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Expanded Access Study of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

Primary Purpose

Acute Myeloid Leukemia (AML), FMS-like Tyrosine Kinase-3 (FLT3) Mutations

Status
Approved for marketing
Phase
Locations
International
Study Type
Expanded Access
Intervention
gilteritinib
Sponsored by
Astellas Pharma Global Development, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Acute Myeloid Leukemia (AML) focused on measuring Acute myeloid leukemia (AML), FMS-like Tyrosine kinase-3 (FLT3) mutations, gilteritinib, Expanded access, ASP2215

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

Inclusion Criteria:

  • Subject is considered an adult according to local regulation at the time of signing informed consent.
  • Subject has a diagnosis of primary AML or AML secondary to myelodysplastic syndrome (MDS) or therapy-related AML according to World Health Organization (WHO) classification.
  • Subject has presence of the FLT3-mutated relapsed or refractory AML or FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD in bone marrow or peripheral blood. [Specific to investigational sites in Japan: FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD subjects will not be included.]
  • Subject has refractory or relapsed AML (with or without hematopoietic stem cell transplant [HSCT]) or AML in CRc (CR, CRi, CRp) with MRD by flow cytometry or genetic testing for the FLT3 mutation after induction/consolidation regimen or HSCT. [Specific to investigational sites in Japan: FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD subjects will not be included.]
  • Subject must wait for at least 5 half-lives after stopping therapy with any investigational agent and before starting ASP2215.
  • Subject must meet the following criteria as indicated on clinical laboratory tests:

    • Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3x institutional upper limit of normal (ULN)
    • Serum total bilirubin ≤ 2.5 mg/dL, except for subjects with Gilbert's syndrome
    • Serum potassium and serum magnesium ≥ institutional lower limit of normal (LLN).
  • Subject is able to tolerate oral administration of study drug.
  • Subject who has developed overall grades II-IV acute graft-versus-host disease (GVHD) must satisfy the following criteria:

    • No requirement of > 0.5 mg/kg of prednisone (or equivalent) daily dose within 1 week of enrollment
    • No escalation of immunosuppression in terms of increase of corticosteroids or addition of new agent/modality in prior 2 weeks (note that increasing calcineurin inhibitors or sirolimus to achieve therapeutic trough levels is allowed)
  • Female subject must either:

    • Be of nonchildbearing potential:
    • Postmenopausal (defined as at least 1 year without any menses) prior to screening, or
    • Documented surgically sterile (e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy) at least 1 month prior to screening.
    • Or, if of childbearing potential,
    • Agree not to try to become pregnant during the study and for at least 180 days after the final study drug administration
    • And have a negative urine pregnancy test at screening
    • And, if heterosexually active, agree to use consistently 2 forms of effective contraception per locally accepted standards (1 of which must be a barrier method) starting at screening and throughout the study period and for at least 180 days after the final study drug administration.
  • Female subject must agree not to breastfeed or donate ova starting at screening and throughout the study period, and for at least 180 days after the final study drug administration.
  • Male subject (even if surgically sterilized) and partners who are women of childbearing potential must agree to practice 2 forms of effective contraception per locally accepted standards

    (1 of which must be a barrier method), starting at screening and throughout the study period and for 120 days after the final study drug administration.

  • Male subject must not donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration.
  • Subject agrees not to participate in another interventional study for AML while on treatment.
  • Subject who has a diagnosis of HIV may be enrolled as long as the disease is under control on antiretroviral therapy. Precautions should be taken to modify highly active antiretroviral therapy (HAART) regimen to minimize drug interactions.
  • There is no comparable or satisfactory alternative therapy to treat the subject's AML.

Exclusion Criteria:

  • Subject is eligible to participate in an ongoing clinical study of ASP2215; or has previously participated in a randomized clinical study of ASP2215 with a primary endpoint of overall survival that is not closed for efficacy.
  • Subject with QTcF > 450 ms at screening based on local reading.
  • Subject with a known history of Long QT Syndrome at screening.
  • Subject was diagnosed with acute promyelocytic leukemia (APL).
  • Subject has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis).
  • Subject has clinically significant coagulation abnormality unless secondary to AML.
  • Subject has active hepatitis B or C or an active hepatic disorder.
  • Subject has uncontrolled angina, severe uncontrolled ventricular arrhythmias, electrocardiographic evidence of acute ischemia, or New York Heart Association (NYHA) Class IV heart failure.
  • Subject requires treatment with concomitant drugs that are strong inducers of CYP3A.
  • Subject has any condition which makes the subject unsuitable for study participation.
  • Subject has hypersensitivity to any of the study drug components.

