Study of Mocetinostat in Patients With Urothelial Carcinoma Having Inactivating Alterations of Specific Genes

An Open-Label, Single-Arm, Phase 2 Study of Mocetinostat in Selected Patients With Inactivating Alterations of Acetyltransferase Genes in Previously Treated Locally Advanced or Metastatic Urothelial Carcinoma

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02236195

Enrollment

Registered

2014-09-10

Start date

2014-10-31

Completion date

2016-07-31

Last updated

2017-09-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Urothelial Carcinoma

Keywords

Urothelial, Bladder, HDAC, MGCD0103, Mocetinostat, CREBBP, EP300, Mirati, MethylGene

Brief summary

Mocetinostat is an orally administered histone deacetylase (HDAC) inhibitor. This study is a Phase 2 trial evaluating the efficacy of mocetinostat in patients that have advanced urothelial carcinoma that has specific changes in tumor genes. Patients must have previously received treatment with chemotherapy that included a platinum-containing agent such as cisplatin. The study will enroll in stages, with 15 patients in the first stage. More patients will be added to the study if enough patients having beneficial responses are observed. Mocetinostat will be administered using oral capsules three times each week (eg, Monday, Wednesday and Friday). The study is designed to evaluate whether the number of patients responding to treatment is substantially higher than would be expected with other available treatments.

Detailed description

To be eligible for this study, patient tumor testing must demonstrate a genetic alteration resulting in the loss of function of the Histone Acetyltransferase (HAT) family genes, CREBBP and/or EP300, including gene deletions or selected inactivating mutations. If testing has not already been performed, the study will provide for the testing.

Interventions

DRUGMocetinostat

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Diagnosis of urothelial carcinoma * Metastatic or locally advanced disease * Prior chemotherapy that included a platinum agent * Test results showing genetic change in tumor gene for CREBBP and/or EP300 * At least one tumor that can be measured

Exclusion criteria

* Uncontrolled tumor in the brain * Impaired heart function

Design outcomes

Primary

Measure	Time frame	Description
Number of patients experiencing tumor size reduction	Up to 4 months	Tumors will be measured using radiographic scans. Tumor size reduction and overall disease response will be categorized according to the Response Evaluation Criteria In Solid Tumors (RECIST 1.1).

Secondary

Measure	Time frame
Number of patients experiencing adverse events	Up to 12 months
Peak blood plasma concentration of mocetinostat	Up to 48 hours

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 4, 2026