Dose Escalation Study Investigating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics of ASP2215 in Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT ID: NCT02014558

Last Updated: 2024-12-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

265 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-10-09

Study Completion Date

2018-03-07

Brief Summary

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The objective of this study was to assess the safety and tolerability, including the maximum tolerated dose, of gilteritinib in participants with relapsed or treatment-refractory acute myeloid leukemia (AML). This study also determined the pharmacokinetic (PK) parameters of gilteritinib.

Detailed Description

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Conditions

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Acute Myeloid Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Gilteritinib 20 mg in Escalation Phase

Participants received a single dose of 20 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 20 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Voriconazole

Intervention Type DRUG

Participants received 200 mg voriconazole tablets daily every 12 hours starting from day 16 of cycle 1 through day 1 of cycle 2.

Gilteritinib 40 mg in Escalation Phase

Participants received a single dose of 40 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 40 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 80 mg in Escalation Phase

Participants received a single dose of 80 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 80 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 120 mg in Escalation Phase

Participants received a single dose of 120 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 120 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 200 mg in Escalation Phase

Participants received a single dose of 200 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 200 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 300 mg in Escalation Phase

Participants received a single dose of 300 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 300 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 450 mg in Escalation Phase

Participants received a single dose of 450 mg gilteritinib orally on day -2 to evaluate pharmacokinetics of gilteritinib. Then starting on day 1 of cycle 1, participants received 450 mg gilteritinib orally once daily in 28-day cycles until disease progression or participant discontinuation in the escalation phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 20 mg in Expansion Phase

Participants received 20 mg gilteritinib orally once daily stating on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study. Starting on day 16 of cycle 1, participants also received 200 mg voriconazole orally every 12 hours through day 1 of cycle 2.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 40 mg in Expansion Phase

Participants received 40 mg gilteritinib orally once daily starting on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 80 mg in Expansion Phase

Participants received 80 mg gilteritinib orally once daily starting on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 120 mg in Expansion Phase

Participants received 120 mg gilteritinib orally once daily starting on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Gilteritinib 200 mg in Expansion Phase

Participants received 200 mg gilteritinib orally once daily starting on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study. On day -1 and day 15 of cycle 1, certain participants also received 500 mg cephalexin as a single oral dose.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Cephalexin

Intervention Type DRUG

Participants received a single oral dose of 500 mg cephalexin tablet or capsule on day -1 and day 15 of cycle 1.

Gilteritinib 300 mg in Expansion Phase

Participants received 300 mg gilteritinib orally once daily starting on day 1 of cycle 1 and continued in 28-day cycles until disease progression or participant discontinuation in the expansion phase of the study. On day -1 and day 15 of cycle 1, participants also received 2 mg midazolam as a single oral dose.

Group Type EXPERIMENTAL

Gilteritinib

Intervention Type DRUG

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Midazolam

Intervention Type DRUG

Participants received a single oral dose of 2 mg of midazolam syrup on day -1 and day 15 of cycle 1.

Interventions

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Gilteritinib

Participants received gilteritinib oral tablets (10 mg, 40 mg or 100 mg, depending on the dose) once daily without food allowed for at least 2 hours before and 1 hour after dosing starting from day -2 and day of cycle 1, for continuous 28-day cycles.

Intervention Type DRUG

Voriconazole

Participants received 200 mg voriconazole tablets daily every 12 hours starting from day 16 of cycle 1 through day 1 of cycle 2.

Intervention Type DRUG

Midazolam

Participants received a single oral dose of 2 mg of midazolam syrup on day -1 and day 15 of cycle 1.

Intervention Type DRUG

Cephalexin

Participants received a single oral dose of 500 mg cephalexin tablet or capsule on day -1 and day 15 of cycle 1.

Intervention Type DRUG

Other Intervention Names

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ASP2215

Eligibility Criteria

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Inclusion Criteria

* Subject is defined as morphologically documented primary or secondary AML by the World Health Organization (WHO) criteria (2008) and fulfills one of the following:

* Refractory to at least 1 cycle of induction chemotherapy
* Relapsed after achieving remission with a prior therapy
* Subject has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
* Subject's interval from prior treatment to time of study drug administration is at least 2 weeks for cytotoxic agents (except hydroxyurea given for controlling blast cells), or at least 5 half-lives for prior experimental agents or noncytotoxic agents.
* Subject must meet the following criteria as indicated on the clinical laboratory tests\*:

* Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<2.5 x institutional upper limit normal (ULN)
* Total serum bilirubin \< 1.5x institutional ULN
* Serum creatinine \< 1.5 x institutional ULN or an estimated glomerular filtration rate (eGFR) of \> 50 ml/min as calculated by the Modification of Diet in Renal Disease (MDRD) equation.
* Subject agrees not to participate in another interventional study while on treatment.

