Phase 1-2 Study of ASTX660 in Subjects With Advanced Solid Tumors and Lymphomas

NCT ID: NCT02503423

Last Updated: 2025-01-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

230 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-31

Study Completion Date

2025-12-31

Brief Summary

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This is an open-label, dose-escalation Phase 1/2 study to assess the safety of ASTX660, determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and recommended dosing regimen, and to obtain preliminary efficacy, pharmacokinetic (PK), and target engagement data, in subjects with advanced solid tumors or lymphoma for whom standard life-prolonging measures are not available.

Detailed Description

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ASTX660 is a synthetic small molecule dual antagonist of cellular inhibitor of apoptosis protein (cIAP) 1 and X-linked inhibitor of apoptosis protein (XIAP) that has been shown to have potent proapoptotic and tumor growth inhibitory activity in nonclinical models. The Phase 1 portion of the study (completed) will determine the MTD, RP2D, and recommended dosing regimen. The Phase 2 portion will evaluate activity in selected tumor types. Subjects will continue to receive their assigned treatment throughout the study until the occurrence of disease progression, death, or unacceptable treatment-related toxicity, or until the study is closed by the sponsor.

Conditions

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Solid Tumors Lymphoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1 - Part 1 (completed)

Dose-escalation stage to identify the MTD and the RP2D, defined as either the MTD or a dose below the MTD that the Data and Safety Review Committee (DSRC) agree shows adequate pharmacological evidence of target engagement and/or clinical activity. Subjects will receive ASTX660 once a day for 7 consecutive days every other week of each 28-day cycle (ie, \[7 days on/ 7 days off\] ×2; daily dosing on Days 1-7 and 15-21). The starting dose will be escalated stepwise in successive cohorts of 3 to 6 evaluable subjects each (standard 3+3 study design), until the RP2D is determined.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 1 - Part 2 (completed)

Dose-expansion stage to confirm tolerability of ASTX660 at the RP2D using the every-other-week daily dosing regimen. Up to a total of 12 subjects (including the 3 or 6 subjects treated at the RP2D in Part 1) will be treated at the RP2D.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 1 - Part 3 (optional)

The purpose of the optional Part 3 is to allow for exploration of an alternative dosing regimen of ASTX660 based on emerging safety, PK, and pharmacodynamic (PD) data from Parts 1 and 2 (using the original every-other-week dosing regimen), with agreement of the DSRC. If Part 3 is conducted, the plan is to enroll up to 18 evaluable subjects in 1 or more cohorts using a standard 3+3 study design.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 1

Treatment with ASTX660 for recurrent/metastatic head and neck squamous cell carcinoma (HNSCC) not responsive or relapsed after standard therapy.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 2

Treatment with ASTX660 for relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 3

Treatment with ASTX660 for progressive or relapsed peripheral T-cell lymphoma (PTCL).

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 4

Treatment with ASTX660 for relapsed or refractory cutaneous T-cell lymphoma (CTCL).

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 5

Treatment with ASTX660 for other tumor types that are characterized by a molecular feature that may confer sensitivity to ASTX660 (eg, oncogenic activation of the NF-κB pathway or documented amplification of the gene loci encoding c-IAP1 or c-IAP2), pending confirmation in writing by the Astex medical monitor.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Phase 2 - Cohort 6

Treatment with ASTX660 for cervical carcinoma not responsive or relapsed after standard therapy.

Group Type EXPERIMENTAL

ASTX660

Intervention Type DRUG

described above

Interventions

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ASTX660

described above

Intervention Type DRUG

Other Intervention Names

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Treatment of ASTX660 for advanced solid tumors and lymphomas

Eligibility Criteria

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Inclusion Criteria

1. Able to understand and comply with the protocol and study procedures, understand the risks involved in the study, and provide written informed consent before any study-specific procedure is performed.
2. Men and women 18 years of age or older.
3. Subjects with histologically or cytologically confirmed advanced solid tumors or lymphoma that is metastatic or unresectable, and for whom standard life-prolonging measures are not available. Specific tumor types that will be selected for study in Phase 2 are detailed in the protocol.

