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Active, not recruitingNCT05071209Updated Jan 23, 2026Results posted

Elimusertib for the Treatment of Relapsed or Refractory Solid Tumors

A Phase 1/2 interventional study of Elimusertib in Recurrent Alveolar Rhabdomyosarcoma, Recurrent Ewing Sarcoma and Recurrent Lymphoma, sponsored by National Cancer Institute (NCI). Active, not recruiting at 29 sites in 2 countries. Open to participants aged 12 Months to 30 Years. Per ClinicalTrials.gov, last updated 2026-01-23.

Sponsored by National Cancer Institute (NCI) · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
31
Allocation
Not applicable
Ages
12 Months to 30 Years
Sex
All
01

Study 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.

Read the detailed description

PRIMARY OBJECTIVES:

I. To estimate the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of BAY 1895344 (elimusertib) administered as an oral tablet, twice per day for 3 days on and 4 days off to patients \< 18 years of age with recurrent or refractory Ewing sarcoma (and EWS fusions), PAX3-FOXO1 alveolar rhabdomyosarcoma, and non-central nervous system (CNS) solid tumors or lymphoma with specific deleterious deoxyribonucleic acid (DNA) damage response (DDR) pathway alterations. (Phase 1/Part A) II. To define the antitumor activity of BAY 1895344 (elimusertib) in pediatric patients and young adults with recurrent or refractory Ewing sarcoma (and EWS fusions). (Phase 2/Part B) III. To define the antitumor activity of BAY 1895344 (elimusertib) in pediatric patients and young adults with recurrent or refractory PAX3-FOXO1 fusion positive alveolar rhabdomyosarcoma. (Phase 2/Part B) IV. To define and describe the toxicities of BAY 1895344 (elimusertib) administered on this schedule. (Phase 1/Part A)

SECONDARY OBJECTIVES:

I. To characterize the pharmacokinetics of BAY 1895344 (elimusertib) in children and adolescents with recurrent or refractory cancer.

II. To assess the biologic activity of BAY 1895344 (elimusertib) by immunohistochemical assessments of phosphorylated (p)ATR, pH2AX, and pKAP1 in paired tissue samples before and after treatment with BAY 1895344 (elimusertib).

III. To assess whether the activity of BAY 1895344 (elimusertib) is influenced by alternative lengthening of telomeres (ALT), as well as tumor tissue expression of ATM, PGBD5, and/or R-loops.

IV. To assess whether the activity of BAY 1895344 (elimusertib) is associated with tumor mutational processes, as measured by whole genome tumor tissue sequencing.

V. To preliminary determine the anti-tumor activity of BAY 1895344 (elimusertib) in children \< 18 years of age within the confines of a phase 1 study (Phase 1 and 2/Part A and B).

VI. To assess the antitumor activity of BAY 1895344 (elimusertib) in pediatric patients with non-CNS solid tumors or lymphomas with specific deleterious alterations in DDR pathway genes. (Phase 2/Part B)

OUTLINE: This is pediatric a phase I, dose-escalation study as well as a phase II dose expansion study in pediatric patients and young adults.

Patients receive elimusertib orally (PO) twice daily (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.

After completion of study treatment, patients are followed up every 3 months for 12 months, every 6 months for 24 months, and then annually for up to 60 months.

02

Conditions studied

  • Recurrent Alveolar Rhabdomyosarcoma
  • Recurrent Ewing Sarcoma
  • Recurrent Lymphoma
  • Recurrent Malignant Solid Neoplasm
  • Refractory Alveolar Rhabdomyosarcoma
  • Refractory Ewing Sarcoma
  • Refractory Lymphoma
  • Refractory Malignant Solid Neoplasm
03

In context

Sarcoma, Ewing

250 studies on the registry are indexed under Sarcoma, Ewing; 69 are open to participants now.

This study's enrollment of 31 is below the median of 36 across 204 interventional studies indexed under Sarcoma, Ewing.

Browse Sarcoma, Ewing studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Months to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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 solid tumors. Patients must have had histologic verification of 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.)

