Clinical Trials

Multiple clinical trials investigate targeted therapeutic and diagnostic approaches for conditions including NGLY1 deficiency, uveal melanoma, bile duct neoplasms, intraductal papillary mucinous neoplasms, schizophrenia, and schizoaffective disorder. Encompassing early phase 1, combined phase 1/2, and non-interventional designs, these studies report recruiting, completed, and unknown recruitment statuses. The trials are sponsored by diverse academic and commercial entities, including Grace Science LLC, Johns Hopkins University, Institut Curie, the University of Catania, and Fujian Medical University Union Hospital.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06199531 Recruiting
NGLY1 Deficiency
Grace Science LLC
 2024-02-13 Phase 1|Phase 2
NCT05179174 Unknown status
Uveal Melanoma
University of Catania
 2021-04-20 --
NCT03838913 Unknown status
Bile Duct Neoplasms|Intraductal Papillary Mucinous Neoplasm
Fujian Provincial Hospital|Fujian Medical University Union Hospital|First Affiliated Hospital of Fujian Medical University
 2019-02-15 --
NCT03671525 Completed
Schizophrenia|Schizo Affective Disorder
Johns Hopkins University
 2018-10-05 Early Phase 1
NCT02849145 Completed
Uveal Melanoma
Institut Curie
 2014-09 Not Applicable

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the Gambogenic acid product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Gambogenic acid specifically and covalently binds to Cys668 within the SET domain of enhancer of zeste homolog 2 (EZH2) to induce EZH2 ubiquitination while simultaneously inhibiting fibroblast growth factor receptor (FGFR) pathway signaling. This combined activity suppresses downstream oncogenic gene transcription and arrests tumor cell proliferation, demonstrating potential therapeutic application in non-small-cell lung cancer and other oncological conditions studied in clinical trials, such as uveal melanoma and bile duct neoplasms.

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.