NCT05456191

A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)

A Phase IIIb, Multi-center, Open-label, Randomized Study of Tolerability and Efficacy of Oral Asciminib Versus Nilotinib in Patients With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase.

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 568 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study was to assess the tolerability of oral asciminib (80 mg QD) in comparison with that of the second generation (2G) Tyrosine Kinase Inhibitor (TKI) nilotinib (300 mg BID), in adult patients with newly diagnosed Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP).

Detailed description

Participants were randomized in the study in a 1:1 ratio to asciminib or nilotinib. No crossover of study treatment across arms were allowed. Randomization was stratified based on European Treatment Outcome Study (EUTOS) long-term survival (ELTS) score (low versus intermediate versus high) to help achieve a balance between the treatment arms. The primary analysis was performed when approximately 65 discontinuations of either study treatment due to Adverse Event (AE) occured. Eligible participants on both arms could choose to participate in an optional 2-year Treatment Free Remission (TFR) Phase. Participants with loss of Major Molecular Response (MMR) during TFR Phase were to enter the Treatment Re-initiation (TRI) Phase.

Conditions

• Philadelphia Chromosome-Positive Chronic Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Asciminib	Asciminib 80 mg QD administered under fasting conditions.
DRUG	Nilotinib	Nilotinib 300 mg twice a day (BID) was administered under fasting conditions.

Timeline

• Start date: 2022-11-21
• Primary completion: 2025-05-15
• Completion: 2031-07-07
• First posted: 2022-07-13
• Last updated: 2026-02-06

Locations

120 sites across 24 countries: United States, Argentina, Bulgaria, Canada, Czechia, France, Germany, Greece, Hungary, India, Italy, Jordan, Malaysia, Netherlands, Oman, Romania, Singapore, Slovakia, South Africa, South Korea, Switzerland, Turkey (Türkiye), United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study