    • B2, PAX3-FOXO1 ARMS Cohort:

      • 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

    • B3, DDR Non-statistical Cohort:

      • 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
  • All the genes on the DDR panel are annotated with OncoKB, a precision oncology knowledge base which is publicly available here: https://www.oncokb.org/. Alterations which are categorized either 'Oncogenic' or 'Likely Oncogenic' would be considered sufficient for eligibility on either the phase 1 or phase 2 portions of this study. Alterations which are not annotated in OncoKB will need to be reviewed with locally qualified experts in molecular pathology, such as via an established molecular tumor board, in order to determine the likely oncogenicity AND will require approval by the study chair, Dr. Michael Ortiz. If such experts are not available at any institution, the study chair will review
  • In cases where multiple mutations are present or multiple samples are available, either at different locations or different points in time, the presence of a single qualifying genomic alteration in any of those samples will is considered sufficient for eligibility on the phase 2 portions of this study
  • Qualifying aberrations must be detected in either DNA or ribonucleic acid (RNA) in any tumor tissue sample (i.e. detection of a variant on circulating tumor DNA/RNA is not sufficient to qualify) using a somatic (and/or germline) mutational testing approach with either a targeted panel or whole exome/genome sequencing in the context of a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory setting. Any CLIA certified laboratory is acceptable to use
  • Part A: Patients must have either measurable or evaluable disease
  • Part B (1, 2, 3): Patients must have
  • Patients with a prior history of may enroll on study provided there is no current evidence of active disease at the time of enrollment
  • Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
  • Patients must have a performance status corresponding to (ECOG) scores of 0, 1 or 2. Use >= 50% for patients > 16 years of age and Lansky >= 50% for patients =\< 16 years of age. Note that neurologic deficits in patients with tumors previously to the CNS (or other non-oncologic reasons) must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score

  • Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately

    • Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
    • Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced or [ANC] counts): >= 7 days after the last dose of agent
    • Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
    • : If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
    • Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
    • Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)

    • Stem cell Infusions (with or without total-body irradiation [TBI]):

      • Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusions (DLI) or boost infusion: >= 84 days after infusion and no evidence of (GVHD)
      • Autologous stem cell infusion including boost infusion: >= 30 days
    • Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
    • Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
    • Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
    • Study specific prior therapy: Patients must not have received prior exposure to BAY 1895344 (elimusertib) or any other specific ATR inhibitors including berzosertib (M6620, VX-970), ceralasertib (AZD6738), M4344 (VX-803), M1774, and RP-3500. Treatment with other DNA damage repair inhibitors which do not specifically inhibit ATR (e.g. PARP inhibitors, WEE1 inhibitors, CHEK1 inhibitors, etc.) does not exclude them from eligibility on this study

  • For patients with solid tumors without known bone marrow involvement

    • Peripheral absolute neutrophil count (ANC) >= 1000/uL

  • For patients with solid tumors without known bone marrow involvement

    • Platelet count >= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)

  • For patients with solid tumors without known bone marrow involvement

    • >= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions)
  • Patients with known or possible bone marrow metastatic disease will be eligible for study provided they meet the blood counts in above inclusion criteria (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled must be evaluable for hematologic toxicity

  • Serum or radioisotope (GFR) >= 70 mL/min/1.73 m\^2 or a creatinine based on age/gender as follows:

    • Age: 1 to \< 2 years; Maximum serum creatinine (mg/dL): 0.6 (male); 0.6 (female)
    • Age: 2 to \< 6 years; Maximum serum creatinine (mg/dL): 0.8 (male); 0.8 (female)
    • Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): 1 (male); 1 (female)
    • Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): 1.2 (male); 1.2 (female)
    • Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): 1.5 (male); 1.4 (female)
    • Age: >= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male); 1.4 (female)
  • (sum of conjugated + unconjugated or total) =\< 1.5 x (ULN) for age
  • Serum glutamate pyruvate transaminase () (alanine aminotransferase [ALT]) =\< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L
  • Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled as evidenced by no increase in seizure frequency in the prior 7 days. For patients a history of seizure but not on anticonvulsants, no seizure in the past 3 months
  • Nervous system disorders ( [CTCAE] version [v]5) resulting from prior therapy must be =\< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible

Exclusion criteria

Exclusion Criteria:

  • 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 may not participate unless they have agreed to use two effective methods of , 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
  • : 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 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 post bone marrow transplant are not eligible for this trial
  • Patients who are currently receiving drugs that are strong inducers or inhibitors of 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 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
Terms in these criteria, in plain language(22)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

Brain metastases

Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).

Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

Reproductive potential

Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.

Criteria about it usually ask for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
31 participants (actual)

Study arms

  • Experimental
    Treatment (elimusertib)

    Patients 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.

    Drug: Elimusertib

Interventions

  • DrugElimusertib

    Given PO

    Also known as: ATR Inhibitor BAY1895344, ATR Kinase Inhibitor BAY1895344, BAY 1895344, BAY-1895344, BAY1895344

06

What researchers measure

Primary outcomes

  1. 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).

    Time frame: Up to 28 days (cycle 1)

  2. 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.

    Time frame: Through study completion, up to 37 months

Secondary outcomes

  1. Incidence of Adverse Events of BAY 1895344 (Elimusertib)

    Frequency (%) of patients with adverse events stratified by study part and dose level.

    Time frame: Through study completion, up to 37 months

  2. Area Under the Drug Concentration Time Curve of BAY 1895344 (Elimusertib)

    Median (min, max) of the area under the drug concentration time curve (AUC) in cycle 1 stratified by study part and dose level.

    Time frame: Up to 28 days

  3. Change in Phosphorylated (p)ATR of BAY 1895344 (Elimusertib)

    Median (min, max) of the change in (p)ATR stratified by study part and dose level.

    Time frame: Up to 28 days

  4. Change in pH2AX of BAY 1895344 (Elimusertib)

    Median (min, max) of the change in pH2AX stratified by study part and dose level.

    Time frame: Up to 28 days

  5. Change in pKAP1 of BAY 1895344 (Elimusertib)

    Median (min, max) of the change in pKAP1 stratified by study part and dose level.

    Time frame: Up to 28 days

  6. Influence of ALT on Activity of BAY 1895344

    Median (min, max) of ALT in responders (CR or PR) versus non-responders among all response-evaluable patients.

    Time frame: Up to 28 days

  7. Influence of Tumor Tissue Expression of ATM on Activity of BAY 1895344

    Median (min, max) of ATM in responders (CR or PR) versus non-responders among all response-evaluable patients.

    Time frame: Up to 28 days

  8. Influence of Tumor Tissue Expression of PGBD5 on Activity of BAY 1895344

    Median (min, max) of PGBD5 in responders (CR or PR) versus non-responders among all response-evaluable patients.

    Time frame: Up to 28 days

  9. Influence of Tumor Tissue Expression of R-loops on Activity of BAY 1895344

    Median (min, max) of R-loops in responders (CR or PR) versus non-responders among all response-evaluable patients.

    Time frame: Up to 28 days

  10. Response of BAY 1895344 (Elimusertib) in Children < 18 Years

    Frequency (%) of patients with best response of complete response or partial response among response-evaluable patients in the dose escalation part of the study (Part A).

    Time frame: Up to 60 months

  11. Response of BAY 1895344 in Pediatric Patients With Non-CNS Solid Tumors or Lymphomas

    Frequency of patients with best response of complete response or partial response among response-evaluable patients with non-CNS solid tumors or lymphomas with specific deleterious alternations in DDR pathway.

    Time frame: Up to 60 months

07

Results

Posted Jan 23, 2026

Participant flow

Participant flow — Overall Study
MilestonePhase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1
Started811093
Completed00000
Not completed811093
Withdrew: Lack of efficacy81982
Withdrew: Withdrawal by subject00111

Outcome measures

PrimaryFrequency 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).

Time frame:
Up to 28 days (cycle 1)
Reported as:
Count of participants · Participants
Frequency of Cycle 1 Dose Limiting Toxicities for BAY 1895344 (Elimusertib) During Dose Escalation
ParticipantsPhase 1- Part A/ Dose Level 1Phase 1-Part PK/ Dose Level 1
Frequency of Cycle 1 Dose Limiting Toxicities for BAY 1895344 (Elimusertib) During Dose Escalation00
PrimaryResponse 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.

