Safety Study of Mocetinostat in Combination With Azacitidine in Subjects With MDS

A Phase I/II, Multi-Center Study of Mocetinostat (MGCD0103) in Combination With Azacitidine in Subjects With Intermediate or High Risk Myelodysplastic Syndrome (MDS)

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02018926

Enrollment

Registered

2013-12-23

Start date

2013-12-31

Completion date

2015-09-30

Last updated

2017-09-14

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

Conditions

Myelodysplastic Syndrome

Keywords

Mocetinostat, Azacitidine, Vidaza, Myelodysplastic syndrome, HDAC, HMA, MDS

Brief summary

Mocetinostat is an orally administered histone deacetylase (HDAC) inhibitor. Azacitidine is a hypomethylating agent (HMA) used to treat MDS. In this study, patients with intermediate- or high-risk MDS will receive treatment with mocetinostat and azacitidine to evaluate the safety of the study treatment. Safety assessments will include echocardiograms, electrocardiograms and routine safety laboratory studies (hematology and serum chemistry). In addition, clinical response to treatment will be monitored using bone marrow aspirates or biopsies, and other routine methods.

Detailed description

The study will include multiple cohorts of patients. In the first cohort, patients may have previously received treatment with hypomethylating agents such as azacitidine. In later cohorts, prior treatment with this class of anti-cancer agents will be excluded. Later cohorts will include patients that are receiving this class of agents, specifically azacitidine, for the first time.

Interventions

DRUGMocetinostat

Mocetinostat (a histone deacetylase \[HDAC\] inhibitor) 70 mg or 90 mg dose, oral capsules 3 times weekly beginning on day 5, for 10 doses in each 28 day cycle

DRUGAzacitidine

Azacitidine (a hypomethylating agent \[HMA\]) 75 mg/m2 dose, by intravenous (IV) infusion or subcutaneous (SC) injection beginning on day 1 for 7 doses in each 28 day cycle

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 intermediate- or high-risk (IPSS criteria) myelodysplastic syndrome. Cohort 1: Any prior treatment, enrollment complete. Cohort 2: Limited or no prior treatment for MDS. Prior treatment should not include hypomethylating agents such as azacitidine or decitabine, or HDAC inhibitors. ECOG Performance Status 0 or 1.

Exclusion criteria

Current or history of small, moderate or large pericardial effusion, tamponade and/or pericarditis. Significant cardiac abnormalities such as recent myocardial infarction, congestive heart failure ≥ Class 3, or symptomatic, uncontrolled atrial fibrillation, atrial flutter or sinus tachycardia. Prolonged QT/QTc interval. Other active cancer excluding basal cell carcinoma or cervical intraepithelial neoplasia.

Design outcomes

Primary

Measure	Time frame
Number of subjects with adverse events, including pericardial events, as a measure of safety	6 months

Secondary

Measure	Time frame
Number of subjects experiencing clinical disease response	1 year

Countries

United States

Outcome results

None listed

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