Clinical Trials

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)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Perifosine targets the pleckstrin homology domain of Akt to block its membrane translocation and activation, which subsequently modulates membrane lipid composition and shifts the balance toward pro-apoptotic SAPK/JNK signaling over anti-apoptotic MAPK pathways. This biochemical disruption induces cell cycle arrest and apoptosis, thereby suppressing cellular proliferation in malignancies such as colon cancer, malignant gliomas, and advanced solid 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>).

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