Acute Myeloid LeukemiaPhase 1NCT04637009
A Study of TAS1553 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms
A Phase 1 study testing TAS1553 in people with Acute Myeloid Leukemia and Myeloproliferative Neoplasm, run by Astex Pharmaceuticals, Inc. at 10 sites in 2 countries.
Trial summary
This is a Phase 1, 2-part, open-label, multicenter, first-in-human (FIH) study to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TAS1553 administered orally to participants ≥18 years of age with relapsed or refractory (R/R) acute myeloid leukemia (AML) or other… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myeloproliferative Neoplasm, Myelodysplastic/Myeloproliferative Neoplasm
- What is being tested?
- TAS1553
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 21 Dec 2020to 20 Feb 2023
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Capable of giving signed informed consent.
- Participant must be 18 years of age or older, at the time of signing the informed consent.
- Life expectancy of at least 12 weeks as assessed by the investigator.
- Participants with R/R AML or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and MDS transformed into AML. Other myeloid neoplasms include accelerated phase MPN, and chronic or accelerated phase MPN-U and MDS-MPN. Blast crisis phase of MPN, MPN-U, and MDS-MPN are considered secondary AML and will be included in the AML cohort.
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Have platelet count ≥10,000/μL (transfusions to achieve this level are allowed).
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- Have adequate renal function as demonstrated by a 24-hour urine measured creatinine clearance ≥60 mL/min.
- Adequate hepatic function as evidenced by:
- aspartate aminotransferase (AST) ≤3× upper limit of normal (ULN)
- alanine aminotransferase (ALT) ≤3×ULN
- total bilirubin ≤1.5×ULN.
- Participants must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study.
- Women of child-bearing potential (according to recommendations of the Clinical Trial Facilitation Group [CTFG]) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening.
Who cannot join
- Participants who have MPN, MPN-U, or MDS/MPN and display hypoplastic bone marrow and would also not ordinarily benefit from cytoreductive therapy such as hydroxyurea (HU).
- Participants with highly proliferative disease are excluded as follows:
- Part 1/AML: white blood cells (WBC) >20,000/μL and >50% blasts in blood. Measures to reduce WBC, such as HU treatment within the last 2 weeks and cytotoxic chemotherapy within the last 4 weeks are not allowed to meet this eligibility criterion.
- Part 1/other myeloid neoplasms: WBC >20,000/μL. A short course of HU may be used to meet this eligibility criterion, as long as HU is discontinued 96 hours and any encountered drug-related toxicity must be resolved to Grade ≤1 before the first dose of study treatment.
- Part 2/Cohort 1, AML: WBC>20,000/μL and >50% blasts in blood. A short course of HU may be used to meet this eligibility criterion, as long as HU is discontinued 96 hours, and any encountered drug-related toxicity must be resolved to Grade ≤1 before the first dose of study treatment.
- Part 2/Cohort 2, other myeloid neoplasms: Specific WBC exclusion criterion not defined. A short course of HU may be used to reduce WBC if judged to be necessary by the investigator, as long as HU is discontinued 96 hours and any encountered drug-related toxicity must be resolved to Grade ≤1 before the first dose of study treatment.
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- Known clinically active central nervous system (CNS) leukemia.
- Diagnosis of BCR-ABL-positive leukemia, acute promyelocytic leukemia (M3 AML or APML), or juvenile myelomonocytic leukemia (JMML).
- Second malignancy requiring active systemic therapy, except breast or prostate cancer stable on or responding to endocrine therapy.
- Ongoing Grade ≥3 Graft Versus Host Disease (GVHD), or any grade GVHD requiring active treatment (for example, calcineurin inhibitors, ≥5mg/day prednisone or other steroid equivalent, or other immunosuppressive agents). (Note: Prednisone at any dose for other indications is allowed).
- Advanced human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection; Inactive hepatitis carrier status and participants with laboratory evidence of no active replication and participants on antiviral medication(s) who have a viral load below limit of detection will be permitted.
- Known significant mental illness or other condition such as active alcohol or other substance abuse or addiction that, in the opinion of the investigator, predisposes the participant to high risk of non-compliance with the protocol.
- Active infection resistant to antibiotics; or non-leukemia-associated pulmonary disease requiring >2 liters per minute oxygen or any other condition that puts the participant at an imminent risk of death.
- 24-hour urinary protein excretion ≥1g or urinalysis of 2+proteinuria.
