NCT04514497

Brief Summary

This phase I trial tests the safety, side effects and best dose of BAY 1895344 when given together with usual chemotherapy (irinotecan or topotecan) in treating patients with solid tumors that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced), with a specific focus on small cell lung cancer, poorly differentiated neuroendocrine cancer, and pancreatic cancer. BAY 1895344 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as irinotecan and topotecan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding BAY 1895344 to irinotecan or topotecan may be safe and tolerable in treating patients with advanced solid tumors.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
28

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2021

Longer than P75 for phase_1

Geographic Reach
1 country

23 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 14, 2020

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 17, 2020

Completed
1.2 years until next milestone

Study Start

First participant enrolled

October 20, 2021

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 15, 2025

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

March 4, 2026

Completed
Last Updated

March 6, 2025

Status Verified

March 1, 2025

Enrollment Period

3.3 years

First QC Date

August 14, 2020

Last Update Submit

March 5, 2025

Conditions

Outcome Measures

Primary Outcomes (2)

  • Maximum tolerated dose (MTD) (Dose Escalation Phase)

    Defined by occurrence of \>= 2 dose limiting toxicities (DLTs) defined as grade 4 neutropenia lasting \>= 7 days, grade 4 thrombocytopenia, grade 4 anemia, grade 3 neutropenia with fever, grade 3 thrombocytopenia with bleeding, any grade 3 hematologic toxicity lasting \>= 7 days (counting from first day of toxicity grade recognition) or any non-hematologic grade \>= 2 adverse events (AEs) lasting \>= 7 days (with the exception of grade 2 \[G2\] fatigue, G2 nausea or G2 diarrhea) (counting from first day of toxicity grade recognition) in any dose level during cycle 1 of treatment. DLTs will be graded by Common Terminology Criteria for Adverse Events version 5.0.

    Up to 21 days

  • Occurrence of grade 4 hematologic AEs (Dose Expansion Phase)

    Grade 4 hematologic toxicity will be monitored using the Bayesian approach of Thall, Simon, Estey as extended by Thall and Sung. Clinical safety data (e.g. AEs) will be tabulated and summarized using descriptive statistics as requested by the sponsor investigator, executive committee, medical monitor or Data Safety Monitoring Board using methods described in the Data Safety Monitoring Plan.

    Up to 6 months post-treatment

Secondary Outcomes (8)

  • Objective response rate (ORR)

    Up to 12 weeks

  • Duration of response (DOR)

    From when a patient achieves disease control (complete response, partial response, stable disease) on a restaging scan to the time of radiographic progression, assessed up to 6 months post-treatment

  • Progression-free survival (PFS)

    From when a patient starts treatment to when they demonstrate radiographic progression or succumb to the disease, assessed up to 6 months post-treatment

  • Overall survival (OS)

    From when a patient starts treatment to the date they succumb to the disease, assessed up to 6 months post-treatment

  • Maximum concentration (Cmax)

    Cycle 1, days 1, 2, 3, and 4

  • +3 more secondary outcomes

Other Outcomes (2)

  • Tumor ATM expression loss

    Baseline

  • Tumor deoxyribonucleic acid damage response (DDR) gene mutations present

    Baseline

Study Arms (3)

Cohort I (elimusertib, irinotecan)

EXPERIMENTAL

Patients receive elimusertib PO BID on days 1 and 2 and irinotecan IV over 90 minutes on day 1 of each cycle. Cycles repeat every 14 days in the absence of disease progression or unacceptable toxicity. Patients undergo CT and/or MRI throughout the study, tumor biopsy at screening and on study, and collection of blood samples at screening.

Procedure: BiopsyProcedure: Biospecimen CollectionProcedure: Computed TomographyDrug: ElimusertibDrug: Irinotecan HydrochlorideProcedure: Magnetic Resonance Imaging

Cohort II (elimusertib, irinotecan)

EXPERIMENTAL

Patients receive elimusertib PO QD on days 2, 3, 9, 10, 16, and 17 of cycle 1 and 2, and on days 2, 3, 9, and 10 of each cycle thereafter. Patients receive irinotecan IV over 90 minutes on days 1, 8, and 15 of cycle 1 and 2, and on days 1 and 8 of each cycle thereafter. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo CT and/or MRI throughout the study, tumor biopsy at screening and on study, and collection of blood samples at screening.

