Study of ASTX029 in Subjects With Advanced Solid Tumors

NCT ID: NCT03520075

Last Updated: 2025-07-03

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

192 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-05-07

Study Completion Date

2025-03-03

Brief Summary

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This study is a first-in-human, open-label, multicenter, Phase 1-2 study to assess the safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of ASTX029 administered orally to participants with advanced solid malignancies who are not candidates for approved or available therapies.

Detailed Description

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ASTX029 is a synthetic small molecule inhibitor of extracellular signal-regulated kinases (ERKs) 1/2. ASTX029 has not been previously evaluated in human participants. The Phase 1 portion of this study will assess safety and determine the maximum tolerated dose, the recommended Phase 2 dose (RP2D), and the recommended dosing regimen of ASTX029 administered orally. The Phase 2 portion will assess preliminary clinical activity in tumors characterized by gene aberrations in the mitogen-activated protein kinase (MAPK) signal pathway that may confer sensitivity to ASTX029.

Conditions

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Solid Tumor, Adult

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1A: Cohort 1 Dose Escalation

Participants received ASTX029 10 milligrams (mg), powder in bottle (PiB), orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

PiB

Phase 1A: Cohort 2 Dose Escalation

Participants received ASTX029 20 mg, PiB, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

PiB

Phase 1A: Cohort 3 Dose Escalation

Participants received ASTX029 60 mg, PiB, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

PiB

Phase 1A: Cohort 4 Dose Escalation

Participants received ASTX029 120 mg, PiB, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

PiB

Phase 1A: Cohort 5 Dose Escalation

Participants received ASTX029 200 mg, orally, PiB, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

PiB

Phase 1A: Cohort 6 Dose Escalation

Participants received ASTX029 80 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 7 Dose Escalation

Participants received ASTX029 120 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fed state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 8 Dose Escalation

Participants received ASTX029 40 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 9 Dose Escalation

Participants received ASTX029 80 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 10 Dose Escalation

Participants received ASTX029 120 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 11 Dose Escalation

Participants received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1A: Cohort 12 Dose Escalation

Participants received ASTX029 280 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 1B Dose Expansion

Participants received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months, under fasted state.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort A

Participants with neuroblastoma RAS (NRAS)-mutant melanoma received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort B

Participants with Kirsten RAS (KRAS)-mutant or KRAS-amplified non-small cell lung cancer (NSCLC) received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort C

Participants with B isoform of RAF kinase (BRAF) V600-mutant cancers (non-colorectal cancers) received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort D

Participants with BRAF-fusion cancers received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort E

Participants with gynecological cancers with alterations in the mitogen-activated protein kinase (MAPK) pathway received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Phase 2: Cohort F

Participants with tumors that were characterized by other gene aberrations (that upregulate the MAPK signal pathway) received ASTX029 200 mg, tablets, orally, once daily, on Days 1-21 of each 21-day cycle, up to median duration of 1.6 months.

Group Type EXPERIMENTAL

ASTX029

Intervention Type DRUG

Tablet

Interventions

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ASTX029

PiB

Intervention Type DRUG

ASTX029

Tablet

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Able to understand and comply with study procedures, understand the risks involved in the study, and provide written informed consent before any study-specific procedure is performed.
2. Men or women 18 years of age or older.
3. Participants with histologically or cytologically confirmed advanced solid tumors that are metastatic or unresectable, who are refractory or have relapsed after treatment with available therapies or for whom standard life-prolonging measures or approved therapies are not available. In Phase 1 Part B and in the Phase 2 portion of the protocol, participants must also have documented gene alterations in the MAPK pathway as detailed in the protocol.
4. In Phase 1 Part B of the protocol, participants must have disease lesions that are amenable to biopsy.
5. In the Phase 2 portion of the protocol, participants must have measurable disease according to RECIST v1.1.
6. Eastern Cooperative Oncology Group performance status 0 to 2.
7. Acceptable organ function as evidenced by the following laboratory data:

