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

NCT ID: NCT04795427

Last Updated: 2025-04-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-06

Study Completion Date

2024-11-18

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Leukemia, Chronic Myelogenous

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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asciminb arm

Patients will receive asciminib (40 mg BID continuous)

Group Type EXPERIMENTAL

asciminib

Intervention Type DRUG

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

best available treatment arm

Patients will receive best available therapy chosen by investigator

Group Type EXPERIMENTAL

best available treatment

Intervention Type OTHER

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

Interventions

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asciminib

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

Intervention Type DRUG

best available treatment

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

Intervention Type OTHER

Other Intervention Names

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ABL001

Eligibility Criteria

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Inclusion Criteria

Diagnosed as CML-CP:

1. 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
2. Prior treatment with a minimum of 2 prior ATP-competitive TKIs.
3. Failure (adapted from the 2013 European Leukemia Net (ELN) Guidelines) or intolerance to the most recent TKI therapy at the time of screening.
4. Evidence of typical BCR-ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification

Exclusion Criteria

1. Known presence of the T315I mutation at any time prior to study entry
2. Known second chronic phase of CML after previous progression to AP/BC
3. Previous treatment with a hematopoietic stem cell transplantation
4. Participants planning to undergo allogeneic hematopoietic stem cell transplantation
5. 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)

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
6. History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Chongqing, Chongqing Municipality, China

Site Status

Novartis Investigative Site

Guangzhou, Guangdong, China

Site Status

Novartis Investigative Site

Shenzhen, Guangdong, China

Site Status

Novartis Investigative Site

Zhengzhou, Henan, China

Site Status

Novartis Investigative Site

Zhengzhou, Henan, China

Site Status

Novartis Investigative Site

Wuhan, Hubei, China

Site Status

Novartis Investigative Site

Nanjing, Jiangsu, China

Site Status

Novartis Investigative Site

Nantong, Jiangsu, China

Site Status

Novartis Investigative Site

Suzhou, Jiangsu, China

Site Status

Novartis Investigative Site

Nanchang, Jiangxi, China

Site Status

Novartis Investigative Site

Changchun, Jilin, China

Site Status

Novartis Investigative Site

Xian, Shanxi, China

Site Status

Novartis Investigative Site

Chengdu, Sichuan, China

Site Status

Novartis Investigative Site

Hangzhou, Zhejiang, China

Site Status

Novartis Investigative Site

Wenzhou, Zhejiang, China

Site Status

Novartis Investigative Site

Beijing, , China

Site Status

Novartis Investigative Site

Beijing, , China

Site Status

Novartis Investigative Site

Shanghai, , China

Site Status

Novartis Investigative Site

Shenyang, , China

Site Status

Novartis Investigative Site

Tianjin, , China

Site Status

Countries

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China

Other Identifiers

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CABL001A2202

Identifier Type: -

Identifier Source: org_study_id

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