Solid TumorPhase 1NCT03975387

Study of ASTX295 in Patients With Solid Tumors With Wild-Type p53

A Phase 1 study testing ASTX295 in people with Solid Tumor, run by Taiho Oncology, Inc. at 11 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial summary

Study ASTX295-01 is a first in human Phase 1/2 open-label study of the safety, pharmacokinetics, and preliminary activity of ASTX295 in participants with wild-type TP53 advanced solid tumors. Phase 1 is a dose escalation and dose expansion study design. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Solid Tumor
What is being tested?: ASTX295
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 106 participants
Where: 11 sitesUnited States
Study dates: 11 Jul 2019to 15 Aug 2024
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

Age
Participant must be 18 years of age or older, at the time of signing the informed consent.
Type of Participant and Disease Characteristics
Have histologically or cytologically confirmed advanced solid tumors that are metastatic or unresectable and are refractory or have relapsed after treatment with standard available therapies or for whom standard life-prolonging measures are not available.
Phase 1: any tumor type is eligible
Phase 2: eligible tumor types as follows: malignant pleural mesothelioma (MPM) (Cohort 1); Liposarcoma (well-differentiated (WD) , de- differentiated (DD), or mix), intimal sarcoma, and other sarcomas with human murine double minute 2 (MDM2) amplification (Cohort 2); Glioblastoma multiforme (GBM) and tumors with CDNK2A loss of function (LOF) excluding MPM, liposarcoma, intimal sarcoma, and uveal melanoma (UVM) (Cohort 3); any solid tumors with molecular feature that may confer sensitivity to ASTX295 (Cohort 4); Uveal melanoma (Cohort 5); Any cancer type with MDM2 amplification excluding MPM, sarcoma, and UVM(Cohort 6).

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Documented wild-type TP53 and other molecular feature requirements.
Have an Eastern Cooperative Oncology Group (ECOG) Performance status (PS) of 0 to 2.
Acceptable bone marrow function, as evidenced by the following laboratory data:
Absolute neutrophil count (ANC) ≥1500 cells/mm3
Platelet count ≥100,000 cells/mm3
Hemoglobin >9 g/dL
Adequate hepatic function as evidenced by:
Serum total bilirubin ≤1.5 × upper limit of normal (ULN).
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × ULN (≤ 3 ULN in the presence of liver metastases).
Serum creatinine ≤1.5 × ULN OR calculated creatinine clearance (by the standard Cockcroft-Gault formula) of ≥50 mL/min or measured glomerular filtration rate of ≥50 mL/min.
Sex
Participant can be male or female
Informed Consent
Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol, and willing to participate in the study.
Participants are eligible to be included in Phase 1 Part B of the study only if all of the following additional criteria apply:
In Phase 1 Part B (dose expansion) of the protocol, subjects must have disease lesions that are amenable to biopsy and must agree and be able to undergo a pre- and on- treatment biopsy.
Participants are eligible to be included in Phase 2 of the study only if all of the following additional criteria apply:
Have sufficient tumor specimen either from archival formalin-fixed, paraffin embedded (FFPE) tissue or tissue obtained by a fresh biopsy for analyzing TP53 at a central laboratory.
Measurable disease according to appropriate criteria as per protocol.

Who cannot join

Medical Conditions
Poor medical risk in the investigator's opinion because of systemic diseases in addition to the cancer under study, for example, uncontrolled infections.
Life-threatening illness, significant organ system dysfunction, or other condition that, in the investigator's opinion, could compromise subject safety, or the integrity of study outcomes, or interfere with the absorption or metabolism of ASTX295.
History of, or at risk for, cardiac disease, as evidenced by any of the following conditions:
Abnormal left ventricular ejection fraction.
Congestive cardiac failure of ≥Grade 3.