Sites / Locations

  • UCLA
  • Rocky Mountain Cancer Center-M
  • Memorial Healthcare System
  • Northside Hospital
  • Georgia Cancer Center at Augusta University
  • Northwestern University Medical Center
  • University of Chicago
  • Indiana Blood and Marrow Transplantation at Franciscan Health Indianapolis
  • Norton Cancer Institute
  • Tulane University
  • University of Maryland Medical Center
  • Johns Hopkins Hospital
  • The Sidney Kimmel Comprehensive Cancer Center -Johns Hopkins University
  • Massachusetts General Hospital
  • Beth Israel Deaconess Medical Center
  • Dana Farber Cancer Institute
  • West Michigan Regional Cancer Center
  • Washington University School of Medicine
  • New Mexico Cancer Care Alliance
  • Roswell Park Cancer Institute
  • Memorial Sloan Kettering
  • Weill Cornell Medical College
  • Duke University Medical Center
  • Wake Forest Baptist Hospital
  • Ohio State University
  • Providence Portland Medical Center
  • Penn State Hershey Medical Center
  • Thomas Jefferson University
  • UPCI
  • Vanderbilt University
  • Huntsman Cancer Institute University of Utah
  • WVU Medicine Cancer
  • Site CA15002
  • Site CA15003
  • Site CA15001
  • Site CA15005
  • Site JP81001
  • Site JP81002
  • Site JP81003

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 28, 2017
Last Updated
April 18, 2021
Sponsor
Astellas Pharma Global Development, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03070093
Brief Title
Expanded Access Study of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)
Official Title
A Multicenter, Open-label Treatment Protocol of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)
Study Type
Expanded Access

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Approved for marketing
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Astellas Pharma Global Development, Inc.

4. Oversight

5. Study Description

Brief Summary
The purpose of this study is to provide expanded access to ASP2215 for subjects with FLT3-mutated relapsed or refractory AML or FLT3-mutated AML in composite complete remission (CRc) (complete remission [CR], complete remission with incomplete hematologic recovery [CRi], complete remission with incomplete platelet recovery [CRp]) with MRD without access to comparable or alternative therapy.
Detailed Description
The United States Food and Drug Administration (FDA), the Japanese Ministry of Health, Labour and Welfare (MHLW) and Health Canada have approved ASP2215/Gilteritinib (XOSPATA®) for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation. This treatment protocol is being conducted while phase 3 ASP2215 studies are ongoing in FLT3-mutated AML subjects. Subjects will complete visits on cycle 1 - days 1, 4, 8, 15; cycle 2 - days 1, 15; cycles 3 to 6 - day 1; and day 1 of every 2 cycles thereafter (i.e., cycle 8 day 1, cycle 10 day 1, etc.) until discontinued from the study. Subjects will be provided with study medication until the investigator determines the subject is no longer receiving clinical benefit. An end of treatment visit will be performed within 7 days after last dose of investigational product (ASP2215), or prior to initiation of another anticancer therapy, whichever occurs earlier, followed by a 30-day follow-up. [Specific to investigational sites in Japan: Study population does not include subjects with FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD. Hence, efficacy (MRD response rate and duration of response) data will not be collected for subjects enrolled in Japan.]

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia (AML), FMS-like Tyrosine Kinase-3 (FLT3) Mutations
Keywords
Acute myeloid leukemia (AML), FMS-like Tyrosine kinase-3 (FLT3) mutations, gilteritinib, Expanded access, ASP2215

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
gilteritinib
Other Intervention Name(s)
ASP2215
Intervention Description
oral

