Individual Patient Expanded Access Gilteritinib (ASP2215)

NCT03315299 | Unknown

• 1 other identifier: CHLA-17-00428
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to provide expanded access to ASP2215 for a single subject with refractory FLT3-mutated AML without access to comparable or alternative therapy.

Conditions

AML

Interventions

ASP2215 DRUG

Subject will take ASP2215 by mouth daily for 28 day cycles

Also known as: gilteritinib

Eligibility Criteria

• Age: 12 Years - 15 Years
• Gender Eligibility Details: Single patient.
• Age Groups: Child (0-17)

You may qualify if:

• Institutional Review Board-/Independent Ethics Committee-approved written Informed Consent and privacy language as per national regulation (e.g., Health Insurance Portability and Accountability Act authorization for United States sites) must be obtained from the subject or legally authorized representative prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).
• Subject has a diagnosis of primary AML or AML secondary to myelodysplastic syndrome (MDS) or therapy-related AML according to World Health Organization classification \[Swerdlow et al., 2008\] as determined by pathology review at the treating institution.
• Subject has presence of the FLT3 mutation (internal tandem duplication \[ITD\] and/or tyrosine kinase domain \[TKD\] \[D835/I836\] mutation) in bone marrow or peripheral blood as determined by local laboratory.
• Subject has refractory or relapsed AML (with or without hematopoietic stem cell transplant \[HSCT\]) or AML in complete remission (CR) with minimal residual disease by flow cytometry or genetic testing after induction/consolidation regimen.
• In the judgment of the investigator, there is no comparable or satisfactory alternative therapy to treat the subject's AML.
• Subject has not received any chemotherapy or investigational agent within at least 5 half-lives after stopping that drug and before starting gilteritinib.
• Subject must meet the following criteria as indicated on clinical laboratory tests:
• Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x 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 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 randomization
• 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:

You may not qualify if:

• Subject will be excluded from participation if any of the following apply:
• Subject is eligible for enrollment in another ongoing clinical study of gilteritinib.
• Subject has participated in a previous or ongoing open-label, randomized treatment study of gilteritinib.
• Subject with Fridericia-corrected QT interval (QTcF) \> 450 ms at screening based on local reading.
• Subject with Long QT Syndrome at screening.
• Subject was diagnosed as 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 in the opinion of the investigator.
• 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 requires treatment with concomitant drugs that are strong inhibitors or inducers of P-gp with the exception of drugs that are considered absolutely essential for the care of the subject.
• Subject requires treatment with concomitant drugs that target serotonin 5HT1R or 5HT2BR or sigma nonspecific receptor with the exception of drugs that are considered absolutely essential for the care of the subject.
• Subject has any condition which, in the investigator's opinion, makes the subject unsuitable for study participation.

Sponsors & Collaborators

• Etan Orgel: lead
• Astellas Pharma Global Development, Inc.: collaborator

Study Sites (1)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

MeSH Terms

Interventions

gilteritinib

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 16, 2017
• First Posted: October 20, 2017
• Last Updated: February 7, 2024

Record last verified: 2024-02

Locations

US (1)