1. Aspartate aminotransferase (AST) and alanine aminotransferase ≤2×upper limit of normal (ULN) or ≤3 ULN in the presence of liver metastases.
2. Total serum bilirubin ≤1.5×ULN.
3. Absolute neutrophil count (ANC) ≥1500 cells/mm3.
4. Platelet count ≥100,000 cells/mm3.
5. Calculated creatinine clearance (by the standard Cockcroft Gault formula) of ≥50 mL/min or glomerular filtration rate of ≥50 mL/min.
8. Women of child-bearing potential (according to recommendations of the Clinical Trial Facilitation Group \[CTFG\]; see protocol for details) must not be pregnant or breastfeeding and must have a negative pregnancy test within 24 hours before the first dose of study treatment. While receiving study treatment and for at least 5 half-lives of ASTX029 or metabolite plus 30 days after completing treatment, women of child-bearing potential must agree to practice highly effective contraceptive measures (as described in the protocol) and must refrain from donating eggs (ova, oocytes) for the purpose of reproduction.
9. Men with female partners of child-bearing potential (according to recommendations of the CTFG; see protocol for details) must agree to, during the treatment period and for at least 5 half-lives of ASTX029 or metabolite plus 90 days after completing treatment, practice highly effective contraceptive measures (as described in the protocol), not to father a child, and to refrain from donating sperm.

Exclusion Criteria

1. Hypersensitivity to ASTX029 or excipients of the drug product.
2. Poor medical risk in the investigator's opinion because of systemic diseases in addition to the cancer under study, for example, uncontrolled infections.
3. Life-threatening illness, significant organ system dysfunction, or other condition that, in the investigator's opinion, could compromise participants safety or the integrity of study outcomes or interfere with the absorption or metabolism of ASTX029.
4. Prior anticancer treatments or therapies within the indicated time window prior to first dose of study treatment (ASTX029), as follows:

1. Cytotoxic chemotherapy or radiotherapy within 3 weeks prior. Palliative radiotherapy to a single lesion within 2 weeks prior. Any encountered treatment-related toxicities (excepting alopecia) not stabilized or resolved to ≤Grade 1.
2. Monoclonal antibodies within 4 weeks prior. Any encountered treatment-related toxicities not stabilized or resolved to ≤Grade 1.
3. Molecularly targeted drug or 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 (excepting alopecia) not stabilized or resolved to ≤Grade 1.
5. Prior treatment with extracellular signal-regulated kinase (ERK) inhibitors.
6. History of, or at risk for, cardiac disease, as evidenced by 1 or more of the following conditions:

1. Abnormal left ventricular ejection fraction (LVEF; \<50%) on echocardiogram (ECHO) or multiple-gated acquisition (MUGA) scan.
2. Congestive cardiac failure of ≥Grade 3 severity according to New York Heart Association functional classification defined as patients with marked limitation of activity and who are comfortable only at rest.
3. Unstable cardiac disease including unstable angina or hypertension as defined by the need for overnight hospital admission within the last 3 months (90 days).
4. History or evidence of long QT interval corrected for heart rate (QTc), ventricular arrhythmias including ventricular bigeminy, complete left bundle branch block, clinically significant bradyarrhythmias such as sick sinus syndrome, second- and third-degree atrioventricular (AV) block, presence of cardiac pacemaker or defibrillator, or other significant arrhythmias.
5. Screening 12-lead electrocardiogram (ECG) with measurable QTc interval of ≥470 msec. (Fridericia's formula should be used to calculate the QTc interval throughout the study.)
7. Known history of human immunodeficiency virus (HIV) infection or seropositive results consistent with active hepatitis B virus (HBV) or active hepatitis C virus (HCV) infection.
8. Known brain metastases, unless previously treated and stable for at least 3 months with or without steroids.
9. Known significant mental illness or other conditions, such as active alcohol or other substance abuse that, in the opinion of the investigator, predispose the participant to high risk of noncompliance with the protocol treatment or assessments.
10. History or current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) including:

1. Presence of predisposing factors to RVO or CSR (eg, uncontrolled glaucoma or ocular hypertension, uncontrolled diabetes mellitus) or
2. Visible retinal pathology as assessed by ophthalmic examination at screening that is considered a risk factor for RVO or CSR such as:

