AMLNCT03315299
Individual Patient Expanded Access Gilteritinib (ASP2215)
A study testing ASP2215 in people with AML, run by Etan Orgel at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 15 years, women only
- Conditions the study is for
- AML
- What is being tested?
- ASP2215
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 15 years
- Women only
- Child
Who may be able to join
- 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.
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- 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:
- Be of nonchildbearing potential:
- Postmenopausal (defined as at least 1 year without any menses) prior to screening, or
- Documented surgically sterile or status posthysterectomy (at least 1 month prior to screening)
- Or, if of childbearing potential,
- Agree not to try to become pregnant during the study and for 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 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 180 days after the final study drug administration.
- Subject agrees not to participate in another interventional study while on treatment.
- Subject who has a diagnosis of human immunodeficiency virus (HIV) may be enrolled as long as their disease is under control on antiretroviral therapy. Precautions should be taken to modify their highly active antiretroviral therapy (HAART) regimen to minimize drug interactions.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Los Angeles
Los Angeles, California, 90027
About this study
Official title: Individual Patient Expanded Access for Use of Gilteritinib (ASP2215) for Patient Y.A.
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.
Read the detailed description
This treatment protocol is being conducted in a single pediatric patient while phase 3 ASP2215 studies are ongoing in adult subjects with FLT3-mutated AML.
The subjects will enter the screening period up to 2 weeks prior to the start of treatment.
The subject will be administered treatment over 28-day cycles.
The subjects will complete visits on cycle 1 days 1, 4, 8, 15; cycle 2 days 1, 15, and day 1 of each cycle thereafter until discontinued from the study for toxicity, disease progression, or lack of continued benefit in the judgement of the investigator.
An end of treatment visit will be performed within 7 days after last dose of the investigation product (ASP2215), or prior to initiation of another anticancer therapy, whichever occurs earlier, followed by a 30-day follow up.
- Conditions
- AML
- Being tested
- ASP2215
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASP2215Drug
Also called gilteritinib
Subject will take ASP2215 by mouth daily for 28 day cycles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMLStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Etan Orgel
- Sponsor type
- University, hospital or other
- Collaborators
- Astellas Pharma Global Development, Inc.
- Responsible party
- Sponsor-investigator, Etan Orgel, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03315299
- Sponsor's protocol ID
- CHLA-17-00428
- Status last verified
- Feb 2024
- First submitted
- 16 Oct 2017
- Last updated
- 7 Feb 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individual Patient Expanded Access for Use of Gilteritinib (ASP2215) for Patient Y.A.
- ClinicalTrials.gov ID
- NCT03315299
- Acronym / sponsor ID
- CHLA-17-00428
- Conditions
- AML
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Etan OrgelUniversity, hospital or other
- Collaborators
- Astellas Pharma Global Development, Inc.
- First posted
- 20 Oct 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This treatment protocol is being conducted in a single pediatric patient while phase 3 ASP2215 studies are ongoing in adult subjects with FLT3-mutated AML.
The subjects will enter the screening period up to 2 weeks prior to the start of treatment.
The subject will be administered treatment over 28-day cycles.
The subjects will complete visits on cycle 1 days 1, 4, 8, 15; cycle 2 days 1, 15, and day 1 of each cycle thereafter until discontinued from the study for toxicity, disease progression, or lack of continued benefit in the judgement of the investigator.
An end of treatment visit will be performed within 7 days after last dose of the investigation product (ASP2215), or prior to initiation of another anticancer therapy, whichever occurs earlier, followed by a 30-day follow up.
Study design
Who may qualify
- Ages
- 12 to 15 years
- Sex
- Female only
- Age groups
- Child
Key inclusion criteria (25 listed)
- 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.
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ASP2215Also: gilteritinibSubject will take ASP2215 by mouth daily for 28 day cycles | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ASP2215 in people with AML, run by Etan Orgel at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Feb 2024.