NCT03578367

Brief Summary

To evaluate efficacy, safety and pharmacokinetic profile of asciminib 40mg+imatinib or asciminib 60mg+imatinib versus continued imatinib and versus nilotinib in pre-treated patients with Chronic Myeloid Leukemia in chronic phase (CML-CP). An asciminib single agent arm (80 mg daily) was added after the primary analysis to evaluate if asciminib alone could lead to MR4.5 patients in Imatinib for at least one year who have never achieved deep molecular response (DMR).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
104

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Nov 2018

Longer than P75 for phase_2

Geographic Reach
15 countries

30 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 15, 2018

Completed
21 days until next milestone

First Posted

Study publicly available on registry

July 6, 2018

Completed
5 months until next milestone

Study Start

First participant enrolled

November 22, 2018

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 8, 2021

Completed
3.3 years until next milestone

Results Posted

Study results publicly available

February 14, 2025

Completed
12 days until next milestone

Study Completion

Last participant's last visit for all outcomes

February 26, 2025

Completed
Last Updated

April 21, 2026

Status Verified

April 1, 2026

Enrollment Period

3 years

First QC Date

June 15, 2018

Results QC Date

October 4, 2024

Last Update Submit

April 17, 2026

Conditions

Keywords

CMLChronic Myelogenous Leukemialeukemia, myeloid chronicHematologic DiseasesAsciminibABL001ImatinibNilotinibdeep molecular responseDMRPh+ CMLchronic phasecancer of the white blood cellstyrosine kinase inhibitorleukemia, myeloidleukemiaCML with Ph+

Outcome Measures

Primary Outcomes (1)

  • Molecular Response (MR)^4.5 Rate at 48 Weeks and Difference in Rate Between Asciminib + Imatinib and Imatinib Alone

    Percentage of participants still treated with the randomized treatment at 48 weeks and are in MR\^4.5 (BCR::ABL1 ratio of ≤ 0.0032%) at 48 weeks (± assessment window), among all participants in the asciminib add-on arms vs imatinib arm.

    at Week 48

Secondary Outcomes (20)

  • Rate of MR^4.5 at 48 Weeks (Asciminib add-on Arms vs Nilotinib)

    at Week 48

  • Rate of MR^4.5 by 48 Weeks (Randomized Arms)

    by 48 weeks

  • Rate of MR^4.5 at 96 Weeks (Randomized Arms) and Difference in Rate Between Asciminib + Imatinib and Nilotinib Alone

    at Week 96

  • Rate of MR^4.5 by 96 Weeks (Randomized Arms)

    by 96 weeks

  • Sustained MR^4.5 From at 96 Weeks (Randomized Arms)

    at 96 weeks

  • +15 more secondary outcomes

Study Arms (5)

Asciminib 60mg QD + Imatinib 400mg QD

EXPERIMENTAL

Asciminib 60 mg taken once daily in combination with Imatinib 400 mg taken once daily

Drug: Asciminib add-onDrug: Imatinib

Asciminib 40mg QD + Imatinib 400mg QD

EXPERIMENTAL

Asciminib 40 mg taken once daily in combination with Imatinib 400 mg taken once daily

Drug: Asciminib add-onDrug: Imatinib

Imatinib 400mg QD

ACTIVE COMPARATOR

Imatinib 400 mg taken once daily

Drug: Imatinib

Nilotinib 300mg BID

ACTIVE COMPARATOR

Nilotinib 300 mg taken twice daily

Drug: Nilotinib

Asciminib 80mg QD (ASAC)

EXPERIMENTAL

Asciminib 80 mg taken once daily

Drug: Asciminib 80mg QD (asciminib single agent (ASAC))

Interventions

Asciminib was supplied as 40 mg and 20 mg tablets and taken orally once daily.

Also known as: ABL001 (asciminib)
Asciminib 40mg QD + Imatinib 400mg QDAsciminib 60mg QD + Imatinib 400mg QD

Imatinib was supplied as 400 mg and 100 mg tablets and taken orally once daily.

Also known as: STI571
Asciminib 40mg QD + Imatinib 400mg QDAsciminib 60mg QD + Imatinib 400mg QDImatinib 400mg QD

Nilotinib was supplied as 150 mg and 200 mg hard gelatin capsules and taken orally twice daily.

Also known as: AMN107
Nilotinib 300mg BID

Asciminib was supplied as 40 mg and 20 mg tablets and taken orally once daily (in the fasted state) on a continuous schedule (QD).

Also known as: ABL001
Asciminib 80mg QD (ASAC)

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female patients ≥ 18 years of age with a confirmed diagnosis of CML-CP.
  • Minimum of one year (12 calendar months) treatment with imatinib first line for CML-CP (patients have to be on imatinib 400 mg QD at randomization and had no dose change in the past three months).
  • For Korea only:
  • (i) a minimum of one year (12 calendar months) of prior treatment with imatinib for patients with BCR::ABL1 levels \> 0.1%, ≤ 1% IS at the time of randomization.
  • (ii) a minimum of two years (24 calendar months) of prior treatment with imatinib for patients with BCR::ABL 1 levels \> 0.01%, ≤ 0.1% IS at the time of randomization.
  • BCR::ABL1 levels \> 0.01% IS (International Scale) and ≤ 1% IS at the time of randomization as confirmed with a central assessment at screening; patients must not have achieved deep molecular response (MR4 IS) confirmed by 2 consecutive tests at any time during prior imatinib treatment. An isolated, single test result with BCR::ABL1 levels \< 0.01 % (MR4 IS) is allowed, however, it should not have been observed within the 9 months prior to randomization
  • Patient must meet the following laboratory values before randomization:
  • Absolute Neutrophil Count ≥ 1.5 x 10E9/L
  • Platelets ≥ 75 x 10E9/L
  • Hemoglobin ≥ 9 g/dL
  • Serum creatinine \< 1.5 mg/dL
  • Total bilirubin ≤ 1.5 x ULN (Upper Limit of Normal) except for patients with Gilbert's syndrome who may only be included with total bilirubin ≤ 3.0 x ULN
  • Aspartate transaminase (AST) ≤ 3.0 x ULN
  • Alanine transaminase (ALT) ≤ 3.0 x ULN
  • Alkaline phosphatase ≤ 2.5 x ULN
  • +2 more criteria