a. For Phase 2 Cohort 3, subjects must have histologically confirmed PTCL (local pathology report) as defined by 2016 World Health Organization (WHO) classification. The following subtypes are eligible for the study: adult T-cell lymphoma/leukemia, extranodal natural killer (NK)/T-cell lymphoma nasal type, enteropathy-associated T-cell lymphoma, monomorphic epitheliotropic intestinal T-cell lymphoma, hepatosplenic T-cell lymphoma, subcutaneous panniculitis-like T-cell lymphoma, peripheral T-cell lymphoma not otherwise specified, angioimmunoblastic T-cell lymphoma, follicular T-cell lymphoma, nodal peripheral T-cell with T-follicular helper (THF) phenotype, and anaplastic large-cell lymphoma.
4. For Phase 2 Cohorts 3 and 4, patients must have evidence of documented progressive disease and must have received at least two prior systemic therapies.

1. Subjects with CD30-positive lymphoma must have received, be ineligible for, or intolerant to brentuximab vedotin, provided that brentuximab vedotin is locally approved and available.
2. Subjects with mycosis fungoides or Sezary syndrome must have received, be ineligible or intolerant to mogamulizumab, provided that mogamulizumab is locally approved and available.
5. In the Phase 2 portion of the protocol only, subjects must have measurable disease according to response criteria appropriate for their type of cancer.

a. For Phase 2 Cohort 3 (PTCL), measurable disease by contrast-enhanced diagnostic CT (at least 1 nodal lesion \>1.5 cm or extranodal lesions \>1.0 cm) is required.
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
7. Acceptable organ function, as evidenced by the following laboratory data:

1. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<=2.0 \* upper limit of normal (ULN).
2. Total serum bilirubin \<=1.5 \* ULN
3. Absolute neutrophil count (ANC):

* Phase 1 and 2 (except Phase 2 subjects with known lymphoma; ie, not applicable for Cohorts 3 or 4) \>=1500 cells/mm3
* Phase 2 subjects with known lymphoma: \>=1000 cells/mm3 (\>750 cell/mm3 for subjects with lymphoma in bone marrow)
4. Platelet count:

* Phase 1 and 2 (except Phase 2 subject with known lymphoma; ie, not applicable for Cohorts 3 or 4) \>=100,000 cells/mm3
* Phase 2 subjects with known lymphoma: \>= 50,000 cells/mm3; \>=25,000 cells/mm3 for subjects with lymphoma in bone marrow
5. Serum creatinine levels \<= 1.5 \* ULN, or calculated (by Cockcroft-Gault formula or other accepted formula) or measure creatinine clearance \>=50 mL/min.
6. Amylase and lipase \<=ULN.
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 at screening. Women of child-bearing potential and men with female partners of child-bearing potential must agree to practice 2 highly effective contraceptive measures of birth control (as described in the protocol) and must agree not to become pregnant or father a child while receiving treatment with study drug and for at least 3 months after completing treatment. Contraceptive measures which may be considered highly effective comprise combined hormonal contraception (oral, vaginal, or transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, sexual abstinence, and surgically successful vasectomy. Abstinence is acceptable only if it is consistent with the preferred and usual lifestyle of the subject. Periodic abstinence (eg, calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of birth control.

Exclusion Criteria

1. Hypersensitivity to ASTX660, excipients of the drug product, or other components of the study treatment regimen.
2. Poor medical risk because of systemic diseases (e.g. active uncontrolled infections) in addition to the qualifying disease under study.
3. Life-threatening illness, significant organ system dysfunction, or other condition that, in the investigator's opinion, could compromise subject safety or the integrity of the study outcomes, or interfere with the absorption or metabolism of ASTX660.
4. 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%) or echocardiogram ECHO or multiple gated acquisition scan (MUGA).
2. Congestive cardiac failure of \>= Grade 3 severity according to New York Heart Association (NYHA) functional classification defined as subjects with marked limitation of activity and who are comfortable only at rest.
3. Unstable cardiac disease including angina or hypertension as defined by the need for overnight hospital admission within the last 3 months (90 days).
4. History or presence of complete left bundle branch block, heart block, cardiac pacemaker or significant arrhythmia.
5. Concurrent treatment with any medical that prolongs QT interval and may induce torsades de pointes, and which cannot be discontinued at least 2 weeks before treatment with ASTX660. \[Applies to Phase 1 only\].
6. Personal history of long QTc syndrome or ventricular arrhythmias including ventricular bigeminy.
7. Screening 12-lead ECG with measurable QTc interval (according to either Fridericia's or Bazett's correction) of \>=470 msec).
8. Any other condition that, in the opinion of the investigator, could put the subject at increased cardiac risk.
5. 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.
6. Grade 2 or greater neuropathy \[Applies to Phase 1\]. Grade 3 or greater neuropathy \[Applies to Phase 2\].
7. Known brain metastases, unless stable or previously treated.
8. Known significant mental illness or other conditions such as active alcohol or other substance abuse that, in the opinion of the investigator, predisposes the subject to high risk of noncompliance with the protocol treatment or assessments.
9. Prior anticancer treatments or therapies within the indicated time window prior to first dose of study treatment (ASTX660), as follows:

