Numerous Phase 1, Phase 2, and Phase 3 clinical trials have evaluated perifosine as monotherapy and combination therapy for conditions including solid tumors, colon cancer, pediatric malignancies, soft tissue sarcomas, malignant gliomas, and Waldenström's macroglobulinemia. Sponsored by academic centers, cooperative networks, and industry entities such as the National Cancer Institute, Memorial Sloan Kettering Cancer Center, and AEterna Zentaris, these mostly completed or terminated studies represent extensive efforts to assess perifosine as a targeted therapy for refractory or advanced neoplasms.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT00590954 | COMPLETED | Malignant Gliomas; CNS; Brain Cancer; Cancer |
Memorial Sloan Kettering Cancer Center |
2006-05 | PHASE2 |
| NCT01051557 | COMPLETED | Adult Anaplastic Astrocytoma; Adult Anaplastic Oligodendroglioma; Adult Diffuse Astrocytoma; Adult Giant Cell Glioblastoma; Adult Glioblastoma; Adult Gliosarcoma; Adult Mixed Glioma; Adult Oligodendroglioma; Recurrent Adult Brain Neoplasm |
National Cancer Institute (NCI) |
2010-01-27 | PHASE1; PHASE2 |
| NCT02238496 | COMPLETED | Brain Tumor, Recurrent; Glioblastoma; Anaplastic Astrocytoma; Anaplastic Oligodendroglioma; Mixed Glioma |
Andrew B Lassman, MD |
2014-12-08 | PHASE1 |
| NCT00776867 | COMPLETED | Solid Tumors |
Memorial Sloan Kettering Cancer Center |
2008-10 | PHASE1 |
| NCT01049841 | COMPLETED | Pediatric Solid Tumors |
Memorial Sloan Kettering Cancer Center |
2010-01 | PHASE1 |
| NCT01224730 | COMPLETED | Cancer |
AEterna Zentaris |
2012-01-24 | PHASE1 |
| NCT01002248 | TERMINATED | Multiple Myeloma |
AEterna Zentaris |
2009-12 | PHASE3 |
| NCT00847366 | COMPLETED | Non Small Cell Lung Cancer; Solid Tumors; Metastatic Breast Cancer; Sarcomas |
AEterna Zentaris |
2008-05 | |
| NCT00415064 | COMPLETED | Multiple Myeloma |
AEterna Zentaris |
2006-12 | PHASE1 |
| NCT00431054 | COMPLETED | Ovarian Cancer |
M.D. Anderson Cancer Center |
2007-02 | PHASE1 |
| NCT00301938 | COMPLETED | Accelerated Phase Chronic Myelogenous Leukemia; Adult Acute Megakaryoblastic Leukemia (M7); Adult Acute Minimally Differentiated Myeloid Leukemia (M0); Adult Acute Monoblastic Leukemia (M5a); Adult Acute Monocytic Leukemia (M5b); Adult Acute Myeloblastic Leukemia With Maturation (M2); Adult Acute Myeloblastic Leukemia Without Maturation (M1); Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; Adult Acute Myeloid Leukemia With Inv(16)(p13;q22); Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Adult Acute Myelomonocytic Leukemia (M4); Adult Acute Promyelocytic Leukemia (M3); Adult Erythroleukemia (M6a); Adult Pure Erythroid Leukemia (M6b); Blastic Phase Chronic Myelogenous Leukemia; Myelodysplastic/Myeloproliferative Neoplasms; Previously Treated Myelodysplastic Syndromes; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Acute Myeloid Leukemia; Relapsing Chronic Myelogenous Leukemia; Secondary Acute Myeloid Leukemia; T-cell Adult Acute Lymphoblastic Leukemia; Untreated Adult Acute Lymphoblastic Leukemia; Untreated Adult Acute Myeloid Leukemia |
National Cancer Institute (NCI) |
2005-12 | PHASE1 |
| NCT01097018 | COMPLETED | Colorectal Cancer |
AEterna Zentaris |
2010-04 | PHASE3 |
| NCT01224730 | Completed | Cancer |
AEterna Zentaris |
2012-01-24 | Phase 1 |
| NCT00873457 | COMPLETED | Chronic Lymphocytic Leukemia; Small Lymphocytic Lymphoma |
Daphne Friedman |
2009-08 | PHASE2 |
| NCT00398814 | COMPLETED | Renal Cancer; Tumors |
AEterna Zentaris |
2006-10 | PHASE1 |
| NCT00398710 | COMPLETED | Waldenström's Macroglobulinemia |