- History of, or at risk for, cardiac disease, as evidenced by any of the following conditions:
- Abnormal left ventricular ejection fraction (LVEF; <50%) on echocardiogram (ECHO) or multiple-gated acquisition (MUGA) scan at Screening.
- Congestive cardiac failure of Class ≥III severity according to New York Heart Association (NYHA) functional classification defined as patients with marked limitation of activity and who are comfortable at rest, while Class IV patients have symptoms of heart failure at rest.
- Unstable cardiac disease including unstable angina or hypertension as defined by the need for overnight hospital admission within the last 3 months (90 days).
- Ventricular arrhythmias including ventricular bigeminy, clinically significant brady arrhythmias such as sick sinus syndrome, third-degree atrioventricular (AV) block, presence of cardiac pacemaker or defibrillator, or other clinically significant arrhythmias.
- Screening 12-lead electrocardiogram (ECG) with measurable QTcF interval of ≥470 msec (Fridericia's formula should be used).
- Known hypersensitivity to TAS1553 or any of its components.
- Allogenic hematopoietic stem cell transplantation (HSCT) within 180 days of the first dose of TAS1553, or participants on immunosuppressive therapy post HSCT at the time of screening (calcineurin inhibitors or similar must be discontinued ≥4 weeks prior to the time of study drug initiation).
- Treated with any systemic anticancer therapy within 2 weeks of the first dose of study treatment. Any encountered treatment-related toxicities (excepting alopecia) must be resolved to Grade 1 or less.
- Phase 1 Part 1 only: participants who require concomitant use of strong CYP3A4 inducers.
- Inability to swallow oral medication.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States8 sites
Augusta University - Georgia Cancer Center
Augusta, Georgia, 30912
University of Alabama - Birmingham Comprehensive Cancer Center
Birmingham, Alabama, 35233
Cleveland Clinic Taussig Cancer Institute
Cleveland, Ohio, 44195
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
University of Southern California Keck School of Medicine
Los Angeles, California, 90033
Norton Cancer Institute
Louisville, Kentucky, 40241
Weill Cornell Medicine and New York - Presbyterian Hospital
New York, New York, 10065
HonorHealth Research Institute
Scottsdale, Arizona, 82528
Canada2 sites
University of Alberta Hospital - Hematology Research
Edmonton, Alberta, T6G 2V2
Princess Margaret Hospital
Toronto, Ontario, M5G 2L7
About this study
Official title: A Phase 1 Study of Safety, Pharmacokinetics and Preliminary Activity of TAS1553 in Subjects With Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms
This is a Phase 1, 2-part, open-label, multicenter, first-in-human (FIH) study to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TAS1553 administered orally to participants ≥18 years of age with relapsed or refractory (R/R) acute myeloid leukemia (AML) or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and myelodysplastic syndrome (MDS)-transformed into AML. Other myeloid neoplasms include accelerated phase myeloproliferative neoplasms (MPN), and chronic or accelerated phase MPN-unclassifiable (MPN-U) and MDS-MPN. Blast crisis phase of MPNs are considered secondary AML and will be included in the AML cohort.
Part 1 is a multicenter, sequential group treatment feasibility study with 1 treatment arm and no masking (dose escalation). Part 2 is a multicenter, two-stage, multiple group, dose confirmation study with 1 treatment arm and no masking (exploratory dose expansion).
