Clinical Trials

Multiple Phase 1 and Phase 2 clinical trials sponsored by Sumitomo Pharma America, Inc. and Sumitomo Pharma Co., Ltd. have evaluated Amcasertib as both monotherapy and combination therapy for various oncological indications. The clinical program targeted advanced solid tumors, gastrointestinal stromal tumors, hepatobiliary cancers including hepatocellular carcinoma and cholangiocarcinoma, and urologic malignancies such as renal cell carcinoma. Although several trials were successfully completed, others evaluating specific advanced malignancies were ultimately terminated or withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT01781455 COMPLETED
Cancer, Advanced Solid Tumors
Sumitomo Pharma America, Inc.
 2012-02 PHASE1
NCT02432326 COMPLETED
Cancer; Advanced Solid Tumors
Sumitomo Pharma America, Inc.
 2015-04 PHASE1
NCT02279719 COMPLETED
Hepatocellular Carcinoma
Sumitomo Pharma America, Inc.
 2014-12 PHASE1; PHASE2
NCT02483247 COMPLETED
Cancer
Sumitomo Pharma America, Inc.
 2015-09 PHASE1
NCT02354898 COMPLETED
Advanced Solid Tumors; Hepatocellular Carcinoma
Sumitomo Pharma Co., Ltd.
 2015-03 PHASE1
NCT02232646 WITHDRAWN
Urologic Malignancies; Renal Cell Carcinoma; Urothelial Carcinoma
Sumitomo Pharma America, Inc.
 2017-01 PHASE2
NCT02232633 COMPLETED
Hepatocellular Carcinoma; Cholangiocarcinoma
Sumitomo Pharma America, Inc.
 2015-02 PHASE2
NCT02232620 TERMINATED
Gastrointestinal Stromal Tumors
Sumitomo Pharma America, Inc.
 2017-03-13 PHASE2
NCT02232620 Terminated
Gastrointestinal Stromal Tumors
Sumitomo Pharma America Inc.
 2017-03-13 Phase 2
NCT02432690 TERMINATED
Cancer; Ovarian Cancer
Sumitomo Pharma America, Inc.
 2015-06 PHASE2
NCT02232646 Withdrawn
Urologic Malignancies|Renal Cell Carcinoma|Urothelial Carcinoma
Sumitomo Pharma America Inc.
 2017-01 Phase 2
NCT02432326 Completed
Cancer|Advanced Solid Tumors
Sumitomo Pharma America Inc.
 2015-04 Phase 1
NCT02354898 Completed
Advanced Solid Tumors|Hepatocellular Carcinoma
Sumitomo Pharma Co. Ltd.
 2015-03 Phase 1
NCT02232633 Completed
Hepatocellular Carcinoma|Cholangiocarcinoma
Sumitomo Pharma America Inc.
 2015-02 Phase 2

(data from https://clinicaltrials.gov, updated on 2023-11-14)

Check the Amcasertib (BBI503) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Amcasertib selectively inhibits cancer stemness kinases, suppressing downstream Nanog expression and key self-renewal pathways to induce cancer stem cell depletion. By blocking stemness-mediated driver mechanisms, the compound suppresses tumor progression and drug resistance in advanced solid malignancies, such as hepatocellular carcinoma and gastrointestinal stromal 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>).

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