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Unstable cardiac disease.
History or evidence at Screening of long QT interval corrected for heart rate (QTcF), ventricular arrhythmias, clinically significant bradyarrhythmias, 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 advanced human immunodeficiency virus (HIV) infection (including AIDS): clinical stage ≥ 3 according to WHO classification and/or HIV-associated immunodeficiency.
Known active hepatitis B virus (HBV) or active hepatitis C virus (HCV) infection (Inactive Hepatitis Carrier and subjects with laboratory evidence of no active replication on antivirals - viral load below limit of detection- will be permitted).
Known brain metastases, unless previously treated and clinically stable for at least 4 weeks with or without steroids.
Known significant mental illness or other conditions, such as active alcohol or other substance abuse that, in the opinion of the investigator, predispose the subject to high risk of noncompliance with the protocol treatment or assessments.
Prior/Concomitant Therapy
Prior anticancer treatments or therapies within the indicated time window prior to first dose of study treatment (ASTX295), as follows:
Cytotoxic chemotherapy within 3 weeks prior. Any encountered treatment-related toxicities (excepting alopecia) must be stabilized or resolved to ≤Grade 1.
Monoclonal antibodies, biologics, or immunotherapy within 4 weeks prior. Any encountered treatment-related toxicities must be stabilized or resolved to ≤Grade 1.
Molecularly targeted drug or other investigational drugs, without the potential for delayed toxicity, within 4 weeks of the first dose of study treatment or 5 half-lives (minimum 14 days), whichever is shorter. Any encountered treatment-related toxicities must be stabilized or resolved to ≤Grade 1.
Major surgery or radiation within 4 weeks prior to first dose (palliative radiotherapy to a single lesion within 2 weeks).
Prior treatment with MDM2 antagonist
Inability to swallow oral medication or inability or unwillingness to comply with the administration requirements related to ASTX295.
Participants are excluded from the Phase 2 part of the study if any of the following additional criteria apply:
Active malignancy other than the cancer under study (excludes low risk prostate cancer or early breast cancer with or without hormonal therapy, basal cell carcinoma of the skin and superficial bladder cancer).

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

United States11 sites

University of Michigan Rogel Cancer Center Site#109
Ann Arbor, Michigan, 48109
City of Hope Comprehensive Cancer Center Site#114
Duarte, California, 91010
Virgnia Cancer Specialists Site #103
Fairfax, Virginia, 22031
The University of Texas MD Anderson Cancer Center Site #102
Houston, Texas, 77030
Holden Comprehensive Cancer Center Site#108
Iowa City, Iowa, 52242
Cedars-Sinai Medical Center Site #105
Los Angeles, California, 90048
Columbia University Irving Medical Center - Herbert Irving Pavilion Site#104
New York, New York, 10032
Hoag Hospital Site#110
Newport Beach, California, 92663
University of Pennsylvania-Abramson Cancer Center Site#113
Philadelphia, Pennsylvania, 19104
Regions Cancer Center Site #115
Saint Paul, Minnesota, 55101
NEXT Oncology Site #101
San Antonio, Texas, 78229

Other trials in United States

About this study

Official title: Phase 1/2 Open-Label Study of the Safety, Pharmacokinetics, and Preliminary Activity of ASTX295 in Subjects With Wild-Type TP53 Advanced Solid Tumors

Conditions: Solid Tumor
Being tested: ASTX295
Keywords: solid tumor, mesothelioma, TP53
Medical subject headings: Mesothelioma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ASTX295Drug
ASTX295 orally for 28-day cycle continuous or on an intermittent dosing schedule.

Study groups in NCT03975387
Study group	Role	What they receive
ASTX295	Experimental	Not described Receives: Drug: ASTX295

What is being measured

The primary outcome is the main question the study is designed to answer.

Phase 1a: Safety and tolerability of ASTX295 including determination of maximum tolerated dose (MTD), and/or recommended dose for expansion (RDE) to Phase 1b
Measured: From the date of the first dose until 30 days after discontinuation of study treatment
Phase 1b: Recommended Phase 2 dose (RP2D) and regimen of ASTX295 to proceed to Phase 2
The RP2D will be based on incidence and severity of AEs, including SAEs and will be determined by the data and safety review committee (DSRC)
Measured: From the date of the first dose until 30 days after discontinuation of study treatment, an average of 6 months to 1 year
Phase 2: Disease control rate (DCR) in Cohort 1
DCR will be calculated in Cohort 1 as the number of subjects whose response at Week 16 is CR, partial response (PR), or stable disease, divided by the total number of subjects evaluable for DCR analysis.
Measured: From the date of the first dose until Week 16
Phase 2: Overall response rate (ORR) in Cohorts 2, 3, 4, 5, and 6
ORR in Cohorts 2, 3, 4, 5, and 6 will be calculated as the number of subjects whose best response is CR or PR, divided by the total number of subjects evaluable for ORR analysis.
Measured: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year