- Conditions
- Acute Myeloid Leukemia, Myeloproliferative Neoplasm, Myelodysplastic/Myeloproliferative Neoplasm
- Being tested
- TAS1553
- Keywords
- MDS/MPN, unclassifiable in chronic phase, Accelerated phase MPN, Chronic neutrophilic leukemia (CNL), accelerated phase, Polycythemia vera (PV), accelerated phase, Primary myelofibrosis (PMF), accelerated phase, Essential thrombocythemia (ET), accelerated phase, Chronic eosinophilic leukemia, not otherwise specified (NOS), accelerated phase, MPN, unclassifiable, MDS/MPN, Chronic myelomonocytic leukemia (CMML), Atypical chronic myeloid leukemia (aCML), BCR-ABL1-negative, MDS/MPN with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), MDS/MPN, unclassifiable
- Medical subject headings
- Leukemia, Myeloid, Acute, Myeloproliferative Disorders, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Neutrophilic, Chronic, Polycythemia Vera, Primary Myelofibrosis, Thrombocythemia, Essential, Pdgfra-Associated Chronic Eosinophilic Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TAS1553Drug
Form: tablet; Route of Administration: oral
| Study group | Role | What they receive |
|---|---|---|
| Part 1 (dose escalation) | Experimental | Oral administration of TAS1553 once daily at specific time points. Receives: Drug: TAS1553 |
| Part 2 (dose expansion) | Experimental | Oral administration of TAS1553 once daily at specific time points. Receives: Drug: TAS1553 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Number of participants with treatment-emergent adverse events in Part 1
Measured: Up to 12 months
Safety: Number of participants with dose-limiting toxicities in Part 1
Measured: Up to 12 months
Response rate in Cohort 1 (AML): Number of participants with complete response (CR) + complete response with partial hematological recovery (CRh), and with CR + incomplete blood count recovery (CRi) in Part 2
Measured: Up to 33 months
Response rate in Cohort 2 (other myeloid neoplasms): Number of participants with overall response rate (ORR) of CR + partial response (PR) in Part 2
Measured: Up to 33 months
Secondary outcomes (12)
Pharmacokinetic parameter: Area under the curve (AUC)
Measured: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Maximum plasma concentration (Cmax)
Measured: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Minimum plasma concentration (Cmin)
Measured: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Time to reach maximum plasma concentration (Tmax)
Measured: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Half-life (t½)
Measured: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Hematological improvement: Number of participants in Cohort 2 (other myeloid neoplasms) with hematological improvement in Part 2
Measured: Up to 33 months
Time to response (TTR): Number of days from the first dose to the first documented evidence of response
Measured: Up to 33 months
Duration of response (DOR): Number of days from the start of response until disease progression or relapse
Measured: Up to 33 months
Overall survival (OS): Number of days from date of first dose until death due to any cause
Measured: Up to 33 months
Safety: Number of participants with treatment-emergent adverse events in Part 2
Measured: Up to 33 months
Pharmacodynamic biomarker: Change from baseline in deoxyadenosine triphosphate (dATP) pool levels in peripheral blood mononuclear cells (PBMCs)
Measured: At specific timepoints from predose up to Day 2 of Cycle 2 (28 days per cycle)
Pharmacodynamic biomarker: Change from baseline in phosphorylated checkpoint kinase 1 (pCHK1) levels in bone marrow
Measured: At specific timepoints from predose up to Day 2 of Cycle 2 (28 days per cycle)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2020
- Study start21 Dec 2020
- Primary completion20 Feb 2023
- Study completion20 Feb 2023
Why it stopped: termination due to portfolio prioritization
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astex Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04637009
- Sponsor's protocol ID
- TAS1553-01
- Status last verified
- Aug 2024
- First submitted
- 15 Nov 2020
- Last updated
- 2 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of Safety, Pharmacokinetics and Preliminary Activity of TAS1553 in Subjects With Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms
- ClinicalTrials.gov ID
- NCT04637009
- Acronym / sponsor ID
- TAS1553-01
- Conditions
- Acute Myeloid Leukemia, Myeloproliferative Neoplasm, Myelodysplastic/Myeloproliferative Neoplasm
- Keywords
- MDS/MPN, unclassifiable in chronic phase, Accelerated phase MPN, Chronic neutrophilic leukemia (CNL), accelerated phase, Polycythemia vera (PV), accelerated phase, Primary myelofibrosis (PMF), accelerated phase, Essential thrombocythemia (ET), accelerated phase, Chronic eosinophilic leukemia, not otherwise specified (NOS), accelerated phase, MPN, unclassifiable
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: termination due to portfolio prioritization
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Astex Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Dec 2020 (actual)
- Primary completion
- 20 Feb 2023 (actual)
- Study completion
- 20 Feb 2023 (actual)
- First posted
- 19 Nov 2020 (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.
This is a Phase 1, 2-part, open-label, multicenter, first-in-human (FIH) study to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TAS1553 administered orally to participants ≥18 years of age with relapsed or refractory (R/R) acute myeloid leukemia (AML) or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and myelodysplastic syndrome (MDS)-transformed into AML. Other myeloid neoplasms include accelerated phase myeloproliferative neoplasms (MPN), and chronic or accelerated phase MPN-unclassifiable (MPN-U) and MDS-MPN. Blast crisis phase of MPNs are considered secondary AML and will be included in the AML cohort.
