NCT01730248

A Study to Find the Maximum Tolerated Dose of the Experimental Combination of the Drugs INC424 and BKM120 in Patients With Primary or Secondary Myelofibrosis

A Phase Ib, Open-label, Multi-center, Two-arm, Dose-finding Study to Assess Safety and Efficacy of the Oral Combination or INC424 (INC424) and BKM120 in Patients With Primary Myelofibrosis (PMF), Postpolycythemia Vera-myelofibrosis (PPV-MF), or Post-essential Thrombocythemia-myelofibrosis (PET-MF)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 63 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this phase Ib clinical trial is to evaluate the safety of the combination of INC424 and BKM120 in the myelofibrosis population and to establish the maximum tolerated dose and or the Recommended Phase II dose of the combination guided by the Bayesian dose escalation model. INC424 has shown efficacy in myelofibrosis (MF) and is approved in the US and EU for the treatment of MF. BKM120 is a PI3K inhibitor. Preclinical and early clinical experience support inhibition of the PI3K/mTOR pathway in MF as aberrant activation of the pathway has been observed in MF models and may contribute to the pathogenesis of the disease.

Conditions

• Myelofibrosis

Interventions

Type	Name	Description
DRUG	INC424	5 mg tablets administered orally twice daily
DRUG	BKM120	10 mg and 50 mg hard gelatin capsules administered orally once daily

Timeline

• Start date: 2012-12-18
• Primary completion: 2017-09-28
• Completion: 2017-09-28
• First posted: 2012-11-21
• Last updated: 2020-12-19

Locations

15 sites across 9 countries: Australia, Austria, France, Germany, Israel, Italy, Singapore, Spain, United Kingdom