Secondary outcomes (11)

Phase 1: Preliminary clinical activity of ASTX295 as assessed by disease control rate (DCR)
DCR will be calculated as the number of subjects whose response at Week 16 is CR, PR, or stable disease, divided by the total number of subjects evaluable for DCR analysis
Measured: From the date of the first dose until Week 16
Phase 1: Preliminary clinical activity as assessed by objective response rate (ORR) of ASTX295
ORR will be calculated as the number of subjects whose best response is CR or PR, divided by the total number of subjects evaluable for ORR analysis
Measured: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year
Phase 2: Safety profile of ASTX295
Incidence and severity of adverse events (AEs) including serious adverse events (SAEs)
Measured: From the date of the first dose until 30 days after discontinuation of study treatment, an average of 6 months to 1 year
Phase 2: Progression free survival (PFS)
PFS is defined as the time from date of the first dose until the earliest date of disease progression or death from any cause, whichever comes first
Measured: Up to approximately 1 year
Phase 2: Overall survival (OS)
OS is defined as the time from the date of first dose to date of death due to any cause
Measured: Up to approximately 1 year
Phase 2: Overall response rate (ORR) in Cohort 1
ORR in Cohort 1 will be calculated as the number of subjects whose best response is CR or PR, divided by the total number of subjects evaluable for ORR analysis.
Measured: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year
Pharmacokinetic (PK) profile of ASTX295 (area under the curve [AUC])
Measured: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (minimum concentration [Cmin])
Measured: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (maximum concentration [Cmax])
Measured: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (time to reach maximum concentration [Tmax])
Measured: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (elimination half-life [t½])
Measured: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

11 Jul 201915 Aug 2024

First posted5 Jun 2019
Study start11 Jul 2019
Primary completion11 Sep 2023
Study completion15 Aug 2024

Why it stopped: Sponsor Decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Solid TumorStudies of the same condition, open and under way.
More trials run by Taiho Oncology, Inc.The sponsor's other registered studies.
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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 106 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03975387
Sponsor's protocol ID: ASTX295-01
Other IDs: 2021-005033-16
Status last verified: Apr 2025
First submitted: 28 May 2019
Last updated: 9 Apr 2025
Results posted: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
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A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
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A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Relapsed Solid Tumor Malignancies, Refractory Solid Tumor Malignancies
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Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
Malignant Solid Neoplasm

All Solid Tumor trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 1/2 Open-Label Study of the Safety, Pharmacokinetics, and Preliminary Activity of ASTX295 in Subjects With Wild-Type TP53 Advanced Solid Tumors
ClinicalTrials.gov ID: NCT03975387
Acronym / sponsor ID: ASTX295-01
Conditions: Solid Tumor
Keywords: solid tumor, mesothelioma, TP53
Phase: Phase 1
Study type: Interventional
Recruitment status: TerminatedReason stopped: Sponsor Decision
Enrolment: 106 participants (actual)
Lead sponsor: Taiho Oncology, Inc.Industry (pharma, biotech, devices)
Study start: 11 Jul 2019 (actual)
Primary completion: 11 Sep 2023 (actual)
Study completion: 15 Aug 2024 (actual)
First posted: 5 Jun 2019 (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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
ASTX295	Experimental	Drug: ASTX295

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (25 listed)

Age
Participant must be 18 years of age or older, at the time of signing the informed consent.
Type of Participant and Disease Characteristics
Have histologically or cytologically confirmed advanced solid tumors that are metastatic or unresectable and are refractory or have relapsed after treatment with standard available therapies or for whom standard life-prolonging measures are not available.
Documented wild-type TP53 and other molecular feature requirements.