Exclusion Criteria

* Subject was diagnosed as acute promyelocytic leukemia (APL).
* Subject has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis).
* Subject has active malignant tumors other than AML or Myelodysplastic syndrome (MDS).
* Subject has persistent nonhematological toxicities of \>= Grade 2 (Common Terminology Criteria for Adverse Events v4), with symptoms and objective findings, from prior AML treatment (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, radiation, or surgery).
* Subject has had hematopoietic stem cell transplant (HSCT) and meets any of the following:

* Is within 2 months of transplant from C1D1
* Has clinically significant graft-versus-host disease requiring treatment
* Has \>= Grade 2 persistent non-hematological toxicity related to the transplant. Donor lymphocytes infusion (DLI) is not permitted \<= 30 days prior to study registration or during the first cycle of treatment on the study in Cohort 1 and first two cycles of the treatment in Cohort 2
* Subject has clinically active central nervous system leukemia
* Subject has disseminated intravascular coagulation abnormality (DIC)
* Subject has had major surgery within 4 weeks prior to the first study dose.
* Subject has had radiation therapy within 4 weeks prior to the first study dose
* Subject has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or subject with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram performed within 3 months prior to study entry results in a left ventricular ejection fraction that is ≥ 45%
* Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of Cytochrome P450-isozyme3A4 (CYP3A4) with the exception of antibiotics, antifungals, and antivirals that are used as standard of care post-transplant or to prevent or treat infections and other such drugs that are considered absolutely essential for the care of the subject
* Subject required treatment with concomitant drugs that target serotonin 5HT1R or 5HT2BR receptors or sigma nonspecific receptor with the exception of drugs that are considered absolutely essential for the care of the subject.
* Subject has an active uncontrolled infection
* Subject is known to have human immunodeficiency virus infection
* Subject has active hepatitis B or C, or other active hepatic disorder
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Astellas Pharma Global Development, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Executive Medical Director

Role: STUDY_DIRECTOR

Astellas Pharma Global Development

Locations

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Site US10021

Birmingham, Alabama, United States

Site Status

Site US10023

Scottsdale, Arizona, United States

Site Status

Site US10022

Duarte, California, United States

Site Status

Site US10008

Los Angeles, California, United States

Site Status

Site US10005

San Francisco, California, United States

Site Status

Site US10001

Chicago, Illinois, United States

Site Status

Site US10015

Chicago, Illinois, United States

Site Status

Site US10012

Baltimore, Maryland, United States

Site Status

Site US10003

Baltimore, Maryland, United States

Site Status

Site US10006

Minneapolis, Minnesota, United States

Site Status

Site US10011

Rochester, Minnesota, United States

Site Status

Site US10020

Hackensack, New Jersey, United States

Site Status

Site US10010

Buffalo, New York, United States

Site Status

Site US10009

New York, New York, United States

Site Status

Site US10013

New York, New York, United States

Site Status

Site US10019

New York, New York, United States

Site Status

Site US10014

Cleveland, Ohio, United States

Site Status

Site US10018

Hershey, Pennsylvania, United States

Site Status

Site US10004

Philadelphia, Pennsylvania, United States

Site Status

Site US10017

Charleston, South Carolina, United States

Site Status

Site US10007

Nashville, Tennessee, United States

Site Status

Site US10002

Houston, Texas, United States

Site Status

Site US10026

Fairfax, Virginia, United States

Site Status

Site DE49002

Berlin, , Germany

Site Status

Site DE49004

Dresden, , Germany

Site Status

Site IT39001

Bologna, , Italy

Site Status

Countries

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United States Germany Italy

References

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James AJ, Smith CC, Litzow M, Perl AE, Altman JK, Shepard D, Kadokura T, Souda K, Patton M, Lu Z, Liu C, Moy S, Levis MJ, Bahceci E. Pharmacokinetic Profile of Gilteritinib: A Novel FLT-3 Tyrosine Kinase Inhibitor. Clin Pharmacokinet. 2020 Oct;59(10):1273-1290. doi: 10.1007/s40262-020-00888-w.

Reference Type DERIVED
PMID: 32304015 (View on PubMed)

Perl AE, Altman JK, Cortes J, Smith C, Litzow M, Baer MR, Claxton D, Erba HP, Gill S, Goldberg S, Jurcic JG, Larson RA, Liu C, Ritchie E, Schiller G, Spira AI, Strickland SA, Tibes R, Ustun C, Wang ES, Stuart R, Rollig C, Neubauer A, Martinelli G, Bahceci E, Levis M. Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study. Lancet Oncol. 2017 Aug;18(8):1061-1075. doi: 10.1016/S1470-2045(17)30416-3. Epub 2017 Jun 20.

Reference Type DERIVED
PMID: 28645776 (View on PubMed)

Kasi PM, Litzow MR, Patnaik MM, Hashmi SK, Gangat N. Clonal evolution of AML on novel FMS-like tyrosine kinase-3 (FLT3) inhibitor therapy with evolving actionable targets. Leuk Res Rep. 2016 Jan 12;5:7-10. doi: 10.1016/j.lrr.2016.01.002. eCollection 2016.

Reference Type DERIVED
PMID: 26904404 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://astellasclinicalstudyresults.com/study.aspx?ID=303

Link to results on the Astellas Clinical Study Results website

Other Identifiers

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2215-CL-0101

Identifier Type: -

Identifier Source: org_study_id