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Leukemia, Chronic MyelogenousPhase 2NCT04795427

Study of Efficacy and Safety of CML-CP Patients Treated With Asciminib Versus Best Available Therapy, Previously Treated With 2 or More Tyrosine Kinase Inhibitors

A Phase 2 study testing asciminib and best available treatment in people with Leukemia, Chronic Myelogenous, run by Novartis Pharmaceuticals at 20 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this Chinese bridging study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of asciminib versus best available therapy in Chinese patients with Chronic Myelogenous Leukemia in chronic phase, previously treated with 2 or more tyrosine kinase inhibitors to support related indication… Read more about this study

Who is it for?
Ages 18 to 100 years
Conditions the study is for
Leukemia, Chronic Myelogenous
What is being tested?
asciminib and best available treatment
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
84 participants
Where
20 sitesChina
Study dates
6 Dec 2021to 18 Nov 2024
Phase
Phase 2
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 to 100 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosed as CML-CP:
  • Participants 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 10^9/ L (≥ 50,000/mm3) platelets 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
  • Prior treatment with a minimum of 2 prior ATP-competitive TKIs.
  • Failure (adapted from the 2013 European Leukemia Net (ELN) Guidelines) or intolerance to the most recent TKI therapy at the time of screening.
  • Evidence of typical BCR-ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification

Who cannot join

  • Known presence of the T315I 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
  • Participants 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, angina pectoris, coronary artery bypass graft Clinically significant cardiac arrhythmias , complete left bundle branch block, high-grade AV block QTcF at screening ≥450 msec (male participants), ≥460 msec (female participants)
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  • 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 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" that cannot be discontinued or replaced 7 days prior to starting study drug by safe alternative medication.
  • Inability to determine the QTcF interval
  • History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
  • Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

20 sites in 1 country.

20 sites in 1 country

China20 sites

  • Novartis Investigative Site

    Beijing, 100044

  • Novartis Investigative Site

    Beijing, 100730

  • Novartis Investigative Site

    Changchun, Jilin, 130021

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041

  • Novartis Investigative Site

    Chongqing, Chongqing Municipality, 400010

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510515

  • Novartis Investigative Site

    Hangzhou, Zhejiang, 310003

  • Novartis Investigative Site

    Nanchang, Jiangxi, 330006

  • Novartis Investigative Site

    Nanjing, Jiangsu, 210000

  • Novartis Investigative Site

    Nantong, Jiangsu, 226000

  • Novartis Investigative Site

    Shanghai, 200025

  • Novartis Investigative Site

    Shenyang, 110004

  • Novartis Investigative Site

    Shenzhen, Guangdong, 518037

  • Novartis Investigative Site

    Suzhou, Jiangsu, 215004

  • Novartis Investigative Site

    Tianjin, 300020

  • Novartis Investigative Site

    Wenzhou, Zhejiang, 325000

  • Novartis Investigative Site

    Wuhan, Hubei, 430022

  • Novartis Investigative Site

    Xian, Shanxi, 710004

  • Novartis Investigative Site

    Zhengzhou, Henan, 450008

  • Novartis Investigative Site

    Zhengzhou, Henan, 450052

Other trials in China

About this study

Official title: A Randomized, Open-label, Multi-center, Phase II Study of Asciminib Versus Best Available Therapy in Chinese Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors

The purpose of this Chinese bridging study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of asciminib versus best available therapy in Chinese patients with Chronic Myelogenous Leukemia in chronic phase, previously treated with 2 or more tyrosine kinase inhibitors to support related indication registration in China.

The primary objective of the study is to evaluate the Major Molecular Response (MMR) rate of asciminib treatment at 24 weeks.

Read the detailed description

The purpose of this Chinese bridging study is to evaluate the efficacy, safety, tolerability and pharmacokinetics (PK) of asciminib versus best available therapy (BAT) in Chinese patients with Chronic Myelogenous Leukemia in chronic phase (CML-CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs) to support related indication registration in China.

This study will enroll the participants 1) who failed their most recent TKI therapy by meeting the definition of treatment failure as per the 2013 European Leukemia Net (ELN) guidelines, or 2) who were intolerant to the most recent TKI therapy and must have BCR-ABL1 ratio > 0.1% IS at screening.

Eligible participants will be randomized into asciminib arm or the BAT arm on a 2:1 ratio, to receive asciminib treatment (continuous 40 mg BID) or BAT from Day 1 until the end of study treatment period defined as 96 weeks after the last participant receives the first dose.

