asciminib monotherapy in patients with chronic-phase chronic myeloid leukemia with the T315I mutation after >/=1 prior tyrosine kinase inhibitor: 2-year follow-up results. Leukemia. 2024 Jul;38(7):1522-1533. doi: 10.1038/s41375-024-02278-8. Epub 2024 May 16.
- 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 31826340Hughes TP, Mauro MJ, Cortes JE, Minami H, Rea D, DeAngelo DJ, Breccia M, Goh YT, Talpaz M, Hochhaus A, le Coutre P, Ottmann O, Heinrich MC, Steegmann JL, Deininger MWN, Janssen JJWM, Mahon FX, Minami Y, Yeung D, Ross DM, Tallman MS, Park JH, Druker BJ, Hynds D, Duan Y, Meille C, Hourcade-Potelleret F, Vanasse KG, Lang F, Kim DW. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019 Dec 12;381(24):2315-2326. doi: 10.1056/NEJMoa1902328.
- Novartis results databaseLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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
18 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Asciminib (ABL001), Nilotinib and Imatinib in people with Chronic Myelogenous Leukemia and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals at 18 sites in 10 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 8 inclusion and 8 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 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Germany, France, Australia, Italy and 5 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of dose limiting toxicities (DLTs) during the first cycle of study treatment (time frame: First Cycle is 28 days). 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 NCT02081378, which also lists 4 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 Mar 2024.