Clinical Trials

Multiple early-stage Phase 1 and Phase 1a/1b clinical trials have evaluated citarinostat (ACY-241) across oncological indications including malignant melanoma, non-small cell lung cancer, advanced solid tumors, multiple myeloma, and smoldering multiple myeloma. Sponsored by both industry entities and academic institutions, these studies encompass completed, active not recruiting, and terminated recruitment statuses. The trials primarily evaluate the safety, pharmacokinetics, and preliminary therapeutic activity of citarinostat in monotherapy and combination regimens.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02886065 ACTIVE_NOT_RECRUITING
Smoldering Multiple Myeloma
Massachusetts General Hospital
 2017-03-07 PHASE1
NCT02400242 TERMINATED
Multiple Myeloma
Celgene
 2015-05-07 PHASE1
NCT02635061 TERMINATED
Non Small Cell Lung Cancer
Celgene
 2016-08-25 PHASE1
NCT02551185 COMPLETED
Advanced Solid Tumors
Celgene
 2015-12-22 PHASE1
NCT02935790 COMPLETED
Malignant Melanoma
Celgene
 2016-09-30 PHASE1
NCT02935790 Completed
Malignant Melanoma
Celgene|Syneos Health|ApoCell Inc.|Celerion|NYU Langone Health
 2016-09-30 Phase 1
NCT02635061 Terminated
Non Small Cell Lung Cancer
Celgene
 2016-08-25 Phase 1
NCT02551185 Completed
Advanced Solid Tumors
Celgene
 2015-12-22 Phase 1
NCT02400242 Active not recruiting
Multiple Myeloma
Celgene
 2015-05-07 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-03-13)

Check the Citarinostat (ACY-241) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Citarinostat selectively binds to and inhibits histone deacetylase 6 (HDAC6), blocking the deacetylation of cytoplasmic substrates like alpha-tubulin and disrupting intracellular protein homeostasis. This biochemical blockade impairs aggresome pathway function and induces apoptotic cell death, underlying its clinical evaluation in suppressing tumor cell proliferation across hematologic malignancies and solid tumors, such as multiple myeloma and non-small cell lung cancer.

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.