* Evidence of optic disc cupping or
* Evidence of new visual field defects on automated perimetry or
* Intraocular pressure \>21 mmHg as measured by tonography.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Taiho Oncology, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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City of Hope Comprehensive Cancer Center

Duarte, California, United States

Site Status

University of Southern California Norris Comprehensive Cancer Center Site#114

Los Angeles, California, United States

Site Status

Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute Site# 107

Los Angeles, California, United States

Site Status

Hoag Memorial Hospital Site#115

Newport Beach, California, United States

Site Status

University of California Davis Medical Center Site #121

Sacramento, California, United States

Site Status

California Pacific Medical Center - Sutter Pacific Medical Center Site#117

San Francisco, California, United States

Site Status

Smilow Cancer Hospital at Yale New Haven Site#105

New Haven, Connecticut, United States

Site Status

Holden Comprehensive Cancer Center

Iowa City, Iowa, United States

Site Status

The Sidney Kimmel Comprehensive Cancer Center Site#106

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital Site#103

Boston, Massachusetts, United States

Site Status

Dana Farber Cancer Institute Site#104

Boston, Massachusetts, United States

Site Status

University of Michigan Rogel Cancer Center Site #113

Ann Arbor, Michigan, United States

Site Status

Columbia University Irving Medical Center - Herbert Irving Pavilion Site#112

New York, New York, United States

Site Status

Providence Portland Medical Center Site #118

Portland, Oregon, United States

Site Status

Oregon Health and Science University Site #122

Portland, Oregon, United States

Site Status

University of Pennsylvania-Abramson Cancer Center

Philadelphia, Pennsylvania, United States

Site Status

The University of Texas MD Anderson Cancer Center Site#111

Houston, Texas, United States

Site Status

START - South Texas Accelerated Research Therapeutics, LLC Site# 101

San Antonio, Texas, United States

Site Status

Virginia Cancer Specialists Site#102

Fairfax, Virginia, United States

Site Status

Centre Léon Bérard Service d'Oncologie Médicale Site#202

Lyon, Auvergne-Rhône-Alpes, France

Site Status

Hôpital de la Timone Site #201

Marseille, , France

Site Status

Institut Català d'Oncologia Badalona Site#240

Barcelona, , Spain

Site Status

Hospital Universitari Vall d'Hebrón Servicio de Oncología, Sala coordinación UITM Site#243

Barcelona, , Spain

Site Status

Hospital Universitari Germans Trias i Pujol

Barcelona, , Spain

Site Status

Hospital Universitario Dexeus Site#241

Barcelona, , Spain

Site Status

Clinica Universidad de Navarra Madrid Site #242

Madrid, , Spain

Site Status

Clínica Universidad de Navarra Site#242

Madrid, , Spain

Site Status

The Christie NHS Foundation Trust Site#220

Manchester, England, United Kingdom

Site Status

Cambridge University Hospitals NHS Foundation Trust

Cambridge, , United Kingdom

Site Status

The Newcastle Upon Tyne Hospitals NHS Foundation Trust Site #221

Newcastle upon Tyne, , United Kingdom

Site Status

Churchill Hospital Site #224

Oxford, , United Kingdom

Site Status

Countries

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United States France Spain United Kingdom

References

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Munck JM, Berdini V, Bevan L, Brothwood JL, Castro J, Courtin A, East C, Ferraldeschi R, Heightman TD, Hindley CJ, Kucia-Tran J, Lyons JF, Martins V, Muench S, Murray CW, Norton D, O'Reilly M, Reader M, Rees DC, Rich SJ, Richardson CJ, Shah AD, Stanczuk L, Thompson NT, Wilsher NE, Woolford AJ, Wallis NG. ASTX029, a Novel Dual-mechanism ERK Inhibitor, Modulates Both the Phosphorylation and Catalytic Activity of ERK. Mol Cancer Ther. 2021 Oct;20(10):1757-1768. doi: 10.1158/1535-7163.MCT-20-0909. Epub 2021 Jul 30.

Reference Type DERIVED
PMID: 34330842 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ASTX029-01

Identifier Type: -

Identifier Source: org_study_id

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