Part 1 is a multicenter, sequential group treatment feasibility study with 1 treatment arm and no masking (dose escalation). Part 2 is a multicenter, two-stage, multiple group, dose confirmation study with 1 treatment arm and no masking (exploratory dose expansion).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1 (dose escalation) | Experimental | Drug: TAS1553Oral administration of TAS1553 once daily at specific time points. |
| Part 2 (dose expansion) | Experimental | Drug: TAS1553Oral administration of TAS1553 once daily at specific time points. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Capable of giving signed informed consent.
- Participant must be 18 years of age or older, at the time of signing the informed consent.
- Life expectancy of at least 12 weeks as assessed by the investigator.
- Participants with R/R AML or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and MDS transformed into AML. Other myeloid neoplasms include accelerated phase MPN, and chronic or accelerated phase MPN-U and MDS-MPN. Blast crisis phase of MPN, MPN-U, and MDS-MPN are considered secondary AML and will be included in the AML cohort.
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
Key exclusion criteria (25 listed)
- Participants who have MPN, MPN-U, or MDS/MPN and display hypoplastic bone marrow and would also not ordinarily benefit from cytoreductive therapy such as hydroxyurea (HU).
- Participants with highly proliferative disease are excluded as follows:
- Known clinically active central nervous system (CNS) leukemia.
- Diagnosis of BCR-ABL-positive leukemia, acute promyelocytic leukemia (M3 AML or APML), or juvenile myelomonocytic leukemia (JMML).
- Second malignancy requiring active systemic therapy, except breast or prostate cancer stable on or responding to endocrine therapy.
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 |
|---|---|---|
| TAS1553Form: tablet; Route of Administration: oral | Drug | Part 1 (dose escalation), Part 2 (dose expansion) |
Primary outcome
Safety: Number of participants with treatment-emergent adverse events in Part 1
Time frame: Up to 12 months
Safety: Number of participants with dose-limiting toxicities in Part 1
Time frame: Up to 12 months
Response rate in Cohort 1 (AML): Number of participants with complete response (CR) + complete response with partial hematological recovery (CRh), and with CR + incomplete blood count recovery (CRi) in Part 2
Time frame: Up to 33 months
Response rate in Cohort 2 (other myeloid neoplasms): Number of participants with overall response rate (ORR) of CR + partial response (PR) in Part 2
Time frame: Up to 33 months
12 secondary outcomes
Pharmacokinetic parameter: Area under the curve (AUC)
Time frame: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Maximum plasma concentration (Cmax)
Time frame: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Minimum plasma concentration (Cmin)
Time frame: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Time to reach maximum plasma concentration (Tmax)
Time frame: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Pharmacokinetic parameter: Half-life (t½)
Time frame: At specific timepoints from predose up to Day 8 of Cycle 6 (28 days per cycle)
Hematological improvement: Number of participants in Cohort 2 (other myeloid neoplasms) with hematological improvement in Part 2
Time frame: Up to 33 months
Time to response (TTR): Number of days from the first dose to the first documented evidence of response
Time frame: Up to 33 months
Duration of response (DOR): Number of days from the start of response until disease progression or relapse
Time frame: Up to 33 months
Overall survival (OS): Number of days from date of first dose until death due to any cause
Time frame: Up to 33 months
Safety: Number of participants with treatment-emergent adverse events in Part 2
Time frame: Up to 33 months
Pharmacodynamic biomarker: Change from baseline in deoxyadenosine triphosphate (dATP) pool levels in peripheral blood mononuclear cells (PBMCs)
Time frame: At specific timepoints from predose up to Day 2 of Cycle 2 (28 days per cycle)
Pharmacodynamic biomarker: Change from baseline in phosphorylated checkpoint kinase 1 (pCHK1) levels in bone marrow
Time frame: At specific timepoints from predose up to Day 2 of Cycle 2 (28 days per cycle)
Results and safety
The study was stopped early (reason given: termination due to portfolio prioritization). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2024.
- Individual participant dataSharing planned: No
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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.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TAS1553 in people with Acute Myeloid Leukemia and Myeloproliferative Neoplasm, run by Astex Pharmaceuticals, Inc. at 10 sites in 2 countries.
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 13 inclusion and 25 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: stopped early, as of the sponsor's last update on 2 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: termination due to portfolio prioritization). 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.
What does the primary outcome measure?
Safety: Number of participants with treatment-emergent adverse events in Part 1 (time frame: Up to 12 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 2 Aug 2024.