1. Cytotoxic chemotherapy or radiotherapy within 3 weeks prior and any encountered treatment-related toxicities (excepting alopecia) not resolved to Grade 1 or less \[Phase 1\] or Grade 2 or less \[Phase 2\].
2. Skin directed treatments, including topicals and radiation within 2 weeks prior.
3. Monoclonal antibodies within 4 weeks prior and any encountered treatment-related toxicities not resolved to Grade 1 or less \[Phase 1\] or Grade 2 or less \[Phase 2\].
4. Small molecules or biologics (investigational or approved) within the longer of 2 weeks or 5 half-lives prior to study treatment and any encountered treatment-related toxicities not resolved to Grade 1 or less \[Phase 1\] or Grade 2 or less \[Phase 2\].
5. At least 6 weeks must have elapsed since CAR-T infusion and subjects must have experienced disease progression, and not have residual circulating CAR-T cells in peripheral blood (based on local assessment). Any encountered treatment-related toxicities must have resolved to Grade ≤1.
10. Concurrent second malignancy currently requiring active therapy, except breast or prostate cancer stable on or responding to endocrine therapy or superficial bladder cancer \[Phase 2\].
11. Known central nervous system (CNS) lymphoma \[Phase 2\].
12. Patients with a history of allogenic transplant must not have ≥Grade 3 graft-versus-host disease (GVHD) or any clinically significant GVHD requiring systemic immunosuppression \[Phase 2\].
13. Systemic corticosteroids \>20 mg prednisone equivalent (unless patient has been taking a continuous dose for \>3 weeks prior to study entry and there is documented radiological progression) \[Phase 2\]. Stable dose of medium or low potency topical corticosteroids for at least 3 weeks prior to study entry are permitted \[Phase 2\].
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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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

HonorHealth Research Institute

Scottsdale, Arizona, United States

Site Status

USC/Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

Simlow Cancer Hospital at Yale

New Haven, Connecticut, United States

Site Status

Emory University winship Cancer Institute

Atlanta, Georgia, United States

Site Status

Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Chicago, Illinois, United States

Site Status

The Sidney Kimmel Comprehensive Cancer Center at John Hopkins

Baltimore, Maryland, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Dartmouth-Hitchcock Medical Center (DHMC)

Lebanon, New Hampshire, United States

Site Status

Summit Medical Group - Florham Park Campus/Atlantic Health

Florham Park, New Jersey, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

New York University Langone Medical Center

New York, New York, United States

Site Status

New York Presbyterian Hospital Columbia University Medical Center

New York, New York, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Rochester Skin Lymphoma Medical Group