Procedure: BiopsyProcedure: Biospecimen CollectionProcedure: Computed TomographyDrug: ElimusertibDrug: Irinotecan HydrochlorideProcedure: Magnetic Resonance Imaging

Cohort III (elimusertib, topotecan)

EXPERIMENTAL

Patients receive elimusertib PO QD on days 2 and 5 and topotecan IV over 30 minutes on days 1-5 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo CT and/or MRI throughout the study, tumor biopsy at screening and on study, and collection of blood samples at screening.

Procedure: BiopsyProcedure: Biospecimen CollectionProcedure: Computed TomographyDrug: ElimusertibProcedure: Magnetic Resonance ImagingDrug: Topotecan Hydrochloride

Interventions

BiopsyPROCEDURE

Undergo tumor biopsy

Also known as: BIOPSY_TYPE, Bx
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)Cohort III (elimusertib, topotecan)

Undergo collection of blood samples

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)Cohort III (elimusertib, topotecan)

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, tomography
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)Cohort III (elimusertib, topotecan)

Given PO

Also known as: ATR Inhibitor BAY1895344, ATR Kinase Inhibitor BAY1895344, BAY 1895344, BAY-1895344, BAY1895344
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)Cohort III (elimusertib, topotecan)

Given IV

Also known as: Campto, Camptosar, Camptothecin 11, Camptothecin-11, CPT 11, CPT-11, CPT11, Irinomedac, Irinotecan Hydrochloride Trihydrate, Irinotecan Monohydrochloride Trihydrate, U 101440E, U-101440E, U101440E
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI
Cohort I (elimusertib, irinotecan)Cohort II (elimusertib, irinotecan)Cohort III (elimusertib, topotecan)

Given IV

Also known as: Evotopin, Hycamptamine, Hycamtin, Nogitecan Hydrochloride, Potactasol, SKF S 104864 A, SKF S-104864-A, SKF S104864A, Topotec, Topotecan HCl, topotecan hydrochloride (oral)
Cohort III (elimusertib, topotecan)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • DOSE ESCALATION COHORTS: Patients must have a biopsy-proven solid tumor that is metastatic or unresectable and has progressed on at least one line of standard therapy
  • DOSE ESCALATION COHORTS: Patients must have a solid tumor for which irinotecan or topotecan is considered standard of care
  • DOSE EXPANSION COHORTS: Patients must have biopsy proven metastatic or unresectable small cell lung cancer (SCLC), poorly differentiated neuroendocrine carcinoma (PD-NEC) (any extrapulmonary neuroendocrine carcinoma with small cell or large cell histology) or pancreatic adenocarcinoma (PDA) and have progressed on at least one line of standard therapy
  • DOSE EXPANSION COHORTS: Patients must have at least one measurable lesion outside of the lesion to be biopsied
  • Patients must be able to swallow pills
  • Age \>= 18 years. Because no dosing or adverse event data are currently available on the use of BAY 1895344 in combination with irinotecan or topotecan in patients \< 18 years of age, children are excluded from this study
  • Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%)
  • Hemoglobin \> 9 g/dL
  • Absolute neutrophil count \>= 1,500/mcL
  • Platelets \>= 100,000/mcL
  • Total bilirubin =\< 2 x institutional upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3 x institutional ULN (=\< 5 x institutional ULN if liver metastases present)
  • Glomerular filtration rate (GFR) \>= 60 mL/min/1.73 m\^2
  • Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
  • For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
  • +6 more criteria

You may not qualify if:

