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A Phase I, Multicenter, Open-label Study of Oral ​​ABL001 in Patients With CML or ​Ph+ ALL
Recruitment period: 
26 June 2015
Trial period: 
1 April 2014 to 1 February 2017
Clinical Trial reference link: 
For newly diagnosed patients: 

This study is currently recruiting participants. (see Contacts and Locations)  


Novartis Pharmaceuticals


This first-in-human phase 1 trial as

1. As single agent ABL001 in a dose escalation study to determine the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) of single agent ABL001 in chronic or accelerated phase PH+ CML in patients previously treated with 2 different TKIs and who are resistant or intolerant as determined by investigators, and in chronic or accelerated phase PH+ CML patients positive for the T315i mutation.

2. Combination of ABL001 and Nilotinib in Ph+ CML in chronic and accelerated phase patients.

3. ABL001 in Ph+ALL patients

The safety, tolerability and pharmacokinetic (PK) profile of ABL001 and ABL001+Nilotinib will be assessed together with an evaluation of pharmacodynamic (PD) changes in peripheral blood mononuclear cells (PBMC) and bone marrow aspirates and all data may contribute to the assessment of the RDE.

An understanding of the MTD/RDE, safety profile, PK/PD relationship, and preliminary evidence of anti-CML and ALL activity will be used to inform future development in adults with CML and Ph+ ALL. By virtue of its distinct pharmacological profile and by preclinical pharmacological studies demonstrating an additive effect, a combination of ABL001 and a tyrosine-kinase inhibitor (TKI) has the potential to achieve a deeper molecular response in a higher proportion of CML patients as compared to single agent TKI therapy. Such a combination has the added advantage of targeting the ABL kinase domain at two distinct locations, theoretically preventing single point mutation-associated treatment resistance. The prediction is that a nilotinib/ABL001 combination will increase the percentage of patients who achieve a complete molecular response (CMR) and decrease the time to CMR, thereby increasing the possibility of achieving sustained treatment-free remissions in these patients. In addition, some patients may be intolerant of therapy with TKIs or may develop mutations that promote resistance to TKI therapy. In these patients, ABL001 may provide a novel therapeutic option.


  • United States
  • Australia
  • France
  • Germany
  • Italy
  • Japan
  • Korea, Republic of
  • Netherlands
  • Singapore
  • Spain