Clinical trial NCT02160951: Dose Escalation Study of LGH447 in Japanese Patients With Relapsed and/or Refractory Hematologic Malignancies
Official title: A Multi-center, Open-label, Dose Escalation, Phase 1 Study of Oral LGH447 in Japanese Patients With Relapsed and/or Refractory Hematologic Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Multiple Myeloma
Keywords: Multiple Myeloma,
Phase I,
PIM,
Japanese patients,
LGH447
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-06-11
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a multi-center, open-label, dose escalation, Phase 1 study of oral LGH447 in Japanese patients with relapsed and/or refractory multiple myeloma for which no standard effective treatment options exist.

The study consists of a dose escalation part to estimate the maximum tolerated dose and/or the recommended dose for expansion and a dose expansion part to further assess safety and preliminary anti-cancer activity of LGH447 at the maximum tolerated dose and/or the recommended dose for expansion.


Eligibility criteria: Inclusion Criteria:

-Confirmed diagnosis of relapsed and/or refractory MM for which no standard effective treatment options exist.

Exclusion Criteria:

-Uncontrolled cardiovascular condition, including ongoing cardiac arrhythmias, congestive heart failure, angina, or myocardial infarction within the past 6 months.

Primary outcomes:
measure: Incidence rate of dose limiting toxicities
timeFrame: 28 days
Secondary outcomes:
measure: Number of patients with adverse events as a measure of safety and tolerability of LGH447
timeFrame: 28 days and till the end of the study, an average of 84 days
measure: Pharmacokinetics profile of LGH447 and its metabolites if appropriate
timeFrame: Baseline, 0.5, 1, 2, 3, 4, 5, 6, 8, 24 hours on Cycle1Day1, 14 and 28 and baseline on Cycle2Day14 and Cycle3Day1
measure: Overall Response Rate
timeFrame: Every 28 days till the end of the study, an average of 84 days
measure: Disease control rate
timeFrame: Every 28 days till the end of the study, an average of 84 days
measure: Clinical benefit rate
timeFrame: Every 28 days till the end of the study, an average of 84 days
measure: Duration of Response
timeFrame: Every 28 days till the end of the study, an average of 84 days, from the first documented onset of confirmed PR or better response to the date of documented disease progression/relapse or death due to multiple myeloma
measure: Progression Free Survival
timeFrame: Every 28 days till the end of the study, an average of 84 days, from start of treatment to the date of event defined as the first documented disease progression/relapse, or death due to any cause
measure: Time to response
timeFrame: Every 28 days till the end of the study, an average of 84 days, from start of treatment until first documented best overall response
Other outcomes:
Locations:
city: Nagoya-city
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Kobe-city
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Kyoto-city
country: Japan
facility: Novartis Investigative Site
state: Kyoto
city: Okayama-city
country: Japan
facility: Novartis Investigative Site
state: Okayama
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Iida S, Sunami K, Minami H, Hatake K, Sekiguchi R, Natsume K, Ishikawa N, Rinne M, Taniwaki M. A phase I, dose-escalation study of oral PIM447 in Japanese patients with relapsed and/or refractory multiple myeloma. Int J Hematol. 2021 Jun;113(6):797-806. doi: 10.1007/s12185-021-03096-9. Epub 2021 Feb 27.