Time frame:
Through study completion, up to 37 months
Reported as:
Count of participants · Participants
Response of BAY 1895344 (Elimusertib)
ParticipantsPhase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1
Response of BAY 1895344 (Elimusertib)000
SecondaryIncidence of Adverse Events of BAY 1895344 (Elimusertib)

Frequency (%) of patients with adverse events stratified by study part and dose level.

Time frame:
Through study completion, up to 37 months
Reported as:
Count of participants · Participants
Incidence of Adverse Events of BAY 1895344 (Elimusertib)
ParticipantsPhase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1
Incidence of Adverse Events of BAY 1895344 (Elimusertib)81893
SecondaryArea Under the Drug Concentration Time Curve of BAY 1895344 (Elimusertib)

Median (min, max) of the area under the drug concentration time curve (AUC) in cycle 1 stratified by study part and dose level.

Time frame:
Up to 28 days
Reported as:
Median · hr*ng/mL
Area Under the Drug Concentration Time Curve of BAY 1895344 (Elimusertib)
hr*ng/mLPhase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1
Area Under the Drug Concentration Time Curve of BAY 1895344 (Elimusertib)7617 (3260.0 to 8354.0)7527 (7527.0 to 7527.0)
SecondaryChange in Phosphorylated (p)ATR of BAY 1895344 (Elimusertib)

Median (min, max) of the change in (p)ATR stratified by study part and dose level.

Time frame:
Up to 28 days

No measurements were reported for this outcome.

SecondaryChange in pH2AX of BAY 1895344 (Elimusertib)

Median (min, max) of the change in pH2AX stratified by study part and dose level.

Time frame:
Up to 28 days

No measurements were reported for this outcome.

SecondaryChange in pKAP1 of BAY 1895344 (Elimusertib)

Median (min, max) of the change in pKAP1 stratified by study part and dose level.

Time frame:
Up to 28 days

No measurements were reported for this outcome.

SecondaryInfluence of ALT on Activity of BAY 1895344

Median (min, max) of ALT in responders (CR or PR) versus non-responders among all response-evaluable patients.

Time frame:
Up to 28 days

Results for this outcome have not been posted.

SecondaryInfluence of Tumor Tissue Expression of ATM on Activity of BAY 1895344

Median (min, max) of ATM in responders (CR or PR) versus non-responders among all response-evaluable patients.

Time frame:
Up to 28 days

Results for this outcome have not been posted.

SecondaryInfluence of Tumor Tissue Expression of PGBD5 on Activity of BAY 1895344

Median (min, max) of PGBD5 in responders (CR or PR) versus non-responders among all response-evaluable patients.

Time frame:
Up to 28 days

Results for this outcome have not been posted.

SecondaryInfluence of Tumor Tissue Expression of R-loops on Activity of BAY 1895344

Median (min, max) of R-loops in responders (CR or PR) versus non-responders among all response-evaluable patients.

Time frame:
Up to 28 days

Results for this outcome have not been posted.

SecondaryResponse of BAY 1895344 (Elimusertib) in Children < 18 Years

Frequency (%) of patients with best response of complete response or partial response among response-evaluable patients in the dose escalation part of the study (Part A).

Time frame:
Up to 60 months

Results for this outcome have not been posted.

SecondaryResponse of BAY 1895344 in Pediatric Patients With Non-CNS Solid Tumors or Lymphomas

Frequency of patients with best response of complete response or partial response among response-evaluable patients with non-CNS solid tumors or lymphomas with specific deleterious alternations in DDR pathway.

Time frame:
Up to 60 months

Results for this outcome have not been posted.

