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

NCT04795427 | Completed Phase 2

• 1 other identifier: CABL001A2202
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 20

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

Conditions

Leukemia, Chronic Myelogenous

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

Outcome Measures

Primary Outcomes (1)

• Major molecular response rate of asciminib

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

  week 24

Secondary Outcomes (13)

• Cytogenetic response (CyR) rate

  24, 48, 96 weeks

• Major molecular response rate of best available treatment arm

  week 24

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

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

• major molecular response rate by all scheduled data collection time points

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

• Time to major molecular response rate

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

Study Arms (2)

asciminb arm

EXPERIMENTAL

Patients will receive asciminib (40 mg BID continuous)

Drug: asciminib

best available treatment arm

EXPERIMENTAL

Patients will receive best available therapy chosen by investigator

Other: best available treatment

Interventions

asciminib DRUG

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

Also known as: ABL001

asciminb arm

best available treatment 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

best available treatment arm

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (20)

Novartis Investigative Site

Chongqing, Chongqing Municipality, 400010, China

Location

Novartis Investigative Site

Guangzhou, Guangdong, 510515, China

Location

Novartis Investigative Site

Shenzhen, Guangdong, 518037, China

Location

Novartis Investigative Site

Zhengzhou, Henan, 450008, China

Location

Novartis Investigative Site

Zhengzhou, Henan, 450052, China

Location

Novartis Investigative Site

Wuhan, Hubei, 430022, China

Location

Novartis Investigative Site

Nanjing, Jiangsu, 210000, China

Location

Novartis Investigative Site

Nantong, Jiangsu, 226000, China

Location

Novartis Investigative Site

Suzhou, Jiangsu, 215004, China

Location

Novartis Investigative Site

Nanchang, Jiangxi, 330006, China

Location

Novartis Investigative Site

Changchun, Jilin, 130021, China

Location

Novartis Investigative Site

Xian, Shanxi, 710004, China

Location

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

Location

Novartis Investigative Site

Hangzhou, Zhejiang, 310003, China

Location

Novartis Investigative Site

Wenzhou, Zhejiang, 325000, China

Location

Novartis Investigative Site

Beijing, 100044, China

Location

Novartis Investigative Site

Beijing, 100730, China

Location

Novartis Investigative Site

Shanghai, 200025, China

Location

Novartis Investigative Site

Shenyang, 110004, China

Location

Novartis Investigative Site

Tianjin, 300020, China

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Bronchiolitis Obliterans Syndrome

Interventions

asciminib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2021
• First Posted: March 12, 2021
• Study Start: December 6, 2021
• Primary Completion: May 29, 2023
• Study Completion: November 18, 2024
• Last Updated: April 18, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share

Locations

CN (20)