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Eligibility Criteria
Inclusion Criteria: Subject is considered an adult according to local regulation at the time of signing informed consent. Subject has a diagnosis of primary AML or AML secondary to myelodysplastic syndrome (MDS) or therapy-related AML according to World Health Organization (WHO) classification. Subject has presence of the FLT3-mutated relapsed or refractory AML or FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD in bone marrow or peripheral blood. [Specific to investigational sites in Japan: FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD subjects will not be included.] Subject has refractory or relapsed AML (with or without hematopoietic stem cell transplant [HSCT]) or AML in CRc (CR, CRi, CRp) with MRD by flow cytometry or genetic testing for the FLT3 mutation after induction/consolidation regimen or HSCT. [Specific to investigational sites in Japan: FLT3-mutated AML in CRc (CR, CRi, CRp) with MRD subjects will not be included.] Subject must wait for at least 5 half-lives after stopping therapy with any investigational agent and before starting ASP2215. Subject must meet the following criteria as indicated on clinical laboratory tests: Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3x institutional upper limit of normal (ULN) Serum total bilirubin ≤ 2.5 mg/dL, except for subjects with Gilbert's syndrome Serum potassium and serum magnesium ≥ institutional lower limit of normal (LLN). Subject is able to tolerate oral administration of study drug. Subject who has developed overall grades II-IV acute graft-versus-host disease (GVHD) must satisfy the following criteria: No requirement of > 0.5 mg/kg of prednisone (or equivalent) daily dose within 1 week of enrollment No escalation of immunosuppression in terms of increase of corticosteroids or addition of new agent/modality in prior 2 weeks (note that increasing calcineurin inhibitors or sirolimus to achieve therapeutic trough levels is allowed) Female subject must either: Be of nonchildbearing potential: Postmenopausal (defined as at least 1 year without any menses) prior to screening, or Documented surgically sterile (e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy) at least 1 month prior to screening. Or, if of childbearing potential, Agree not to try to become pregnant during the study and for at least 180 days after the final study drug administration And have a negative urine pregnancy test at screening And, if heterosexually active, agree to use consistently 2 forms of effective contraception per locally accepted standards (1 of which must be a barrier method) starting at screening and throughout the study period and for at least 180 days after the final study drug administration. Female subject must agree not to breastfeed or donate ova starting at screening and throughout the study period, and for at least 180 days after the final study drug administration. Male subject (even if surgically sterilized) and partners who are women of childbearing potential must agree to practice 2 forms of effective contraception per locally accepted standards (1 of which must be a barrier method), starting at screening and throughout the study period and for 120 days after the final study drug administration. Male subject must not donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration. Subject agrees not to participate in another interventional study for AML while on treatment. Subject who has a diagnosis of HIV may be enrolled as long as the disease is under control on antiretroviral therapy. Precautions should be taken to modify highly active antiretroviral therapy (HAART) regimen to minimize drug interactions. There is no comparable or satisfactory alternative therapy to treat the subject's AML. Exclusion Criteria: Subject is eligible to participate in an ongoing clinical study of ASP2215; or has previously participated in a randomized clinical study of ASP2215 with a primary endpoint of overall survival that is not closed for efficacy. Subject with QTcF > 450 ms at screening based on local reading. Subject with a known history of Long QT Syndrome at screening. Subject was diagnosed with acute promyelocytic leukemia (APL). Subject has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis). Subject has clinically significant coagulation abnormality unless secondary to AML. Subject has active hepatitis B or C or an active hepatic disorder. Subject has uncontrolled angina, severe uncontrolled ventricular arrhythmias, electrocardiographic evidence of acute ischemia, or New York Heart Association (NYHA) Class IV heart failure. Subject requires treatment with concomitant drugs that are strong inducers of CYP3A. Subject has any condition which makes the subject unsuitable for study participation. Subject has hypersensitivity to any of the study drug components.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Astellas Pharma Global Development, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Rocky Mountain Cancer Center-M
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80012
Country
United States
Facility Name
Memorial Healthcare System
City
Pembroke Pines
State/Province
Florida
ZIP/Postal Code
33028
Country
United States
Facility Name
Northside Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Georgia Cancer Center at Augusta University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Northwestern University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Indiana Blood and Marrow Transplantation at Franciscan Health Indianapolis
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
45237
Country
United States
Facility Name
Norton Cancer Institute
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40207
Country
United States
Facility Name
Tulane University
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
University of Maryland Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
The Sidney Kimmel Comprehensive Cancer Center -Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Beth Israel Deaconess Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Dana Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
West Michigan Regional Cancer Center
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49007
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
New Mexico Cancer Care Alliance
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87106
Country
United States
Facility Name
Roswell Park Cancer Institute
City
Buffalo
State/Province
New York
ZIP/Postal Code
14263
Country
United States
Facility Name
Memorial Sloan Kettering
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Wake Forest Baptist Hospital
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Ohio State University
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Providence Portland Medical Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97213
Country
United States
Facility Name
Penn State Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19066
Country
United States
Facility Name
UPCI
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Huntsman Cancer Institute University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
WVU Medicine Cancer
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Site CA15002
City
Halifax
State/Province
Nova Scotia
Country
Canada
Facility Name
Site CA15003
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Site CA15001
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H1T 2M4
Country
Canada
Facility Name
Site CA15005
City
Vancouver
Country
Canada
Facility Name
Site JP81001
City
Nagoya
State/Province
Aichi
Country
Japan
Facility Name
Site JP81002
City
Shinagawa-ku
State/Province
Tokyo
Country
Japan
Facility Name
Site JP81003
City
Fukuoka
Country
Japan

12. IPD Sharing Statement

Learn more about this trial

Expanded Access Study of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

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