CML Advocates Network

Study title

AIM4CML (CABL001AUS04) = Asciminib monotherapy for patients with CML-CP with and without T315I mutation

Scientific title

An Open Label, Multi-center Phase IIIb Study of Asciminib (ABL001) Monotherapy in Previously Treated Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) With and Without T315I Mutation (ClinicalTrials.gov NCT04666259)

Indication and most important inclusion criteria

This study includes male or female patients 18 years and older with CML in chronic phase (CML-CP) who meet laboratory values specified in the study protocol.

Eligible patients must have had mutation analysis testing done 6 months before study entry. Patients without the T315I mutation will have received 2 tyrosine kinase inhibitors (TKIs) and patients with the T315I mutation will have received 1TKI before study entry.

Eligible patients must also have adequate end-organ function, and an Eastern Co-Operative Oncology Group (ECOG) status of 0, 1 or 2.

Additional criteria may apply.

Short description of intervention

This is a study of the safety and efficacy asciminib in CML-CP patients without T315I mutation who have had at least 2 prior TKIs and CML-CP patients with the T315I mutation who have had at least 1 prior TKI.

The study consists of three periods: screening and baseline for up to 21 days, treatment with asciminib tablets for up to 72 weeks and a safety follow up period for 30 days.

Patients with CML-CP without T315I mutation will be randomly assigned to either group A or B. Patients with the T315I mutation will be enrolled in group C. During the treatment period, group A will receive aciminib 40 mg twice a day, group B will receive 80 mg once a day and group C will receive 200 mg twice a day.

Type of study

Therapy optimization trial

Current status

Not yet recruiting
First patients are likely to be enrolled in March 2021.

Study sponsor

Novartis Pharmaceuticals

Scientific lead / contact

Novartis Pharmaceuticals

Principal investigator

Novartis Pharmaceuticals

Additional information

Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health 

Study centers / principal investigators

Around 40 sites are likely to be involved across the US.