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)

NCT03409081 | Unknown

• 1 other identifier: 2215-CL-9200
• Study Type: expanded_access
• Target: N/A
• Locations: 3 countries
• Sites: 6

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.

Conditions

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

Keywords

Expanded Access; FMS-like Tyrosine Kinase 3; Acute Myeloid Leukemia (AML); gilteritinib; ASP2215

Interventions

gilteritinib DRUG

oral

Also known as: ASP2215

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:

You may not qualify if:

• 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.

Sponsors & Collaborators

• Astellas Pharma Global Development, Inc.: lead

Study Sites (6)

Site AU61001

Canberra, Australia

Location

Site IT39001

Milan, Italy

Location

Site IT39003

Milan, Italy

Location

Site UK44001

Birmingham, United Kingdom

Location

Site UK44003

Bristol, United Kingdom

Location

Site UK44002

London, United Kingdom

Location

Related Publications (1)

• Dogu MH, Tekgunduz AIE, Deveci B, Korkmaz G, Comert M, Sevindik OG, Yokus O, Serin I. Gilteritinib (XOSPATA(R)) in Turkey: Early Access Program Results. Mediterr J Hematol Infect Dis. 2023 May 1;15(1):e2023031. doi: 10.4084/MJHID.2023.031. eCollection 2023.

  PMID: 37180209 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

gilteritinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Study Director

  Astellas Pharma Global Development, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2018
• First Posted: January 24, 2018
• Last Updated: September 13, 2018

Record last verified: 2018-09

Locations

GB (3); AU (1); IT (2)