Numerous clinical trials across early to late phases evaluate O6-benzylguanine primarily as a chemosensitizing agent in combination regimens for malignancies including glioblastoma multiforme, brain and central nervous system tumors, advanced solid tumors, colorectal cancer, and multiple myeloma. Spearheaded by major sponsors such as the National Cancer Institute, Duke University, and the Case Comprehensive Cancer Center, these investigations encompass completed and terminated historical protocols alongside active research. Currently, several clinical trials are recruiting to evaluate O6-benzylguanine in combination with temozolomide for patients with recurrent glioblastoma.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT05052957 | RECRUITING | Glioblastoma Multiforme; Glioblastoma Multiforme, Adult; Supratentorial Glioblastoma; Supratentorial Gliosarcoma |
Leland Metheny |
2023-01-20 | PHASE2 |
| NCT01269424 | COMPLETED | Glioblastoma Multiforme |
Stanton Gerson MD |
2011-11-22 | PHASE1 |
| NCT02343666 | WITHDRAWN | Human Immunodeficiency Virus 1 Positive; Stage I Adult Hodgkin Lymphoma; Stage I Adult Non-Hodgkin Lymphoma; Stage II Adult Hodgkin Lymphoma; Stage II Adult Non-Hodgkin Lymphoma; Stage III Adult Hodgkin Lymphoma; Stage III Adult Non-Hodgkin Lymphoma; Stage IV Adult Hodgkin Lymphoma; Stage IV Adult Non-Hodgkin Lymphoma |
Fred Hutchinson Cancer Center |
2016-08-15 | PHASE1 |
| NCT01769911 | WITHDRAWN | Adult Nasal Type Extranodal NK/T-cell Lymphoma; AIDS-related Diffuse Large Cell Lymphoma; AIDS-related Diffuse Mixed Cell Lymphoma; AIDS-related Diffuse Small Cleaved Cell Lymphoma; AIDS-related Immunoblastic Large Cell Lymphoma; AIDS-related Lymphoblastic Lymphoma; AIDS-related Peripheral/Systemic Lymphoma; AIDS-related Small Noncleaved Cell Lymphoma; Anaplastic Large Cell Lymphoma; Angioimmunoblastic T-cell Lymphoma; Cutaneous B-cell Non-Hodgkin Lymphoma; Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Hepatosplenic T-cell Lymphoma; HIV-associated Hodgkin Lymphoma; Intraocular Lymphoma; Nodal Marginal Zone B-cell Lymphoma; Noncutaneous Extranodal 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 Hodgkin Lymphoma; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Adult T-cell Leukemia/Lymphoma; Recurrent Cutaneous T-cell Non-Hodgkin 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 Mycosis Fungoides/Sezary Syndrome; Recurrent Small Lymphocytic Lymphoma; Refractory Hairy Cell Leukemia; Small Intestine Lymphoma; Splenic Marginal Zone Lymphoma; Stage I AIDS-related Lymphoma; Stage II AIDS-related Lymphoma; Stage III AIDS-related Lymphoma; Stage IV AIDS-related Lymphoma; T-cell Large Granular Lymphocyte Leukemia; Testicular Lymphoma; Waldenström Macroglobulinemia |
Fred Hutchinson Cancer Center |
2015-02 | |
| NCT00669669 | TERMINATED | Glioblastoma; Gliosarcoma |
Fred Hutchinson Cancer Center |
2009-02-25 | PHASE1; PHASE2 |
| NCT00253487 | COMPLETED | Brain and Central Nervous System Tumors |
Children's Hospital Medical Center, Cincinnati |
2005-08 | |
| NCT00961220 | COMPLETED | Recurrent Primary Cutaneous T-Cell Non-Hodgkin Lymphoma; Stage I Mycosis Fungoides and Sezary Syndrome AJCC v7; Stage II Mycosis Fungoides and Sezary Syndrome AJCC v7 |
National Cancer Institute (NCI) |
2010-02-01 | PHASE1; PHASE2 |
| NCT00275002 | COMPLETED | Brain and Central Nervous System Tumors |
National Cancer Institute (NCI) |
2006-02 | PHASE2 |
| NCT00436436 | TERMINATED | Brain and Central Nervous System Tumors |
National Cancer Institute (NCI) |
2006-11-13 | PHASE2 |
| NCT00389090 | TERMINATED | Glioma; Astrocytoma; Oligodendroglioma; Oligoastrocytoma |
Keryx / AOI Pharmaceuticals, Inc. |
2006-10 | PHASE2 |
| NCT00052780 | COMPLETED | Childhood Central Nervous System Germ Cell Tumor; Childhood Choroid Plexus Tumor; Childhood Craniopharyngioma; Childhood Ependymoblastoma; Childhood Grade I Meningioma; Childhood Grade II Meningioma; Childhood Grade III Meningioma; Childhood High-grade Cerebellar Astrocytoma; Childhood High-grade Cerebral Astrocytoma; Childhood Infratentorial Ependymoma; Childhood Low-grade Cerebellar Astrocytoma; Childhood Low-grade Cerebral Astrocytoma; Childhood Medulloepithelioma; Childhood Mixed Glioma; Childhood Oligodendroglioma; Childhood Supratentorial Ependymoma; Recurrent Childhood Brain Stem Glioma; Recurrent Childhood Cerebellar Astrocytoma; Recurrent Childhood Cerebral Astrocytoma; Recurrent Childhood Ependymoma; Recurrent Childhood Medulloblastoma; Recurrent Childhood Pineoblastoma; Recurrent Childhood Subependymal Giant Cell Astrocytoma; Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor; Recurrent Childhood Visual Pathway and Hypothalamic Glioma |