You may not qualify if:

  • Treatment failure according to European Leukemia Network (ELN) criteria 2013 during imatinib treatment.
  • Known second chronic phase of CML after previous progression to Accelerated Phase (AP)/Blast Crisis (BC).
  • Previous treatment with any tyrosine kinase inhibitors (TKIs) other than imatinib.
  • History or current diagnosis of ECG abnormalities indicating significant risk or safety for participants participating in the study such as:
  • History of myocardial infarction, angina pectoris, coronary artery bypass graft within 6 months prior to randomization
  • Concomitant clinically significant arrhythmias
  • Resting QTcF ≥ 450 msec (male) or ≥ 460 msec (female) prior to randomization
  • Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
  • Risk factors for Torsades de Pointes
  • Concomitant medications with a "known" risk of Torsades de Pointes
  • inability to determine the QTcF interval 5. 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, uncontrolled clinically significant hyperlipidemia and high serum amylase) 6. History of acute pancreatitis within 1 year prior to randomization or medical history of chronic pancreatitis; on-going acute liver disease or history of chronic liver disease 7. History of other active malignancy within 3 years prior to randomization with the exception of basal cell skin cancer, indolent prostate cancer and carcinoma in situ treated curatively.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (30)

Georgia Regents University

Augusta, Georgia, 30912, United States

Location

Sidney Kimmel Comprehensive Cancer Center

Baltimore, Maryland, 21205, United States

Location

Novartis Investigative Site

Vienna, 1140, Austria

Location

Novartis Investigative Site

Montreal, Quebec, H1T 2M4, Canada

Location

Novartis Investigative Site

Brno, 625 00, Czechia

Location

Novartis Investigative Site

Copenhagen, DK-2100, Denmark

Location

Novartis Investigative Site

Bordeaux, 33076, France

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Milan, MI, 20162, Italy

Location

Novartis Investigative Site

Roma, RM, 00161, Italy

Location

Novartis Investigative Site

Krakow, 31 531, Poland

Location

Novartis Investigative Site

Warsaw, 00-791, Poland

Location

Novartis Investigative Site

Wroclaw, 50 367, Poland

Location

Novartis Investigative Site

Lisbon, 1099-023, Portugal

Location

Novartis Investigative Site

Porto, 4200-072, Portugal

Location

Novartis Investigative Site

Moscow, 125167, Russia

Location

Novartis Investigative Site

Moscow, 125284, Russia

Location

Novartis Investigative Site

Saint Petersburg, 191024, Russia

Location

Novartis Investigative Site

Saint Petersburg, 197341, Russia

Location

Novartis Investigative Site

Uijeongbu-si, Gyeonggi-do, 11759, South Korea

Location

Novartis Investigative Site

Seoul, 06591, South Korea

Location

Novartis Investigative Site

Badalona, Barcelona, 08916, Spain

Location

Novartis Investigative Site

Madrid, 28034, Spain

Location

Novartis Investigative Site

Seville, 41009, Spain

Location

Novartis Investigative Site

Valencia, 46026, Spain

Location

Novartis Investigative Site

Changhua, 50006, Taiwan

Location

Novartis Investigative Site

Taoyuan District, 33305, Taiwan

Location

Novartis Investigative Site

Liverpool, CH63 4JY, United Kingdom

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

Novartis Investigative Site

Oxford, OX3 7LE, United Kingdom

Location

Related Publications (1)

  • Hughes TP, Saglio G, Geissler J, Kim DW, Lomaia E, Mayer J, Turkina A, Kapoor S, Cardoso AP, Nieman B, Quenet S, Cortes JE. Asciminib add-on to imatinib demonstrates sustained high rates of ongoing therapy and deep molecular responses with prolonged follow-up in the ASC4MORE study. J Hematol Oncol. 2024 Dec 18;17(1):125. doi: 10.1186/s13045-024-01642-6.

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL PositiveHematologic DiseasesLeukemia, MyeloidLeukemia

Interventions

asciminibImatinib Mesylatenilotinib

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsMyeloproliferative DisordersBone Marrow DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPyrimidines

Limitations and Caveats

The use of zero (0) in the post-treatment period indicates adverse events were not collected.

Results Point of Contact

Title
Clinical Disclosure Office
Organization
Novartis Pharmaceuticals

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Model Details: Treatment arms 1 - 4 randomized (84 participants) and asciminib single agent cohort, not randomized (20 participants)
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 15, 2018

First Posted

July 6, 2018

Study Start

November 22, 2018

Primary Completion

November 8, 2021

Study Completion

February 26, 2025

Last Updated

April 21, 2026

Results First Posted

February 14, 2025

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

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.

More information

Locations