Clinical Trials

Semaxanib (SU5416) has been evaluated across numerous clinical trials spanning early- to late-phase development for solid tumors and hematological malignancies, both as a monotherapy and combined with chemotherapeutics like paclitaxel or doxorubicin. Sponsored by major entities including the National Cancer Institute, the Pediatric Brain Tumor Consortium, and the Gynecologic Oncology Group, these studies encompassed central nervous system tumors, head and neck carcinomas, melanoma, cervical and breast cancers, acute myeloid leukemia, and myelodysplastic syndromes. Although several clinical trials successfully completed early pharmacokinetic testing, other evaluations were ultimately terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT00004252 COMPLETED
Colorectal Cancer
Pfizer
 1999-11 PHASE3
NCT00006014 COMPLETED
Malignant Mesothelioma
National Cancer Institute (NCI)
 2000-08 PHASE2
NCT00006001 TERMINATED
Colorectal Cancer
National Cancer Institute (NCI)
 2000-08 PHASE2
NCT00006384 COMPLETED
Kidney Cancer
City of Hope Medical Center
 2000-11 PHASE2
NCT00004868 COMPLETED
Brain and Central Nervous System Tumors
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2000-03-24 PHASE1; PHASE2
NCT00005862 COMPLETED
Gastrointestinal Stromal Tumor; Sarcoma
National Cancer Institute (NCI)
 2000-10 PHASE2
NCT00017316 COMPLETED
Melanoma (Skin)
National Cancer Institute (NCI)
 2001-03 PHASE2
NCT00006003 TERMINATED
Recurrent Melanoma; Stage IV Melanoma
National Cancer Institute (NCI)
 2000-07 PHASE2
NCT00006247 TERMINATED
Brain and Central Nervous System Tumors
Pediatric Brain Tumor Consortium
 2000-08 PHASE1
NCT00009919 TERMINATED
Recurrent Renal Cell Cancer; Stage IV Renal Cell Cancer
National Cancer Institute (NCI)
 2000-12 PHASE2
NCT00005818 COMPLETED
Adenocarcinoma of the Colon; Adenocarcinoma of the Rectum; Recurrent Colon Cancer; Recurrent Rectal Cancer; Stage III Colon Cancer; Stage III Rectal Cancer; Stage IV Colon Cancer; Stage IV Rectal Cancer
National Cancer Institute (NCI)
 2000-03 PHASE1; PHASE2
NCT00006361 COMPLETED
Carcinoma of Unknown Primary; Head and Neck Cancer; Non-melanomatous Skin Cancer
Memorial Sloan Kettering Cancer Center
 2000-12 PHASE2
NCT00006013 COMPLETED
Multiple Myeloma and Plasma Cell Neoplasm
Fox Chase Cancer Center
 2000-06 PHASE2
NCT00005822 COMPLETED
Breast Cancer
Case Comprehensive Cancer Center
 2000-04 PHASE1
NCT00005647 COMPLETED
Head and Neck Cancer
Case Comprehensive Cancer Center
 2000-05 PHASE1
NCT00005642 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
Case Comprehensive Cancer Center
 2000-05 PHASE1
NCT00006002 COMPLETED
Prostate Cancer
University of Chicago
 2000-06 PHASE2
NCT00026377 COMPLETED
Prostate Cancer
University of Chicago
 2001-11 PHASE1
NCT00005942 COMPLETED
Chronic Myelomonocytic Leukemia; Previously Treated Myelodysplastic Syndromes; Recurrent Adult Acute Myeloid Leukemia; Refractory Anemia With Excess Blasts; Refractory Anemia With Excess Blasts in Transformation
National Cancer Institute (NCI)
 2000-03 PHASE1; PHASE2
NCT00005042 COMPLETED
Sarcoma
AIDS Malignancy Consortium
 2000-01 PHASE2
NCT00006247 Terminated
Brain and Central Nervous System Tumors
Pediatric Brain Tumor Consortium|National Cancer Institute (NCI)
 2000-08 Phase 1
NCT00005642 Completed
Unspecified Adult Solid Tumor Protocol Specific
Case Comprehensive Cancer Center|National Cancer Institute (NCI)
 2000-05 Phase 1
NCT00005647 Completed
Head and Neck Cancer
Case Comprehensive Cancer Center|National Cancer Institute (NCI)
 2000-05 Phase 1
NCT00026260 COMPLETED
Cervical Cancer
Gynecologic Oncology Group
 PHASE2
NCT00023738 COMPLETED
Sarcoma
Radiation Therapy Oncology Group
 2001-08 PHASE1; PHASE2
NCT00006000 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
Ohio State University Comprehensive Cancer Center
 2000-08 PHASE1
NCT00006257 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
City of Hope Medical Center
 2000-11 PHASE1
NCT00003720 COMPLETED
Sarcoma
Pfizer
 1998-07 PHASE1
NCT00021281 UNKNOWN
Colorectal Cancer
Jonsson Comprehensive Cancer Center
 2000-12 PHASE3
NCT00005996 UNKNOWN
Unspecified Adult Solid Tumor, Protocol Specific
Jonsson Comprehensive Cancer Center
 PHASE1
NCT00023725 COMPLETED
Sarcoma
Radiation Therapy Oncology Group
 2001-08 PHASE1; PHASE2
NCT00006155 COMPLETED
Fallopian Tube Neoplasm; Ovarian Cancer; Peritoneal Neoplasm
National Cancer Institute (NCI)
 2000-08 PHASE1
NCT00005931 COMPLETED
Sarcoma, Kaposi; HIV Infections
SUGEN
 PHASE2
NCT00002226 COMPLETED
Sarcoma, Kaposi; HIV Infections
SUGEN

(data from https://clinicaltrials.gov, updated on 2012-09-11)

Check the Semaxanib (SU5416) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Semaxanib selectively binds to vascular endothelial growth factor receptor (VEGFR/Flk-1/KDR), blocking receptor autophosphorylation and downstream signal transduction necessary for vascular endothelial cell proliferation and migration. By disrupting this angiogenic cascade, the compound suppresses new blood vessel formation to inhibit tumor growth, providing therapeutic rationale for its clinical investigation in solid tumors, central nervous system malignancies, and hematologic cancers.

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.