Phase 1
N=110
A Trial to Find and Investigate a Safe Dose of a New Substance (BI 754091) for Patients With Solid Tumours
Neoplasms
Bottom Line
View on ClinicalTrials.gov: NCT02952248 ↗Enrolled (actual)
110
Serious AEs
41.8%
Results posted
Jan 2026
Primary outcome: Primary: Phase Ia Dose Escalation: Number of Participants With Dose-limiting Toxicities (DLTs) in the First Cycle (3 Weeks) — 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- BI 754091 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Boehringer Ingelheim
- Primary completion
- Apr 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Phase Ia Dose Escalation: Number of Participants With Dose-limiting Toxicities (DLTs) in the First Cycle (3 Weeks) |
0; 0; 0 | — |
| PRIMARY Phase Ib Dose Expansion: Number of Participants With Dose-limiting Toxicities (DLTs) During the Entire Treatment Period |
0; 0; 0; 0 | — |
| PRIMARY Phase Ib Dose Expansion: Confirmed Objective Response (OR), Defined as the Best Overall Response of Confirmed Complete Response (CR) or Partial Response (PR) According to RECIST v1.1 as Assessed by the Investigator |
4; 4; 6; 2 | — |
| SECONDARY Phase Ia Dose Escalation: Confirmed Objective Response According to RECIST v.1.1 as Assessed by the Investigator |
0; 1; 0 | — |
| SECONDARY Phase Ia Dose Escalation: Maximum Measured Concentration (Cmax) of Ezabenlimab in Plasma |
25.3; 73.7; 128; 33.3; 96.4; 144 | — |
| SECONDARY Phase Ia Dose Escalation: Area Under the Concentration-time Curve of Ezabenlimab in Plasma Over the Time Interval From 0 to 504 Hours (AUC0-504) |
4640; 14300; 22000; 6790; 18800; 28500 | — |
| SECONDARY Phase Ia Dose Escalation: Number of Participants Experiencing Dose-limiting Toxicities (DLTs) From the Start of Treatment Until End of Treatment |
0; 0; 0 | — |
| SECONDARY Phase Ib Dose Expansion: Confirmed Progression-free Survival (PFS) Defined From Date of Start of Ezabenlimab to the Date of Disease Progression or Death, Whichever Was Earlier, According to RECIST v1.1 as Assessed by the Investigator |
1.9; 2.7; 2.6; 4.2 | — |
| SECONDARY Phase Ib Dose Expansion: Percentage of Participants With Adverse Events (AEs) |
96.7; 96.3; 96.8; 100.0 | — |
| SECONDARY Phase Ib Dose Expansion: Percentage of Participants With Serious Adverse Events (SAEs) |
33.3; 25.9; 48.4; 46.2 | — |
| SECONDARY Phase Ib Dose Expansion: Percentage of Participants With Clinically Relevant Abnormalities in Laboratory Evaluations |
3.3; 3.7; 0.0; 0.0; 0.0; 11.1 | — |
Summary
The main objective of the dose-escalation part of the trial is to determine the safety and tolerability, and to determine the Maximum Tolerated Dose and/or the Recommended Phase 2 Dose (RP2D) of BI 754091 on the basis of patients with dose-limiting toxicities (DLTs) in patients with selected advanced solid malignancies. Safety and tolerability will be evaluated by monitoring the occurrence of adverse events (AEs), serious AEs (SAE), and laboratory parameter abnormalities, as well as changes to vital signs.
Secondary objectives are the determination of the PK profile of BI 754091 after single and multiple doses of BI 754091, and the preliminary assessment of antitumour activity.
In the dose-expansion part of the trial, the main objectives are to further assess the safety, efficacy, PK profile, and biomarkers of BI 754091 in tumours with specific tumour types and/or genetic mutations at the RP2D.
Eligibility Criteria
Inclusion Criteria
- Provision of signed and dated, written Informed Consent Form (ICF) prior to any trial-specific procedures, sampling, or analyses. If a patient declines to participate in the voluntary pharmacogenetics component of the trial, he/she will not be excluded from other aspects of the trial.
- Patients ≥18 years of age at the time of signature of the ICF
- Phase Ia (dose-escalation)
- patients with a histologically confirmed diagnosis of advanced, unresectable, and/or metastatic solid tumours (any type).
- patients who have received all therapy known to confer clinical benefit (including anti-PD-1 or anti-PDL1 therapies, if relevant), or for whom no therapy of proven efficacy exists, or who are not amenable to standard therapies. Patients with anti- PD-1 or anti-PDL1 experience must have a minimum of 60 days between the last dose of the previous anti PD-1/PD-L1 and Cycle 1 Day 1 of BI 754091 treatment.
- Patients may agree to provide optional paired biopsies.
- Phase Ib (dose expansion)
- patients with a histologically confirmed diagnosis of select advanced, unresectable, and/or metastatic solid tumours with either 1) high tumor mutation excluding high microsatellite instability or 2) refractory squamous cell cervical, anal and skin tumors, or 3) recurrent vaginal or vulvar squamous cell carcinoma.
- All patients must have measurable lesions according to Response Evaluation Criteria in Solid Tumours (RECIST) v1.1 must have at least 1 tumour lesion amenable to biopsy, and must be medically fit and willing to undergo a biopsy before first treatment and, unless clinically contraindicated, after 6 weeks on therapy
- patients who are anti-PD-1 and anti-PDL-1naïve but have failed conventional treatment (excluding anti-PD-1 treatment), or for whom no therapy of proven efficacy exists, or who are not amenable to standard therapies.
- Eastern Cooperative Oncology Group (ECOG) score: 0 to 1
- Life expectancy of at least 12 weeks after the start of the treatment according to the Investigator's judgement
- Females of child-bearing potential willing to use adequate contraceptive measures from the time of screening until 6 months after trial discontinuation, who are not or will not be breast feeding, and agree to have pregnancy tests prior to the start of dosing and at regular visits during the trial. Females not of childbearing potential must have evidence of such by fulfilling one of the following criteria at screening:
- Post-menopausal: defined as more than 50 years-of-age and amenorrhoeic for at least 12 months following cessation of all exogenous hormonal treatments
- Documentation of irreversible surgical sterilization by hysterectomy, bilateral oophorectomy, or bilateral salpingectomy
- Women under 50 years-of-age would be considered postmenopausal if they have been amenorrhoeic for at least 12 months following the cessation of exogenous hormonal treatments, and have serum follicle-stimulating hormone and luteinizing hormone levels in the postmenopausal range for the institution.
- For women of childbearing potential using a contraceptive pill, an additional barrier method is necessary. Acceptable highly effective methods of contraception include total sexual abstinence when this is in line with the preferred and usual lifestyle of the study participant (periodic abstinence such as calendar, ovulation, symptothermal, post-ovulation methods and withdrawal are not acceptable methods of contraception), an intrauterine device or intrauterine hormone-releasing system, bilateral tubal ligation, and vasectomised partner (with post-vasectomy proof of absence of sperm)
- Further inclusion criteria apply
Exclusion criteria
- Major surgery (major according to the Investigator's assessment) performed within 12 weeks prior to first trial treatment or planned within 12 months after screening, e.g.,hip replacement
- Patients who must or wish to continue the intake of restricted medications or any drug considered likely to int
Data sourced from ClinicalTrials.gov (NCT02952248). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.