Clinical Trials

Numerous clinical trials, predominantly Phase 1 and Phase 2 studies, have evaluated mocetinostat across hematologic malignancies and solid tumors, including urothelial carcinoma, acute myeloid leukemia, and rhabdomyosarcoma. Sponsored by academic medical centers and pharmaceutical entities, the portfolio encompasses active, recruiting, completed, terminated, and withdrawn studies. Highlighted investigations include a completed Phase 2 study in urothelial carcinoma and an ongoing Phase 1 trial in rhabdomyosarcoma.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03220477 ACTIVE_NOT_RECRUITING
Lung Cancer
Memorial Sloan Kettering Cancer Center
 2017-08-04 PHASE1
NCT04299113 ACTIVE_NOT_RECRUITING
Rhabdomyosarcoma
Jonsson Comprehensive Cancer Center
 2020-05-14 PHASE1
NCT02282358 TERMINATED
Lymphoma; Relapsed and Refractory; Diffuse Large B-Cell Lymphoma and Follicular Lymphoma
Memorial Sloan Kettering Cancer Center
 2014-10 PHASE1; PHASE2
NCT02954991 TERMINATED
Carcinoma, Non-Small-Cell Lung
Mirati Therapeutics Inc.
 2016-11-07 PHASE2
NCT02429375 COMPLETED
Hodgkin Lymphoma
Memorial Sloan Kettering Cancer Center
 2015-04-22 PHASE1; PHASE2
NCT04299113 Recruiting
Rhabdomyosarcoma
Jonsson Comprehensive Cancer Center|Mirati Therapeutics Inc.|Phase One Foundation
 2020-05-14 Phase 1
NCT03565406 TERMINATED
Melanoma
NYU Langone Health
 2018-04-25 PHASE1
NCT02805660 TERMINATED
Advanced Cancer
Mirati Therapeutics Inc.
 2016-06-01 PHASE1; PHASE2
NCT02993991 WITHDRAWN
Squamous Cell Carcinoma, Head And Neck; Squamous Cell Carcinoma Mouth; Resectable Squamous Cell Carcinoma of Oral Cavity
University Health Network, Toronto
 2017-10-10 PHASE1
NCT02993991 Withdrawn
Squamous Cell Carcinoma Head And Neck|Squamous Cell Carcinoma Mouth|Resectable Squamous Cell Carcinoma of Oral Cavity
University Health Network Toronto|Mirati Therapeutics Inc.|AstraZeneca
 2017-10-10 Phase 1
NCT02303262 COMPLETED
Metastatic Leiomyosarcoma
Sarcoma Alliance for Research through Collaboration
 2015-11-12 PHASE2
NCT02236195 COMPLETED
Urothelial Carcinoma
Mirati Therapeutics Inc.
 2014-10 PHASE2
NCT02018926 COMPLETED
Myelodysplastic Syndrome
Mirati Therapeutics Inc.
 2013-12 PHASE1; PHASE2
NCT02236195 Completed
Urothelial Carcinoma
Mirati Therapeutics Inc.
 2014-10 Phase 2
NCT00359086 COMPLETED
Lymphoma
Mirati Therapeutics Inc.
 2006-08 PHASE2
NCT00666497 TERMINATED
Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)
Mirati Therapeutics Inc.
 2008-06 PHASE2
NCT00543582 TERMINATED
Hodgkin Lymphoma; Non-Hodgkin Lymphoma (Follicular or Large Diffuse B-cell Lymphoma or Mantle Cell Lymphoma)
Mirati Therapeutics Inc.
 2007-10 PHASE2
NCT00511576 TERMINATED
Breast Cancer; Lung Cancer; Pulmonary Cancer; Non-Small-Cell Lung Carcinoma; Prostate Cancer; Prostatic Cancer; Gastric Cancer; Stomach Cancer
Mirati Therapeutics Inc.
 2007-08 PHASE1
NCT00358982 TERMINATED
Hodgkin's Lymphoma
Mirati Therapeutics Inc.
 2006-08 PHASE2
NCT00372437 COMPLETED
Tumors
Mirati Therapeutics Inc.
 2006-09 PHASE1; PHASE2
NCT00324220 COMPLETED
Myelodysplastic Syndrome; Acute Myelogenous Leukemia
Mirati Therapeutics Inc.
 2006-01 PHASE1; PHASE2
NCT00431873 COMPLETED
Lymphocytic Leukemia, Chronic
Mirati Therapeutics Inc.
 2007-01 PHASE2
NCT00374296 TERMINATED
Myelogenous Leukemia, Acute; Myelodysplastic Syndromes
Mirati Therapeutics Inc.
 2006-09 PHASE2
NCT00666497 Terminated
Acute Myeloid Leukemia (AML)|Myelodysplastic Syndrome (MDS)
Mirati Therapeutics Inc.
 2008-06 Phase 2
NCT00324194 COMPLETED
Leukemia; Myelodysplastic Syndromes
Mirati Therapeutics Inc.
 2005-02 PHASE1
NCT00323934 COMPLETED
Tumors; Non Hodgkin's Lymphoma
Mirati Therapeutics Inc.
 2004-04 PHASE1
NCT00511576 Terminated
Breast Cancer|Lung Cancer|Pulmonary Cancer|Non-Small-Cell Lung Carcinoma|Prostate Cancer|Prostatic Cancer|Gastric Cancer|Stomach Cancer
Mirati Therapeutics Inc.
 2007-08 Phase 1
NCT00324129 COMPLETED
Leukemia; Myelodysplastic Syndromes
Mirati Therapeutics Inc.
 2005-02 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-08-05)

Check the Mocetinostat (MGCD0103) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mocetinostat potently and selectively binds to and inhibits class I histone deacetylases, specifically HDAC1, HDAC2, and HDAC3, thereby blocking histone deacetylation and inducing chromatin remodeling and histone hyperacetylation. This biochemical blockade leads to cell cycle arrest and apoptosis in malignant cells, providing the therapeutic rationale for its clinical evaluation in hematologic malignancies and solid tumors such as urothelial carcinoma, Hodgkin lymphoma, and non-small-cell lung carcinoma.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.