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Clinical Trials/NCT02078609
NCT02078609
Completed
Phase 1

A Phase I, Multicenter, Open-label Study of Oral LGH447 in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

Novartis Pharmaceuticals1 site in 1 country70 target enrollmentStarted: March 20, 2014Last updated:

Overview

Phase
Phase 1
Status
Completed
Enrollment
70
Locations
1
Primary Endpoint
Incidence rate of dose limiting toxicities (DLTs) of LGH447 monotherapy arm in patients with AML or MDS and of LGH447 + midostaurin in patients with AML

Overview

Brief Summary

This study will assess the safety and preliminary efficacy of escalating doses of LGH447 monotherapy in AML and MDS and LGH447 in combination with midostaurin in AML.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Not provided

Exclusion Criteria

  • Not provided

Arms & Interventions

LGH447 monotherapy arm

Experimental

LGH447 monotherapy in patients with AML or MDS

Intervention: LGH447 (Drug)

LGH447 + midostaurin combination arm

Experimental

LGH447 + midostaurin in patients with AML

Intervention: LGH447 + midostaurin (Drug)

Outcomes

Primary Outcomes

Incidence rate of dose limiting toxicities (DLTs) of LGH447 monotherapy arm in patients with AML or MDS and of LGH447 + midostaurin in patients with AML

Time Frame: 28 days post study treatment

Frequency and characteristics of dose limiting toxicities

Secondary Outcomes

  • Number of participants with the type, frequency, and severity of adverse events (AEs) as a measure of safety and tolerability of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML(weekly to bi-weekly up to 1.5 years)
  • PK parameters of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML(days 1, 2, 15, 16, 29, 30, 44, 57, and approximately monthly through Cycle 3)
  • Changes between pre- and post-treatment levels of pS6RP and p4EBP1 in bone marrow aspirates and p4EBP1 in peripheral blood of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML(screening, days 1 and 29 up to 1.5 years)
  • Anti-tumor activity in AML or high risk MDS associated wtih LGH447(Day 29 up to 1.5 years)
  • Anti-tumor activity in AML or high risk MDS associated wtih LGH447 in combination with midostaurin(Day 29 up to 1.5 years)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (1)

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