NCT03578367

Study of Efficacy and Safety of Asciminib in Combination With Imatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP)

A Phase 2, Multi-center, Open-label, Randomized Study of Oral Asciminib Added to Imatinib Versus Continued Imatinib Versus Switch to Nilotinib in Patients With CML-CP Who Have Been Previously Treated With Imatinib and Have Not Achieved Deep Molecular Response

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 104 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

To evaluate efficacy, safety and pharmacokinetic profile of asciminib 40mg+imatinib or asciminib 60mg+imatinib versus continued imatinib and versus nilotinib versus asciminib 80mg in pre-treated patients with Chronic Myeloid Leukemia in chronic phase (CML-CP)

Detailed description

The study is a Phase 2, multi-center, open-label, randomized study of asciminib in two different doses (40 mg or 60 mg) in combination with imatinib 400 mg versus continued imatinib versus switch to nilotinib in Participants with chronic myeloid leukemia in chronic phase (CML-CP) who have been previously treated with imatinib first line therapy for at least one year and have not achieved deep molecular response (DMR). Eligible participants were randomized 1:1:1:1 to receive asciminib 60 mg once daily (QD) as add-on therapy to imatinib 400 mg QD, or 40 mg QD as add-on therapy to imatinib 400 mg QD, or to continue imatinib 400 mg QD, or to switch to nilotinib 300 mg twice a day (BID). Participants on the imatinib continuation arm who had not achieved MR4.5 at 48 weeks were allowed to cross-over (CO) to receive the add-on treatment within 4 weeks after week 48 visit to receive the asciminib 60 mg combination add-on treatment, as this dose provided higher exposure. The cross-over is at the discretion of the investigator and the participant. Apart from a polymerase chain reaction (PCR) result of below MR4.5 at Week 48 visit, there are no other entry criteria for the cross-over part. Participants on nilotinib are not allowed to cross- over to receive the add-on treatment. Participants on the study will continue on the allocated treatment until treatment failure, intolerability, or for up to 96 weeks (in arms 1 to 4) after the last participant received the first dose of treatment. After the last dose received, every participant will be followed up for safety for 30 days.

Conditions

• CML
• Chronic Myelogenous Leukemia
• Leukemia, Myeloid Chronic
• Hematologic Diseases

Interventions

Type	Name	Description
DRUG	Asciminib add-on	Asciminib 60 mg or 40 mg taken orally once daily.
DRUG	Imatinib	Imatinib 400 mg taken orally once daily
DRUG	Nilotinib	Nilotinib 300 mg taken orally twice daily (total daily dose of 600 mg)

Timeline

• Start date: 2018-11-22
• Primary completion: 2021-11-08
• Completion: 2025-02-26
• First posted: 2018-07-06
• Last updated: 2025-11-10
• Results posted: 2025-02-14

Locations

30 sites across 15 countries: United States, Austria, Canada, Czechia, Denmark, France, Germany, Italy, Poland, Portugal, Russia, South Korea, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study