Clinical Trials

Multiple clinical trials evaluate the therapeutic utility of gossypol (including AT-101) across Phase 1, Phase 2, and Phase 3 stages for diverse malignancies, including adrenocortical carcinoma, non-small cell lung cancer, small cell lung cancer, metastatic colorectal cancer, glioblastoma, and hematologic cancers. Supported by academic and government sponsors such as the National Cancer Institute and Mayo Clinic, these investigations encompass completed studies, terminated protocols, and upcoming Phase 2 trials that are not yet recruiting.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07337551 NOT_YET_RECRUITING
Patients With Metastatic Colorectal Cancer Who Were TP53-mutant and LRPPRC-positive and Had Previously Failed Prior First-line Treatment
Guiying Wang
 2026-02-01 PHASE2
NCT07656766 NOT_YET_RECRUITING
Advanced Colorectal Cancer
Peking University People's Hospital
 2026-07-15 PHASE2
NCT06133088 UNKNOWN
Advanced HR-positive and HER2-negative Breast Cancer; CDK4/6 Inhibitor Treatment Failed
Zhejiang Cancer Hospital
 2023-08-01 PHASE2
NCT01003769 TERMINATED
Recurrent Chronic Lymphocytic Leukemia
Mayo Clinic
 2015-07-09 PHASE1; PHASE2
NCT02697344 COMPLETED
Recurrent Plasma Cell Myeloma
Mayo Clinic
 2016-04-14 PHASE1
NCT01977209 UNKNOWN
Non-small Cell Lung Cancer
Third Military Medical University
 2013-09 PHASE3
NCT00544596 COMPLETED
Extensive Stage Small Cell Lung Cancer; Unspecified Adult Solid Tumor, Protocol Specific
National Cancer Institute (NCI)
 2007-09 PHASE1
NCT00540722 COMPLETED
Adult Giant Cell Glioblastoma; Adult Glioblastoma; Adult Gliosarcoma; Recurrent Adult Brain Tumor
National Cancer Institute (NCI)
 2008-01 PHASE2
NCT00848016 COMPLETED
Recurrent Adrenocortical Carcinoma; Stage III Adrenocortical Carcinoma; Stage IV Adrenocortical Carcinoma
National Cancer Institute (NCI)
 2009-02 PHASE2
NCT00891072 COMPLETED
Adult Grade III Lymphomatoid Granulomatosis; Adult Nasal Type Extranodal NK/T-cell Lymphoma; Contiguous Stage II Adult Burkitt Lymphoma; Contiguous Stage II Adult Diffuse Large Cell Lymphoma; Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma; Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma; Contiguous Stage II Adult Lymphoblastic Lymphoma; Contiguous Stage II Grade 1 Follicular Lymphoma; Contiguous Stage II Grade 2 Follicular Lymphoma; Contiguous Stage II Grade 3 Follicular Lymphoma; Contiguous Stage II Mantle Cell Lymphoma; Contiguous Stage II Marginal Zone Lymphoma; Contiguous Stage II Small Lymphocytic Lymphoma; Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Nodal Marginal Zone B-cell Lymphoma; Noncontiguous Stage II Adult Burkitt Lymphoma; Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma; Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma; Noncontiguous Stage II Adult Lymphoblastic Lymphoma; Noncontiguous Stage II Grade 1 Follicular Lymphoma; Noncontiguous Stage II Grade 2 Follicular Lymphoma; Noncontiguous Stage II Grade 3 Follicular Lymphoma; Noncontiguous Stage II Mantle Cell Lymphoma; Noncontiguous Stage II Marginal Zone Lymphoma; Noncontiguous Stage II Small Lymphocytic Lymphoma; Peripheral T-cell Lymphoma; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Adult Diffuse Small Cleaved Cell Lymphoma; Recurrent Adult Grade III Lymphomatoid Granulomatosis; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Small Lymphocytic Lymphoma; Splenic Marginal Zone Lymphoma; Stage I Adult Burkitt Lymphoma; Stage I Adult Diffuse Large Cell Lymphoma; Stage I Adult Diffuse Mixed Cell Lymphoma; Stage I Adult Diffuse Small Cleaved Cell Lymphoma; Stage I Adult Immunoblastic Large Cell Lymphoma; Stage I Adult Lymphoblastic Lymphoma; Stage I Grade 1 Follicular Lymphoma; Stage I Grade 2 Follicular Lymphoma; Stage I Grade 3 Follicular Lymphoma; Stage I Mantle Cell Lymphoma; Stage I Marginal Zone Lymphoma; Stage I Small Lymphocytic Lymphoma; Stage III Adult Burkitt Lymphoma; Stage III Adult Diffuse Large Cell Lymphoma; Stage III Adult Diffuse Mixed Cell Lymphoma; Stage III Adult Diffuse Small Cleaved Cell Lymphoma; Stage III Adult Immunoblastic Large Cell Lymphoma; Stage III Adult Lymphoblastic Lymphoma; Stage III Grade 1 Follicular Lymphoma; Stage III Grade 2 Follicular Lymphoma; Stage III Grade 3 Follicular Lymphoma; Stage III Mantle Cell Lymphoma; Stage III Marginal Zone Lymphoma; Stage III Small Lymphocytic Lymphoma; Stage IV Adult Burkitt Lymphoma; Stage IV Adult Diffuse Large Cell Lymphoma; Stage IV Adult Diffuse Mixed Cell Lymphoma; Stage IV Adult Diffuse Small Cleaved Cell Lymphoma; Stage IV Adult Immunoblastic Large Cell Lymphoma; Stage IV Adult Lymphoblastic Lymphoma; Stage IV Grade 1 Follicular Lymphoma; Stage IV Grade 2 Follicular Lymphoma; Stage IV Grade 3 Follicular Lymphoma; Stage IV Mantle Cell Lymphoma; Stage IV Marginal Zone Lymphoma; Stage IV Small Lymphocytic Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific; Waldenström Macroglobulinemia
National Cancer Institute (NCI)
 2009-07 PHASE1
NCT00773955 COMPLETED
Extensive Stage Small Cell Lung Cancer; Recurrent Small Cell Lung Cancer
National Cancer Institute (NCI)
 2008-11 PHASE2
NCT00390403 COMPLETED
Brain and Central Nervous System Tumors
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2007-02 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-01-13)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Gossypol functions as an orally active polyphenol that binds anti-apoptotic Bcl-2 family proteins by blocking BH3 peptide interactions with Bcl-2 and Bcl-XL, while concurrently inhibiting 5α-reductase 1 and 3α-hydroxysteroid dehydrogenase. By suppressing anti-apoptotic signalling, gossypol reactivates intrinsic apoptotic pathways and suppresses cell proliferation, providing the mechanistic basis for its therapeutic investigation in solid tumors and hematologic malignancies such as non-small cell lung cancer and adrenocortical carcinoma.

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.