Rochester, New York, United States

Site Status

Wake Forest Baptist Health

Winston-Salem, North Carolina, United States

Site Status

The Ohio State University and Wexner Medical Center, James Cancer Hospital

Columbus, Ohio, United States

Site Status

University of Oklahoma Stephenson Cancer Center

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

West Penn Hospital

Pittsburgh, Pennsylvania, United States

Site Status

Hollings Cancer Center

Charleston, South Carolina, United States

Site Status

Vanderbilt Ingram Cancer Center

Nashville, Tennessee, United States

Site Status

MD Anderson Cancer Center

Houston, Texas, United States

Site Status

CliniCore Texas

Houston, Texas, United States

Site Status

START- South Texas Accelerated Research Therapeutics

San Antonio, Texas, United States

Site Status

Virgina Commonwealth University

Richmond, Virginia, United States

Site Status

University of Washington, Seattle Cancer Care Alliance

Seattle, Washington, United States

Site Status

Centre Hospitalier Universitaire Universite Catholique de Louvain - Site Godinne

Yvoir, Namur, Belgium

Site Status

Universitair Ziekenhuis Gent

Ghent, Oost-Vlaanderen, Belgium

Site Status

Intitut Jules Boredt

Brussels, , Belgium

Site Status

Tom Baker Cancer Centre

Calgary, Alberta, Canada

Site Status

British Columbia Cancer Agency

Vancouver, British Columbia, Canada

Site Status

Cancer Care Manitoba

Winnipeg, Manitoba, Canada

Site Status

Nova Scotia Health Athority-Qeii HSC

Halifax, Nova Scotia, Canada

Site Status

Sunnybrook Hospital

Toronto, Ontario, Canada

Site Status

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status

Jewish General Hospital

Montreal, Quebec, Canada

Site Status

Gustave Roussy Cancer Campus (IGR)

Villejuif, Cedex, France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, Lyon, France

Site Status

Institut Bergonié, Unicancer

Bordeaux, , France

Site Status

Centre Antoine Lacassagne, Oncologie Médicale

Nice, , France

Site Status

Centre Henri Becquerel, Hematology

Rouen, , France

Site Status

Institut Universitaire du Cancer - Oncopôle, Department d'Hématologie

Toulouse, , France

Site Status

CRU de Tours - Hôpital Bretonneau, Hématologie -Thérapy Cellulaire

Tours, , France

Site Status

Semmelweis Egyetem - I. sz. Belgyógyászati Klinika

Budapest, , Hungary

Site Status

Debreceni Egyetem Klinikai Központ

Debrecen, , Hungary

Site Status

Szabolcs-Szatmár-Bereg Megyei Kórházak És Egyetemi Oktatókórház

Nyíregyháza, , Hungary

Site Status

Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi

Bologna, , Italy

Site Status

Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

Brescia, , Italy

Site Status

Instituto Europeo di Oncologia

Milan, , Italy

Site Status

Azienda Socio Santaria Territoriale Monza- Osperdale San Gerado

Monza, , Italy

Site Status

Institut Catala d'Oncologia

Girona, Giona, Spain

Site Status

imCORE - Clínica Universidad de Navarra

Pamplona, Navarre, Spain

Site Status

Hospital Universitario Fundacion Jimenez Diaz Preview

Madrid, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

University Hospitals of Leicester NHS Trust

Leicester, East Midlands, United Kingdom

Site Status

University Hospital Southhampton NHS Foundation Trust - Somers Cancer Research

Southampton, Hampshire, United Kingdom

Site Status

Churchill Hospital, Oxford University Hospital NHS Trust

Oxford, Oxfordshire, United Kingdom

Site Status

The Royal Marsden NHS Foundation Trust

Sutton, Surrey, United Kingdom

Site Status

University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Beatson Cancer Center and University of Glasgow

Glasgow, , United Kingdom

Site Status

University College London Hospitals NHS Foundation Trust

London, , United Kingdom

Site Status

Guy's and Saint Thomas' NHS Foundation Trust

London, , United Kingdom

Site Status

The Christie NHS Foundation Trust, Christie Hospital

Manchester, , United Kingdom

Site Status

Countries

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United States Belgium Canada France Hungary Italy Spain United Kingdom

References

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Johnson CN, Ahn JS, Buck IM, Chiarparin E, Day JEH, Hopkins A, Howard S, Lewis EJ, Martins V, Millemaggi A, Munck JM, Page LW, Peakman T, Reader M, Rich SJ, Saxty G, Smyth T, Thompson NT, Ward GA, Williams PA, Wilsher NE, Chessari G. A Fragment-Derived Clinical Candidate for Antagonism of X-Linked and Cellular Inhibitor of Apoptosis Proteins: 1-(6-[(4-Fluorophenyl)methyl]-5-(hydroxymethyl)-3,3-dimethyl-1 H,2 H,3 H-pyrrolo[3,2- b]pyridin-1-yl)-2-[(2 R,5 R)-5-methyl-2-([(3R)-3-methylmorpholin-4-yl]methyl)piperazin-1-yl]ethan-1-one (ASTX660). J Med Chem. 2018 Aug 23;61(16):7314-7329. doi: 10.1021/acs.jmedchem.8b00900. Epub 2018 Aug 9.

Reference Type DERIVED
PMID: 30091600 (View on PubMed)

Other Identifiers

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ASTX660-01

Identifier Type: -

Identifier Source: org_study_id

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