National Cancer Institute (NCI) |
2002-10 | PHASE1 |
| NCT00612989 | COMPLETED | Glioblastoma; Gliosarcoma |
Duke University |
2005-02 | PHASE1 |
| NCT00362921 | COMPLETED | Recurrent Adult Brain Tumor |
Duke University |
2004-04 | PHASE2 |
| NCT00003567 | TERMINATED | Brain and Central Nervous System Tumors; Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific |
Case Comprehensive Cancer Center |
1999-05 | PHASE1 |
| NCT00612638 | COMPLETED | Glioblastoma; Gliosarcoma |
Duke University |
2005-01 | PHASE1 |
| NCT00003613 | TERMINATED | Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma; Stage I Cutaneous T-cell Non-Hodgkin Lymphoma; Stage II Cutaneous T-cell Non-Hodgkin Lymphoma |
National Cancer Institute (NCI) |
1999-04 | PHASE1 |
| NCT00017147 | COMPLETED | Malignant Neoplasms of Eye, Brain and Other Parts of Central Nervous System |
SWOG Cancer Research Network |
2001-09 | PHASE3 |
| NCT00613093 | COMPLETED | Glioblastoma Multiforme; Anaplastic Glioma |
Duke University |
2002-10 | PHASE2 |
| NCT00003765 | COMPLETED | Brain and Central Nervous System Tumors |
National Cancer Institute (NCI) |
1999-05 | PHASE1 |
| NCT00275002 | Completed | Brain and Central Nervous System Tumors |
National Cancer Institute (NCI) |
2006-02 | Phase 2 |
| NCT00005961 | COMPLETED | Melanoma (Skin) |
National Cancer Institute (NCI) |
2000-06 | PHASE2 |
| NCT00086970 | TERMINATED | Unspecified Adult Solid Tumor, Protocol Specific |
National Cancer Institute (NCI) |
2004-06 | PHASE1 |
| NCT00006474 | COMPLETED | Brain and Central Nervous System Tumors |
Duke University |
2001-03 | PHASE1 |
| NCT00004072 | COMPLETED | Multiple Myeloma and Plasma Cell Neoplasm |
Case Comprehensive Cancer Center |
1999-09 | PHASE2 |
| NCT00045721 | TERMINATED | Brain and Central Nervous System Tumors |
Pediatric Brain Tumor Consortium |
2003-03 | PHASE1 |
| NCT00086970 | Terminated | Unspecified Adult Solid Tumor Protocol Specific |
National Cancer Institute (NCI) |
2004-06 | Phase 1 |
| NCT00005981 | COMPLETED | Colorectal Cancer |
Case Comprehensive Cancer Center |
2000-06 | PHASE2 |
| NCT00002971 | COMPLETED | Brain and Central Nervous System Tumors |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
1997-06-19 | PHASE1 |
| NCT00613093 | Completed | Glioblastoma Multiforme|Anaplastic Glioma |
Duke University|Keryx / AOI Pharmaceuticals Inc.|National Institutes of Health (NIH) |
2002-10 | Phase 2 |
| NCT00003766 | COMPLETED | Unspecified Adult Solid Tumor, Protocol Specific |
University of Chicago |
1999-10 | PHASE1 |
| NCT00005066 | COMPLETED | Sarcoma |
University of Chicago |
2000-06 | PHASE2 |
| NCT00005081 | COMPLETED | Brain and Central Nervous System Tumors |
Duke University |
2000-08 | PHASE2 |
| NCT00003348 | COMPLETED | Brain and Central Nervous System Tumors |
Duke University |
1998-05 | PHASE1 |
| NCT00002604 | COMPLETED | Unspecified Adult Solid Tumor, Protocol Specific |
Case Comprehensive Cancer Center |
1996-01 | PHASE1 |
| NCT00005981 | Completed | Colorectal Cancer |
Case Comprehensive Cancer Center|National Cancer Institute (NCI) |
2000-06 | Phase 2 |
| NCT00004072 | Completed | Multiple Myeloma and Plasma Cell Neoplasm |
Case Comprehensive Cancer Center|National Cancer Institute (NCI) |
1999-09 | Phase 2 |
| NCT00046878 | WITHDRAWN | Brain and Central Nervous System Tumors |
Duke University |
PHASE2 | |
| NCT00020150 | COMPLETED | Brain and Central Nervous System Tumors; Childhood Germ Cell Tumor; Extragonadal Germ Cell Tumor; Kidney Cancer; Liver Cancer; Neuroblastoma; Ovarian Cancer; Sarcoma; Unspecified Childhood Solid Tumor, Protocol Specific |
National Cancer Institute (NCI) |
2000-06 | PHASE1 |
| NCT00004892 | COMPLETED | Brain and Central Nervous System Tumors |
National Cancer Institute (NCI) |
2000-04 | PHASE1 |
(data from https://clinicaltrials.gov, updated on 2025-11-21)
Check the
O6-Benzylguanine
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
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>).
Note: Technical data last updated: Sep 1, 2026.