  • Patients who have previously been treated with irinotecan will not be eligible to participate in the irinotecan arm and patients who have previously been treated with topotecan will not be eligible to participate in the topotecan arm. However, patients who previously received irinotecan may be treated with topotecan (and vice versa) should the other agent be considered a possible standard of care for their disease. Patients who have previously been treated with BAY 1895344 will be excluded from the study
  • Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study
  • Patients who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities \> grade 1) with the exception of alopecia and endocrinopathies from prior immunotherapy
  • Patients who are receiving any other investigational agents
  • The investigator(s) must state a medical or scientific reason if patients who have brain metastases will be excluded from the study
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to BAY 1895344 or other agents used in study
  • Patients receiving any medications or substances that are substrates of CYP3A4 with a narrow therapeutic window, or strong inhibitors/inducers of CYP3A4 are ineligible, if they cannot be transferred to alternative medication. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
  • Patients with uncontrolled intercurrent illness
  • Patients with psychiatric illness/social situations that would limit compliance with study requirements
  • Pregnant women are excluded from this study because BAY 1895344 is agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with BAY 1895344, breastfeeding should be discontinued if the mother is treated with BAY 1895344. These potential risks may also apply to other agents used in this study
  • Patients with an uncontrolled infection requiring IV antibiotics will not be eligible to participate in the study
  • Patients on strong CYP3A4 inhibitors must discontinue them at least 1 week prior to starting irinotecan therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (23)

Mayo Clinic Hospital in Arizona

Phoenix, Arizona, 85054, United States

Location

UC Irvine Health/Chao Family Comprehensive Cancer Center

Orange, California, 92868, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

Smilow Cancer Hospital Care Center-Trumbull

Trumbull, Connecticut, 06611, United States

Location

Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States

Location

University of Kansas Clinical Research Center

Fairway, Kansas, 66205, United States

Location

University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States

Location

University of Kansas Cancer Center-Overland Park

Overland Park, Kansas, 66210, United States

Location

University of Kansas Hospital-Indian Creek Campus

Overland Park, Kansas, 66211, United States

Location

University of Kansas Hospital-Westwood Cancer Center

Westwood, Kansas, 66205, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Siteman Cancer Center at Saint Peters Hospital

City of Saint Peters, Missouri, 63376, United States

Location

Siteman Cancer Center at West County Hospital

Creve Coeur, Missouri, 63141, United States

Location

University of Kansas Cancer Center - North

Kansas City, Missouri, 64154, United States

Location

University of Kansas Cancer Center - Lee's Summit

Lee's Summit, Missouri, 64064, United States

Location

University of Kansas Cancer Center at North Kansas City Hospital

North Kansas City, Missouri, 64116, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Siteman Cancer Center-South County

St Louis, Missouri, 63129, United States

Location

Siteman Cancer Center at Christian Hospital

St Louis, Missouri, 63136, United States

Location

Montefiore Medical Center-Einstein Campus

The Bronx, New York, 10461, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

University of Pittsburgh Cancer Institute (UPCI)

Pittsburgh, Pennsylvania, 15232, United States

Location

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Small Cell Lung CarcinomaNeoplasm MetastasisCarcinoma, NeuroendocrineLung NeoplasmsPancreatic Neoplasms

Interventions

BiopsySpecimen HandlingBAY 1895344IrinotecanMagnetic Resonance SpectroscopyTopotecan

Condition Hierarchy (Ancestors)

Carcinoma, BronchogenicBronchial NeoplasmsRespiratory Tract NeoplasmsThoracic NeoplasmsNeoplasms by SiteNeoplasmsLung DiseasesRespiratory Tract DiseasesNeoplastic ProcessesPathologic ProcessesPathological Conditions, Signs and SymptomsNeuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeAdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueDigestive System NeoplasmsEndocrine Gland NeoplasmsDigestive System DiseasesPancreatic DiseasesEndocrine System Diseases

Intervention Hierarchy (Ancestors)

CytodiagnosisCytological TechniquesClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisDiagnostic Techniques, SurgicalSurgical Procedures, OperativeInvestigative TechniquesCamptothecinAlkaloidsHeterocyclic CompoundsSpectrum AnalysisChemistry Techniques, Analytical

Study Officials

  • Thatcher Heumann

    Yale University Cancer Center LAO

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 14, 2020

First Posted

August 17, 2020

Study Start

October 20, 2021

Primary Completion

February 15, 2025

Study Completion

March 4, 2026

Last Updated

March 6, 2025

Record last verified: 2025-03

Data Sharing

IPD Sharing
Will share

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

More information

Locations