Testing A New Anti-cancer Drug Combination, Entinostat and ZEN003694, for Advanced and Refractory Solid Tumors

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorNational Cancer Institute (NCI)

About this trial

This phase I/II trial tests the safety, side effects, and best dose of entinostat and ZEN003694 in treating patients with solid tumors that have spread to other places in the body (advanced) or does not respond to treatment (refractory). Entinostat is in a class of drugs called histone deacetylase (HDAC) inhibitors. It may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. ZEN003694 is an inhibitor of a family of proteins called the bromodomain and extra-terminal (BET). It may prevent the growth of tumor cells that over produce BET protein. This trial aims to test the safety of combination therapy with entinostat and ZEN003694 in treating patients with advanced or refractory solid tumors.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients must have a) advanced or refractory solid tumor and must meet standard requirements for treatment

For patients in Phase 2: Patients must have locally advanced, unresectable OR metastatic pancreatic cancer refractory to standard therapy

For patients with solid tumors, they must have measurable disease based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria

For patients with solid tumors, they must have received at least one standard of care regimen for metastatic disease

Disqualifiers

Patients who have had any anti-cancer therapy within 30 days (or 5 half-lives, whichever is shorter) prior to the first dose of the investigational products

Patients who have received radiation therapy within 21 days prior to the first dose of the investigational products

Patients who have a diagnosis of NK cell lymphoma

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, or stable chronic grade 2 toxicities that do not overlap with presumed toxicities of entinostat or ZEN003694

Trial design

Design model

Sequential

Treatments tested in this trial

  • BET Bromodomain Inhibitor ZEN-3694

    Drug

    Given PO

  • Computed Tomography

    Procedure/Surgery

    Undergo CT

  • Core Biopsy

    Procedure/Surgery

    Undergo core needle biopsy

  • Entinostat

    Drug

    Given PO

Treatment groups

49 Participants
are divided into 1 treatment group
Group A: Treatment (entinostat, ZEN003694)Experimental treatment 4 interventions

Trial outcomes

Primary outcomes

1

Maximum tolerated dose (MTD) (Phase Ib)

The MTD is defined as the highest dose level at which \< 33% of the dose cohort (0 of 3 or 1 of 6) experience a dose-limiting toxicity (DLT) in the first cycle. Up to 3 additional patients (maximum enrollment 6) will be added at the MTD level to more fully characterize the safety of the drug combination. If \< 33% (2) patients in this expanded cohort experience a DLT, this will be declared the MTD, and thus the phase 2 dose. If 2 or more patients experience a DLT, this dose level will be adopted as the maximum administered dose (MAD) and drop to the dose level immediately below, for the MTD and the phase 2 dose.

Time frame
Up to 28 days
2

Recommended phase 2 dose (RP2D) (Phase Ib)

The RP2D is generally defined as =\<1 out of 6 at highest dose level below the maximally administered dose.

Time frame
Up to 28 days
3

Objective response rate (ORR) (Phase II)

Each patient will be assigned one of the following categories: 1) complete response, 2) partial response, 3) stable disease, 4) progressive disease, 5) early death from malignant disease, 6) early death from toxicity, 7) early death because of other cause, or 9) unknown (not assessable, insufficient data). All of the patients who met the eligibility criteria (with the possible exception of those who received no study medication) should be included in the main analysis of the response rate. Patients in response categories 4-9 should be considered to have a treatment failure (disease progression). Thus, an incorrect treatment schedule or drug administration does not result in exclusion from the analysis of the response rate. Precise definitions for categories 4-9 will be protocol specific.

Time frame
Up to 4 weeks post intervention

Secondary outcomes

1

Response rate (RR) in patients with advanced/progressive pancreatic cancer (Phase II)

The exact two-sided 95% confidence intervals (CIs) for the RR will be reported.

Time frame
Up to 4 weeks post intervention
2

Duration of response

The 95% CIs for the duration of response will be reported.

Time frame
Up to 4 weeks post intervention
3

Overall survival

Will be estimated using the Kaplan-Meier method with the 95% CIs. The CI based on Greenwood's variance will be reported. In addition, the possible risk factors will be compared for survival with log-rank test. In addition, the 95% CIs for the duration of response will be reported.

Time frame
Up to 4 weeks post intervention
4

Progression free survival

Will be estimated using the Kaplan-Meier method with the 95% CIs. The CI based on Greenwood's variance will be reported. In addition, the possible risk factors will be compared for survival with log-rank test. In addition, the 95% CIs for the duration of response will be reported.

Time frame
Up to 4 weeks post intervention

Other outcomes

1

Non-sequencing biomarker analysis

Statistical and bioinformatic data analysis for laboratory non-omics data and clinical data will utilize the following general strategies, as appropriate. For single time-point lab data, tests of hypotheses concerning within-group comparisons will be completed using the paired t-test or Wilcoxon signed-rank test for continuous parameters of interest, or McNemar's Chi-square test for categorical parameters of interest. Between-group comparisons will be assessed using either analysis of variance (ANOVA) with adjusted least squares means or Fisher's exact test, for continuous or categorical variables of interest, respectively. For count or binary multiple time-points or correlated data, tests of between-group comparisons will be completed using the generalized estimating equation (GEE) statistical procedure for longitudinal data analysis with multiple observable vectors for the same subject.

Time frame
Up to 4 weeks post intervention

Sponsors and contacts

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