Key exclusion criteria (23 listed)

Medical Conditions
Poor medical risk in the investigator's opinion because of systemic diseases in addition to the cancer under study, for example, uncontrolled infections.
Life-threatening illness, significant organ system dysfunction, or other condition that, in the investigator's opinion, could compromise subject safety, or the integrity of study outcomes, or interfere with the absorption or metabolism of ASTX295.
History of, or at risk for, cardiac disease, as evidenced by any of the following conditions:
Known advanced human immunodeficiency virus (HIV) infection (including AIDS): clinical stage ≥ 3 according to WHO classification and/or HIV-associated immunodeficiency.

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
ASTX295ASTX295 orally for 28-day cycle continuous or on an intermittent dosing schedule.	Drug	ASTX295

Primary outcome

Phase 1a: Safety and tolerability of ASTX295 including determination of maximum tolerated dose (MTD), and/or recommended dose for expansion (RDE) to Phase 1b
Time frame: From the date of the first dose until 30 days after discontinuation of study treatment
Phase 1b: Recommended Phase 2 dose (RP2D) and regimen of ASTX295 to proceed to Phase 2
Time frame: From the date of the first dose until 30 days after discontinuation of study treatment, an average of 6 months to 1 year
The RP2D will be based on incidence and severity of AEs, including SAEs and will be determined by the data and safety review committee (DSRC)
Phase 2: Disease control rate (DCR) in Cohort 1
Time frame: From the date of the first dose until Week 16
DCR will be calculated in Cohort 1 as the number of subjects whose response at Week 16 is CR, partial response (PR), or stable disease, divided by the total number of subjects evaluable for DCR analysis.
Phase 2: Overall response rate (ORR) in Cohorts 2, 3, 4, 5, and 6
Time frame: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year
ORR in Cohorts 2, 3, 4, 5, and 6 will be calculated as the number of subjects whose best response is CR or PR, divided by the total number of subjects evaluable for ORR analysis.

11 secondary outcomes

Phase 1: Preliminary clinical activity of ASTX295 as assessed by disease control rate (DCR)
Time frame: From the date of the first dose until Week 16
Phase 1: Preliminary clinical activity as assessed by objective response rate (ORR) of ASTX295
Time frame: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year
Phase 2: Safety profile of ASTX295
Time frame: From the date of the first dose until 30 days after discontinuation of study treatment, an average of 6 months to 1 year
Phase 2: Progression free survival (PFS)
Time frame: Up to approximately 1 year
Phase 2: Overall survival (OS)
Time frame: Up to approximately 1 year
Phase 2: Overall response rate (ORR) in Cohort 1
Time frame: From the date of the first dose until study treatment discontinuation, an average of 6 months to 1 year
Pharmacokinetic (PK) profile of ASTX295 (area under the curve [AUC])
Time frame: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (minimum concentration [Cmin])
Time frame: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (maximum concentration [Cmax])
Time frame: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (time to reach maximum concentration [Tmax])
Time frame: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)
Pharmacokinetic (PK) profile of ASTX295 (elimination half-life [t½])
Time frame: Blood will be collected during Cycles 1 and 2 in Phase 1, Cycles 1 and 3 in Phase 2 (each cycle is 28 days)

Results and safety

The study was stopped early (reason given: Sponsor Decision). Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2025.

Related trials for Solid Tumor

RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
Active, not recruitingPhase 1
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
Active, not recruitingPhase 3
A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Relapsed Solid Tumor Malignancies, Refractory Solid Tumor Malignancies
RecruitingObservational study
Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
Malignant Solid Neoplasm

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.

11 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing ASTX295 in people with Solid Tumor, run by Taiho Oncology, Inc. at 11 sites 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 23 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 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites 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.

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: Sponsor Decision). 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?

Phase 1a: Safety and tolerability of ASTX295 including determination of maximum tolerated dose (MTD), and/or recommended dose for expansion (RDE) to Phase 1b (time frame: From the date of the first dose until 30 days after discontinuation of study treatment). 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?

On the ClinicalTrials.gov record for NCT03975387.

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 9 Apr 2025.