Being tested
asciminib, best available treatment
Keywords
Chronic Myelogenous Leukemia in chronic phase, Chronic Myelogenous Leukemia, CML-CP, Chronic, Chronic Phase, ABL001, asciminib, prior treatment with 2 or more tyrosine kinase inhibitors (TKIs), major molecular response, MMR, Chinese patients, phase ll, open label, best available therapy, BAT
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Bronchiolitis Obliterans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • asciminibDrug

    Also called ABL001

    Asciminib comes in 20 mg and 40 mg tablets and is taken orally twice daily

  • best available treatmentOther

    Best available treatment will be based on investigator's choice identified prior to randomization. Dose and frequency will depend on label and institutional guidelines for various BAT

Study groups in NCT04795427
Study groupRoleWhat they receive
asciminb armExperimentalPatients will receive asciminib (40 mg BID continuous)
Receives: Drug: asciminib
best available treatment armExperimentalPatients will receive best available therapy chosen by investigator
Receives: Other: best available treatment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Major molecular response rate of asciminib

    Evaluate the major molecular response rate at 24 weeks in asciminib arm

    Measured: week 24

Secondary outcomes (13)

  1. Cytogenetic response (CyR) rate

    Evaluate the cytogenetic response rate (Complete, Partial, Major, Minor, Minimal, no response) at and by all scheduled data collection time points including 24, 48 and 96 weeks for both asciminib arm and best available treatment arm.

    Measured: 24, 48, 96 weeks

  2. Major molecular response rate of best available treatment arm

    Evaluate the major molecular response rate at week 24 of best available treatment arm, and compare with asciminib arm

    Measured: week 24

  3. Major molecular response rate of both asciminib arm and BAT armn time points

    Evaluate the major molecular response rate, collected at all scheduled data collection time point (except week 24)

    Measured: Up to all participants received at least 96 weeks of randomized study treatment, except week 24

  4. major molecular response rate by all scheduled data collection time points

    Evaluate the major molecular rate by all scheduled data collection time points including 24, 48 and 96 weeks by treatment group

    Measured: Up to all participants received at least 96 weeks of randomized study treatment

  5. Time to major molecular response rate

    Evaluate the time from the date of the first dose of study drug to the date of the first documented MMR by treatment group

    Measured: Up to all participants received at least 96 weeks of randomized study treatment

  6. Duration of major molecular response

    First document major molecular response to loss of MMR up to 96 weeks after last participant receive the first dose

    Measured: Up to all participants received at least 96 weeks of randomized study treatment

  7. Overall survival

    To evaluate the time from the date of randomization to the date of death (including the survival follow-up period)

    Measured: Up to all participants received at least 96 weeks of randomized study treatment, plus 30 days for safety follow up

  8. Progression free survival

    Evaluate 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)

    Measured: Up to all participants received at least 96 weeks of randomized study treatment, plus 30 days for safety follow up

  9. Pharmacokinetics (PK) parameter of asciminib: Cmax

    Characterize PK of asciminib in the Chinese CML-CP population. Cmax is the maximum (peak) observed plasma drug concentration after dose administration (ng/mL).

    Measured: Week 2 Day 1 (W2D1)

  10. PK parameter of asciminib: Tmax

    Characterize PK of asciminib in the Chinese CML-CP population. Tmax is the time to reach maximum (peak) plasma drug concentration after dose administration (hr).

    Measured: Week 2 Day 1 (W2D1)

  11. PK parameter of asciminib: Ctrough

    Characterize PK of asciminib in the Chinese CML-CP population. Trough plasma concentrations (Ctrough)

    Measured: Week 2 Day 1 (W2D1)

  12. PK parameter of asciminib: AUCtau

    Characterize PK of asciminib in the Chinese CML-CP population. The partial area under the plasma concentration-time curve from dose time to tau (ng*hr/mL). For a bid regimen, Tau=12h.

    Measured: Week 2 Day 1 (W2D1)

  13. PK parameter of asciminib: AUClast

    Characterize PK of asciminib in the Chinese CML-CP population. AUClast is the The AUC from the time of dosing to the time of the last measurable plasma concentration (Tlast) (ng*hr/mL)

    Measured: Week 2 Day 1 (W2D1)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Mar 2021
  2. Study start6 Dec 2021
  3. Primary completion29 May 2023
  4. Study completion18 Nov 2024

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
84 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04795427
Sponsor's protocol ID
CABL001A2202
Status last verified
Apr 2025
First submitted
10 Mar 2021
Last updated
18 Apr 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
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 review 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 availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing asciminib and best available treatment in people with Leukemia, Chronic Myelogenous, run by Novartis Pharmaceuticals at 20 sites in China.

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 7 inclusion and 11 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 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in China. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Major molecular response rate of asciminib (time frame: week 24). 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 NCT04795427.

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 Apr 2025.

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