Elimusertib for the Treatment of Relapsed or Refractory Solid Tumors
A Phase 1/ 2 Study of BAY 1895344 (Elimusertib, NSC#810486) in Pediatric Patients With Relapsed or Refractory Solid Tumors
3 other identifiers
interventional
31
2 countries
29
Brief Summary
This phase I/II trial tests the safety, best dose, and whether elimusertib works in treating patients with solid tumors that have come back (relapsed) or does not respond to treatment (refractory). Elimusertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Dec 2021
Longer than P75 for phase_1
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 7, 2021
CompletedFirst Posted
Study publicly available on registry
October 8, 2021
CompletedStudy Start
First participant enrolled
December 22, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2024
CompletedResults Posted
Study results publicly available
January 23, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
March 31, 2026
CompletedJanuary 23, 2026
October 1, 2025
2.8 years
October 7, 2021
October 3, 2025
January 8, 2026
Conditions
Outcome Measures
Primary Outcomes (2)
Frequency of Cycle 1 Dose Limiting Toxicities for BAY 1895344 (Elimusertib) During Dose Escalation
Frequency (%) of patients experiencing a cycle 1 dose limiting toxicity among toxicity-evaluable patients stratified by dose level during the dose escalation part of the study (Part A).
Up to 28 days (cycle 1)
Response of BAY 1895344 (Elimusertib)
Frequency (%) of patients with best response of complete response or partial response among response-evaluable patients stratified by Phase 2 cohort.
Through study completion, up to 37 months
Secondary Outcomes (11)
Incidence of Adverse Events of BAY 1895344 (Elimusertib)
Through study completion, up to 37 months
Area Under the Drug Concentration Time Curve of BAY 1895344 (Elimusertib)
Up to 28 days
Change in Phosphorylated (p)ATR of BAY 1895344 (Elimusertib)
Up to 28 days
Change in pH2AX of BAY 1895344 (Elimusertib)
Up to 28 days
Change in pKAP1 of BAY 1895344 (Elimusertib)
Up to 28 days
- +6 more secondary outcomes
Study Arms (1)
Treatment (elimusertib)
EXPERIMENTALPatients receive elimusertib PO BID on days 1-3, 8-10, 15-17, and 22-24 of each cycle. Treatment repeats every 28 days for 26 cycles in the absence of disease progression or unacceptable toxicity.
Interventions
Given PO
Eligibility Criteria
You may qualify if:
- Part A: Patients between \>= 12 months and \< 18 years of age
- Part B:
- Patients between \>= 12 months and =\< 30 years of age for the phase 2 expansion cohorts for both EWS and PAX3-FOXO1 ARMS.
- Patients between \>= 12 months and =\< 21 years of age for the phase 2 DDR expansion cohort
- The Phase 2 cohorts will initially open concurrently with the Phase 1 portion but will only enroll patients at least 18 years of age. Patients \< 18 years of age will be included in the Phase 2 cohorts only after the RP2D/MTD has been estimated in the Phase 1 portion
- All patients for both Parts A and B must have a minimum body surface area (BSA) \>= 0.74 m\^2
- All patients for both Parts A and B must have the ability to swallow BAY 1895344 (elimusertib) tablets intact
- Patients with recurrent or refractory solid tumors. Patients must have had histologic verification of malignancy at original diagnosis or relapse
- Part A: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one of the following criteria:
- Any Ewing Sarcoma (histological confirmation alone is adequate) or any EWS-fusion positive solid tumor (i.e. including related Ewing's family of tumors with EWS fusions such as EWS-WT1, EWS-ATF1, etc.)
- Alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion. This does not include PAX7-FOXO1 or other variant fusion ARMS. Please note that a FISH showing FOXO1 breakapart is NOT sufficient for eligibility onto this cohort since it cannot distinguish between FOXO1 partners
- Any (non-CNS primary) solid tumor including lymphoma with inactivating alterations of any of the DNA Damage Repair (DDR) genes: ATM, ATRX, BRCA1, BRCA2, CDK12, CHEK1, CHEK2, FANCA, MSH2, MRE11, PALB2, PARP1, POLD1, RAD51, or XRCC2
- Part B: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one of the following criteria:
- B1, EWS Cohort:
- Any Ewing Sarcoma (histological confirmation alone is adequate) or any EWS-fusion positive solid tumor (i.e. including related Ewing's family of tumors with EWS fusions such as EWS-WT1, EWS-ATF1, etc.)
- +43 more criteria
You may not qualify if:
- Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, OR because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study and for 3 months + 2 days for males and 6 months + 2 days for females after receiving the last dose of BAY 1895344 (elimusertib) on the study. Abstinence is an acceptable method of birth control. Female patients must not breastfeed during treatment and until 4 months after last study drug administration
- Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid
- Patients who are currently receiving another investigational drug are not eligible
- Patients who are currently receiving other anti-cancer agents are not eligible
- Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
- Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study. Drugs that are considered sensitive or narrow therapeutic range CYP3A4 substrates should be avoided for the duration of protocol therapy
- Dedicated CNS imaging is not required but patients with current active CNS metastasis whether symptomatic or discovered incidentally without clinical symptoms, will be excluded from study participation
- Patients who have an uncontrolled infection are not eligible
- Patients who have received a prior solid organ transplantation are not eligible
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
UCSF Medical Center-Mission Bay
San Francisco, California, 94158, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232, United States
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030, United States
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Results Reporting Coordinator
- Organization
- Children's Oncology Group
Study Officials
- PRINCIPAL INVESTIGATOR
Michael V Ortiz
Pediatric Early Phase Clinical Trial Network
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 7, 2021
First Posted
October 8, 2021
Study Start
December 22, 2021
Primary Completion
September 30, 2024
Study Completion
March 31, 2026
Last Updated
January 23, 2026
Results First Posted
January 23, 2026
Record last verified: 2025-10
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."