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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03409081




Registration number
NCT03409081
Ethics application status
Date submitted
18/01/2018
Date registered
24/01/2018
Date last updated
13/09/2018

Titles & IDs
Public title
Early Access Program (EAP) of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or With FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)
Scientific title
Early Access Program (EAP) of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or With FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)
Secondary ID [1] 0 0
2215-CL-9200
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Myeloid Leukemia (AML) 0 0
FMS-like Tyrosine Kinase-3 (FLT3) Mutations 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Cancer 0 0 0 0
Other cancer types

Intervention/exposure
Study type
Expanded Access
Description of intervention(s) / exposure
Treatment: Drugs - gilteritinib

Treatment: Drugs: gilteritinib
oral

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
- Patient is considered an adult according to local regulation at the time of signing
informed consent.

- Patient has a diagnosis of primary AML or AML secondary to myelodysplastic syndrome or
therapy-related AML according to World Health Organization (WHO) classification as
determined by pathology review at the treating institution.

- Patient has presence of the FLT3 mutation (internal tandem duplication and/or tyrosine
kinase domain [D835/I836] mutation) in bone marrow or peripheral blood.

- Patient has refractory or relapsed AML (with or without Hematopoietic stem cell
transplant (HSCT)) or has AML in CRc (CR, CRi, CRp) with Minimal residual disease
(MRD) by flow cytometry or genetic testing for the FLT3 mutation after
induction/consolidation regimen or HSCT.

- There is no comparable or satisfactory alternative therapy to treat the patient's AML.

- Patient has not received any chemotherapy or investigational agent within at least 5
half-lives after stopping that drug and before starting gilteritinib (ASP2215).

- Patient must meet the following criteria as indicated on clinical laboratory tests:

- Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) = 3
institutional upper limit of normal

- Serum total bilirubin = 2.5 mg/dL, except for patients with Gilbert's syndrome

- Serum potassium and serum magnesium = institutional lower limit of normal.

- Patient is able to tolerate oral administration of gilteritinib (ASP2215).

- Patient 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 the initiation of gilteritinib (ASP2215) treatment.

- 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 patient must either:

- Be of nonchildbearing potential: Postmenopausal (defined as at least 1 year
without any menses for which there is no other obvious pathological or
physiological cause) 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
program and for at least 180 days after the final medicinal product
administration, and have a negative serum or 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 program period and for at least
180 days after the final medicinal product administration.

- Female patient must agree not to breastfeed or donate ova starting at screening and
throughout the program period, and for at least 180 days after the final medicinal
product administration.

- Male patient (even if surgically sterilized) and their 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 program period and for 120 days after the final medicinal
product administration.

- Male patient must not donate sperm starting at patient evaluation and throughout the
program period and for 120 days after the final medicinal product administration.

- Patient agrees not to participate in an interventional study for AML while on
treatment.

- Patient who has a diagnosis of human immunodeficiency virus may be registered in the
program as long as their disease is under control on antiretroviral therapy.
Precautions should be taken to modify their highly active antiretroviral therapy
regimen to minimize drug interactions.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Patient is able to participate in an ongoing clinical study of gilteritinib (ASP2215);
or has previously participated in a randomized clinical study of gilteritinib
(ASP2215) with a primary endpoint of survival that is not closed for efficacy.

- Patient with Fridericia-corrected QT interval (QTcF) > 450 ms at the screening visit
based on local reading.

- Patient with a known history of Long QT Syndrome at the screening visit.

- Patient was diagnosed with acute promyelocytic leukemia (APL).

- Patient has BCR-ABL-positive leukemia (chronic myelogenous leukemia in blast crisis).

- Patient has clinically significant coagulation abnormality unless secondary to AML.

- Patient has active hepatitis B or C or an active hepatic disorder.

- Patient has uncontrolled angina, severe uncontrolled ventricular arrhythmias,
electrocardiographic evidence of acute ischemia, or New York Heart Association Class
IV heart failure.

- Patient requires treatment with concomitant drugs that are strong inducers of
cytochrome P450 (CYP) 3A.

- Patient has any condition which makes the patient unsuitable for participation in the
program.

- Patient has hypersensitivity to any of the medicinal product components.

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
No longer available
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Site AU61001 - Canberra
Recruitment postcode(s) [1] 0 0
- Canberra
Recruitment outside Australia
Country [1] 0 0
Italy
State/province [1] 0 0
Milan
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Birmingham
Country [3] 0 0
United Kingdom
State/province [3] 0 0
Bristol
Country [4] 0 0
United Kingdom
State/province [4] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Astellas Pharma Global Development, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to provide expanded access to gilteritinib (ASP2215) for
patients with FMS-like tyrosine kinase 3 (FLT3)-mutated relapsed or refractory acute myeloid
leukemia (AML) or with 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 minimal residual disease (MRD)
without access to comparable or alternative therapy.
Trial website
https://clinicaltrials.gov/show/NCT03409081
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Astellas Pharma Global Development, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03409081