Adverse events

Collected over Through study completion, up to 37 months. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Phase 1- Part A/ Dose Level 16/8 (75%)7/8 (87.5%)8/8 (100%)
Phase 1- Part PK/ Dose Level 11/1 (100%)1/1 (100%)1/1 (100%)
Phase 2- Part B1/ Dose Level 19/10 (90%)8/10 (80%)10/10 (100%)
Phase 2- Part B2/ Dose Level 18/9 (88.9%)9/9 (100%)9/9 (100%)
Phase 2- Part B3/ Dose Level 12/3 (66.7%)3/3 (100%)3/3 (100%)
Most frequent serious events
Showing 10 of 26
Most frequent serious events
EventPhase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1
AnemiaBlood and lymphatic system disorders3/80/15/101/93/3
White blood cell decreasedInvestigations1/81/12/103/92/3
Lymphocyte count decreasedInvestigations4/80/14/106/92/3
Neutrophil count decreasedInvestigations3/80/13/102/92/3
DehydrationMetabolism and nutrition disorders0/80/10/100/91/3
HypotensionVascular disorders0/80/10/100/91/3
NauseaGastrointestinal disorders0/80/10/100/91/3
Pain in extremityMusculoskeletal and connective tissue disorders0/80/10/100/91/3
VomitingGastrointestinal disorders0/80/10/100/91/3
Pleural effusionRespiratory, thoracic and mediastinal disorders2/80/12/100/90/3
Most frequent other events
Showing 10 of 172
Most frequent other events
EventPhase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1
AnemiaBlood and lymphatic system disorders8/81/110/109/93/3
Back painMusculoskeletal and connective tissue disorders5/81/14/105/90/3
CoughRespiratory, thoracic and mediastinal disorders4/81/11/101/90/3
Creatinine increasedInvestigations2/81/11/101/92/3
DizzinessNervous system disorders1/81/12/100/91/3
FatigueGeneral disorders and administration site conditions2/81/16/104/92/3
FeverGeneral disorders and administration site conditions0/81/12/100/91/3
HeadacheNervous system disorders1/80/17/102/93/3
HypocalcemiaMetabolism and nutrition disorders0/81/11/102/92/3
HypophosphatemiaMetabolism and nutrition disorders2/80/11/102/93/3

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Phase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1Total
<=18 years8113215
Between 18 and 65 years0096116
>=65 years000000
Age, Continuous
Age, Continuous(years)Phase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1Total
Median13 (10 to 17)11 (11 to 11)20 (18 to 28)22 (14 to 26)18 (16 to 19)19 (10 to 28)
Sex: Female, Male
Sex: Female, Male(Participants)Phase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1Total
Female4168120
Male4041211
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Phase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1Total
Hispanic or Latino202116
Not Hispanic or Latino6088224
Unknown or Not Reported010001
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Phase 1- Part A/ Dose Level 1Phase 1- Part PK/ Dose Level 1Phase 2- Part B1/ Dose Level 1Phase 2- Part B2/ Dose Level 1Phase 2- Part B3/ Dose Level 1Total
American Indian or Alaska Native000000
Asian000101
Native Hawaiian or Other Pacific Islander000000
Black or African American000101
White5097324
More than one race000000
Unknown or Not Reported311005
08

Study locations

29 sites
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: Yes — "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."

Data sharing details ↗ · grants.nih.gov

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Dec 2021 to primary completion in Sep 2024, 33 months.

Results were first posted in Jan 2026, 1 year 3 months after primary completion.

Of 27 phase 1/2 interventional studies under Sarcoma, Ewing, half reached primary completion within 51 months of starting (the median), counting those still running; a quarter within 40 and three quarters within 84. This study's 33 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 7, 2021
First posted
Oct 8, 2021
StartActual
Dec 22, 2021
Primary completionActual
Sep 30, 2024
CompletionEst.
Mar 31, 2026
Results first posted
Jan 23, 2026
Last update
Jan 23, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 23, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05071209
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Oct 8, 2021
Start date
Dec 22, 2021
Primary completion
Sep 30, 2024
Completion
Mar 31, 2026 (estimated)
Results posted
Jan 23, 2026
Last update
Jan 23, 2026

Study contacts

Michael V Ortiz
principal investigator · Pediatric Early Phase Clinical Trial Network

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: BAY 1895344

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