AEterna Zentaris |
2006-10 | PHASE2 |
| NCT00389077 | COMPLETED | Tumors; Lymphomas |
AEterna Zentaris |
2005-01 | PHASE2 |
| NCT00498966 | COMPLETED | Kidney Cancer |
AEterna Zentaris |
2007-07 | PHASE2 |
| NCT01048580 | COMPLETED | Colon Cancer |
AEterna Zentaris |
2009-10 | PHASE1 |
| NCT00401388 | COMPLETED | Chondrosarcomas; Alveolar Soft Part Sarcomas; Extra Skeletal Myxoid Chondrosarcomas |
AEterna Zentaris |
2006-11 | PHASE2 |
| NCT00398879 | COMPLETED | Colon Cancer |
AEterna Zentaris |
2005-08 | PHASE2 |
| NCT00399789 | COMPLETED | Non Small Cell Lung Cancer |
AEterna Zentaris |
2004-09 | PHASE1; PHASE2 |
| NCT00375791 | COMPLETED | Multiple Myeloma |
AEterna Zentaris |
2005-12 | PHASE2 |
| NCT00401011 | COMPLETED | Multiple Myeloma; Multiple Myeloma in Relapse |
AEterna Zentaris |
2006-08 | PHASE1; PHASE2 |
| NCT00455559 | COMPLETED | Gastrointestinal Stromal Tumors |
AEterna Zentaris |
2006-08 | PHASE2 |
| NCT00391560 | COMPLETED | Leukemia |
AEterna Zentaris |
2006-10 | PHASE2 |
| NCT00448721 | COMPLETED | Renal Cell Carcinoma |
AEterna Zentaris |
2007-03 | PHASE2 |
| NCT00399087 | COMPLETED | Neoplasms |
AEterna Zentaris |
2004-11 | PHASE1 |
| NCT01049841 | Completed | Pediatric Solid Tumors |
Memorial Sloan Kettering Cancer Center|University of Wisconsin Madison|Duke University|NATL COMP CA NETWORK|Pfizer|AEterna Zentaris |
2010-01 | Phase 1 |
| NCT01048580 | Completed | Colon Cancer |
AEterna Zentaris|SCRI Development Innovations LLC |
2009-10 | Phase 1 |
| NCT00422656 | COMPLETED | Waldenstrom's Macroglobulinemia |
Dana-Farber Cancer Institute |
2006-09 | PHASE2 |
| NCT00399126 | COMPLETED | Neoplasms |
AEterna Zentaris |
2004-11 | PHASE1 |
| NCT00058214 | TERMINATED | Adenocarcinoma of the Prostate; Recurrent Prostate Cancer; Stage IIB Prostate Cancer; Stage III Prostate Cancer; Stage IV Prostate Cancer |
National Cancer Institute (NCI) |
2003-03 | PHASE2 |
| NCT00399152 | COMPLETED | Renal Cancer; GIST |
AEterna Zentaris |
2006-10 | PHASE1 |
| NCT00776867 | Completed | Solid Tumors |
Memorial Sloan Kettering Cancer Center|University of Wisconsin Madison|Duke University|AEterna Zentaris |
2008-10 | Phase 1 |
| NCT00054145 | COMPLETED | Breast Cancer |
University Health Network, Toronto |
2003-06 | PHASE2 |
| NCT00062387 | TERMINATED | Head and Neck Cancer |
National Cancer Institute (NCI) |
2003-05 | PHASE2 |
| NCT00053924 | COMPLETED | Pancreatic Cancer |
University Health Network, Toronto |
2003-05 | PHASE2 |
| NCT00064324 | COMPLETED | Recurrent Adult Soft Tissue Sarcoma; Stage III Adult Soft Tissue Sarcoma; Stage IV Adult Soft Tissue Sarcoma |
National Cancer Institute (NCI) |
2003-06 | PHASE2 |
| NCT00053794 | COMPLETED | Endometrial Cancer; Sarcoma |
NCIC Clinical Trials Group |
2003-06-09 | PHASE2 |
| NCT00053781 | COMPLETED | Melanoma (Skin) |
NCIC Clinical Trials Group |
2003-06-20 | PHASE2 |
| NCT00398697 | COMPLETED | Neoplasms |
AEterna Zentaris |
2004-08 | PHASE1 |
| NCT00060437 | COMPLETED | Prostate Cancer |
National Cancer Institute (NCI) |
2003-10 | PHASE2 |
| NCT00019656 | COMPLETED | Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms; Unspecified Adult Solid Tumor, Protocol Specific |
National Cancer Institute (NCI) |
1999-08 | PHASE1 |
| NCT00005794 | COMPLETED | Unspecified Adult Solid Tumor, Protocol Specific |
University of Wisconsin, Madison |
2000-02 | PHASE1 |
| NCT00059982 | COMPLETED | Pancreatic Cancer |
Eastern Cooperative Oncology Group |
2003-07 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2020-10-19)
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>).