A Study to Assess the Safety of Xospata in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-Like Tyrosine Kinase 3 (FLT3) Mutation

An Observational Study to Assess the Safety of Xospata® 40 mg Tablet When Administered in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-Like Tyrosine Kinase 3 (FLT3) Mutation

The objective of this study is to describe the observed safety profile of Xospata® 40 mg tablet when administered in patients with relapsed or refractory AML with FLT3 mutation in routine clinical practice in Korea.

Study Overview

• Status: Completed
• Conditions: Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
• Intervention / Treatment: Drug: Gilteritinib Exposure

Detailed Description

This study is being mandated by Ministry of Food and Drug Safety (MFDS) as a part of the Korea-Risk Management Plan (K-RMP) to assess safety in patients with relapsed or refractory AML with FLT3 mutation in routine clinical practice in Korea. This study collects data for 54 months according to the purpose of this study in routine clinical practice as an observational study.

• Study Type: Observational
• Enrollment (Actual): 33

Contacts and Locations

Study Locations

• Korea, Republic of
  • Busan, Korea, Republic of, 47392
    • Site KR82011
  • Daegu, Korea, Republic of, 41944
    • Site KR82010
  • Incheon, Korea, Republic of, 21565
    • Site KR82001
  • Seoul, Korea, Republic of, 03080
    • Site KR82004
  • Seoul, Korea, Republic of, 03722
    • Site KR82005
  • Seoul, Korea, Republic of, 05505
    • Site KR82009
  • Seoul, Korea, Republic of, 07985
    • Site KR82007
  • Gangwon-do
    • Wonju-si, Gangwon-do, Korea, Republic of, 26426
      • Site KR82013
  • Gyeonggi-do
    • Goyang-si, Gyeonggi-do, Korea, Republic of, 10408
      • Site KR82006
  • Hwasun-gun
    • Jeollanam-do, Hwasun-gun, Korea, Republic of, 58128
      • Site KR82003

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients receiving Xospata® 40 mg tablet according to the drug label for 54 months from the start date of marketing in Korea.

Description

Inclusion Criteria:

• Patients who receive Xospata® 40 mg tablet according to the drug label approved at the time of marketing authorization in routine clinical practice.
• Patients who voluntarily signed the written informed consent form.

Exclusion Criteria:

• Patients who meet the section 'Do not administer to the following patients' in the precautions for use given at the time of marketing authorization.
• Patients who use the drug for an off-label purpose.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Xospata; Patients who receive Xospata® 40 mg tablet in routine clinical practice according to the drug label approved at the time of marketing authorization.	Drug: Gilteritinib Exposure; Oral; Other Names: Xospata

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with Adverse Drug Reactions (ADRs) related to important identified and/or potential risks	An Adverse Drug Reaction refers to any unfavorable and unintended reaction occurring with a normal administration or use of the medicinal product that a causal relationship to the medicinal product cannot be ruled out. Number of ADRs related to important identified risks such as posterior reversible encephalopathy syndrome (PRES), QT prolongation, differentiation syndrome, and/or important potential risks such as pancreatitis, embryo-fetal lethality, suppressed fetal growth and teratogenicity, will be recorded.	Up to a maximum of 54 months (until 30 days after the last dose)
Number of participants with serious ADRs related to important identified and/or potential risks	An ADR refers to any unfavorable and unintended reaction occurring with a normal administration or use of the medicinal product that a causal relationship to the medicinal product cannot be ruled out. Number of ADRs related to important identified risks such as posterior reversible encephalopathy syndrome (PRES), QT prolongation, differentiation syndrome, and/or important potential risks such as pancreatitis, embryo-fetal lethality, suppressed fetal growth and teratogenicity, will be recorded. An ADR is considered "serious" if, in the view of either the investigator or sponsor, it results in any of the following outcomes: results in death, is life-threatening, results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly or birth defect, requires inpatient hospitalization or prolongation of hospitalization, or other medically important event.	Up to a maximum of 54 months (until 30 days after the last dose)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with ADRs related to identified risks and considered not important	An ADR refers to any unfavorable and unintended reaction occurring with a normal administration or use of the medicinal product that a causal relationship to the medicinal product cannot be ruled out. Number of ADRs related to identified risks but considered not important, such as other ADRs described by the Korean package insert, will be recorded. An ADR is considered "serious" if, in the view of either the investigator or Sponsor, it results in any of the following outcomes: results in death, is life-threatening, results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly or birth defect, requires inpatient hospitalization or prolongation of hospitalization, or other medically important event.	Up to a maximum of 54 months (until 30 days after the last dose)
Number of participants with AEs	An AE is defined as any untoward medical occurrence in a subject administered a study drug, and which does not necessarily have to have a causal relationship with this treatment. An AE is considered "serious" if, in the view of either the investigator or Sponsor, it results in any of the following outcomes: results in death, is life-threatening, results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, results in congenital anomaly or birth defect, requires inpatient hospitalization or prolongation of hospitalization, or other medically important event. Causal relationship between the study drug is judged by medically qualified investigator either as certain, probable/likely, possible, unlikely, conditional/unclassified or unassessable/unclassifiable.	Up to a maximum of 54 months (until 30 days after the last dose)

Collaborators and Investigators

• Sponsor: Astellas Pharma Korea, Inc.
• Investigators: Study Director: Astellas Pharma Korea, Inc., Astellas Pharma Korea, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 17, 2022
• Primary Completion (Actual): May 4, 2025
• Study Completion (Actual): May 4, 2025

Study Registration Dates

• First Submitted: December 30, 2020
• First Submitted That Met QC Criteria: December 30, 2020
• First Posted (Actual): December 31, 2020

Study Record Updates

• Last Update Posted (Actual): May 31, 2025
• Last Update Submitted That Met QC Criteria: May 30, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Gilteritinib; Xospata; Relapsed or refractory Acute Myeloid Leukemia (AML)
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Tyrosine Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; Gilteritinib
• Other Study ID Numbers: 2215-PV-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.