Clinical Trials

Roblitinib (FGF401) has been evaluated in multiple clinical trials sponsored by Novartis Pharmaceuticals for advanced oncological indications, including hepatocellular carcinoma and biomarker-positive solid malignancies. These completed Phase I and Phase II studies evaluated the safety, tolerability, and preliminary efficacy of oral FGFR4 inhibition in adult patients. Collectively, these trials provide foundational clinical data regarding targeted therapy in liver and solid tumors.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02325739 COMPLETED
Hepatocellular Carcinoma (HCC); Solid Malignancies
Novartis Pharmaceuticals
 2014-12-29 PHASE1; PHASE2
NCT02325739 Completed
Hepatocellular Carcinoma (HCC)|Solid Malignancies
Novartis Pharmaceuticals|Novartis
 2014-12-29 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2024-10-21)

Check the Roblitinib (FGF401) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Roblitinib (FGF401) binds to the FGFR4 kinase domain in a reversible covalent manner with an IC50 of 1.9 nM, effectively blocking receptor autophosphorylation and downstream biochemical signaling cascades. This targeted enzymatic inhibition suppresses cellular proliferation and induces apoptosis, providing clinical relevance for inhibiting tumor growth in FGFR4-driven malignancies such as hepatocellular carcinoma and advanced solid tumors.

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>).

Jump to

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