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Asciminib Treatment Optimization in ≥ 3rd Line CML-CP

NCT ID: NCT04948333

Last Updated: 2025-12-23

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

199 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-13

Study Completion Date

2026-02-03

Brief Summary

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The purpose of the study is to optimize the treatment of asciminib in patients with chronic myelogenous leukemia in chronic phase (CML-CP) previously treated with 2 or more Tyrosine Kinase Inhibitors (TKIs).

Detailed Description

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This study consists of a screening period of up to 28 days, a treatment period of 144 weeks and a post-treatment safety follow-up period of 4 weeks.

Patients will receive asciminib as study treatment continuously for up to 144 weeks or until disease progression, treatment failure or intolerance to treatment. At treatment initiation, asciminib will be provided to all trial patients at a total daily dose of 80 mg. All patients will be randomly assigned 1:1 to 2 groups with 80 mg given either as 40 mg b.i.d. or 80 mg q.d., using IRT to avoid any selection bias.

In patients not achieving Major Molecular Responses (MMR) at 48 weeks or losing the response after the week 48 assessment up to week 108, asciminib dose may be escalated to 200 mg q.d. if in the investigator's opinion the patient may benefit from the escalation. In addition, there must not be any grade 3 or 4 toxicity while on therapy, or persistent grade 2 toxicity, possibly related to asciminib and unresponsive to optimal management.

The trial will enroll a total of approximately 186 patients:

* 156 patients with CML-CP not in MMR at baseline who were treated with two or more TKIs and who were either resistant (ELN 2020 warning or failure) or intolerant to the last treatment will be enrolled. For this population, the primary endpoint for MMR at 48 weeks will be assessed.
* Up to 30 additional patients intolerant only to their last TKI treatment and in MMR at baseline will also be enrolled. This patient population will not be part of primary endpoint analysis; however, all assessments will be done as with the 156 patients from the population of the primary endpoint analysis.

Conditions

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Chronic Myelogenous Leukemia

Keywords

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ABL001 Phase 3 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 Treatment optimization Asciminib European Leukemia Network (ELN) Recommendations 2020 Major Molecular Response (MMR) rate at 48 weeks

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ABL001

Participants will be treated with 80 mg of ABL001 (40 mg BID or 80mg QD). In patients not achieving MMR at 48 weeks or losing the response after the week 48 assessment up to week 108, asciminib dose may be escalated to 200 mg q.d. if in the investigator's opinion the patient may benefit from the escalation.

Group Type EXPERIMENTAL

ABL001 40mg BID

Intervention Type DRUG

One tablet of 40 mg will be taken orally twice a day (BID)

ABL001 80mg QD

Intervention Type DRUG

Two tablets of 40 mg will be taken orally once a day (QD)

ABL001 200mg QD

Intervention Type DRUG

Five tablets of 40 mg will be taken orally once a day (QD)

Interventions

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ABL001 40mg BID

One tablet of 40 mg will be taken orally twice a day (BID)

Intervention Type DRUG

ABL001 80mg QD

Two tablets of 40 mg will be taken orally once a day (QD)

Intervention Type DRUG

ABL001 200mg QD

Five tablets of 40 mg will be taken orally once a day (QD)

Intervention Type DRUG

Other Intervention Names

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asciminib asciminib asciminib

Eligibility Criteria

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Inclusion Criteria

* Signed informed consent must be obtained prior to participation in the study
* Male or female patients with a diagnosis of CML-CP ≥ 18 years of age
* Treatment with a minimum of 2 or more prior TKIs (i.e. imatinib, nilotinib, dasatinib, bosutinib, radotinib or ponatinib)
* Warning or failure (adapted from the 2020 ELN Recommendations) or intolerance to the most recent TKI therapy at the time of screening
* Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
* Adequate end organ function (as per central laboratory tests)

Exclusion Criteria

* Known presence of the BCR::ABL1 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
* Patient planning to undergo allogeneic hematopoietic stem cell transplantation
* Uncontrolled cardiac repolarization abnormality
* 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
* History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
* Testing for Hepatitis B surface antigen (HbsAg) and Hepatitis B core antibody (HBcAb / anti HBc) will be performed at screening. Patients with active Hepatitis B Virus (HBV) infection (hepatitis B surface antigen \[HbsAg\] positive) will be excluded
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 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

CABA, Buenos Aires, Argentina

Site Status

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Buenos Aires, , Argentina

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Graz, , Austria

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Linz, , Austria

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Vienna, , Austria

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Rio de Janeiro, Rio de Janeiro, Brazil

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São Paulo, , Brazil

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Vancouver, British Columbia, Canada

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Toronto, Ontario, Canada

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Bordeaux, , France

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Lyon, , France

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Montpellier, , France

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Nantes, , France

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Paris, , France

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Mannheim, Baden-Wurttemberg, Germany

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Frankfurt am Main, Hesse, Germany

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Jena, Thuringia, Germany

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Berlin, , Germany

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Kiel, , Germany

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München, , Germany

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Athens, , Greece

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Thessaloniki, , Greece

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Monza, MB, Italy

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Roma, RM, Italy

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Verona, VR, Italy

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George Town, Pulau Pinang, Malaysia

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Kota Kinabalu, Sabah, Malaysia

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Johor Bahru, , Malaysia

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Kuala Selangor, , Malaysia

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Khoudh, , Oman

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Katowice, , Poland

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Warsaw, , Poland

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Singapore, , Singapore

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Singapore, , Singapore

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Seoul, Korea, South Korea

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Seoul, , South Korea

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Taegu, , South Korea

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Santiago Compostela, A Coruna, Spain

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Bilbao, Bizkaia, Spain

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Barcelona, Catalonia, Spain

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Santa Cruz, Santa Cruz De Tenerife, Spain

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Madrid, , Spain

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Leeds, West Yorkshire, United Kingdom

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Cambridge, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Hanoi, , Vietnam

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Novartis Investigative Site

Ho Chi Minh City, , Vietnam

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Countries

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Russia Argentina Austria Brazil Canada France Germany Greece Italy Malaysia Oman Poland Singapore South Korea Spain United Kingdom Vietnam

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2024-511381-36-00

Identifier Type: CTIS

Identifier Source: secondary_id

CABL001A2302

Identifier Type: -

Identifier Source: org_study_id