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| Name | Class |
|---|---|
| University of Wisconsin, Madison | OTHER |
| Duke University | OTHER |
| AEterna Zentaris | INDUSTRY |
The purpose of this study is to test whether perifosine- a drug that inhibits the protein AKT, and has had some success in the treatment of adult cancers- is safe and effective in treating cancer. The investigators want to find out what effects, good and/or bad, it has on the patient and the cancer. The investigators are testing different dose schedules of perifosine and the patient will be asked to partake in one of the dose schedules.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| perifosine | Experimental | This will be a dose escalation study to determine the maximum tolerated dose (MTD) of perifosine alone in recurrent/progressive pediatric tumors. A standard 3+3 dose escalation design will be employed with 3-6 patients at each dose level. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| perifosine | Drug | Open label, phase I study of oral perifosine in recurrent/progressive pediatric solid tumors. This will be a dose escalation study to determine the maximum tolerated dose (MTD) of perifosine alone in recurrent/progressive pediatric tumors. A standard 3+3 dose escalation design will be employed with 3-6 patients at each dose level. All patients will receive perifosine at a loading dose on the first day, followed by a maintenance dose to start on day two until progression each patient will be assigned to a dosing group based on one's body surface area (BSA). |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer | conclusion of the study |
| Measure | Description | Time Frame |
|---|---|---|
| To determine whether pharmacokinetic serum levels correlate with toxicity | conclusion of the study | |
| If previously resected tissue is available, determine whether molecular features predict response including Elevated PI3K/AKT/mTOR signaling, Elevated RAS/MAPK signaling, Cell cycle markers |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ira Dunkel, MD | Memorial Sloan Kettering Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Memorial Sloan-Kettering Cancer Center | New York | New York | 10065 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27649927 | Derived | Kushner BH, Cheung NV, Modak S, Becher OJ, Basu EM, Roberts SS, Kramer K, Dunkel IJ. A phase I/Ib trial targeting the Pi3k/Akt pathway using perifosine: Long-term progression-free survival of patients with resistant neuroblastoma. Int J Cancer. 2017 Jan 15;140(2):480-484. doi: 10.1002/ijc.30440. Epub 2016 Sep 30. | |
| 22419878 | Derived |
| Label | URL |
|---|---|
| Memorial Sloan-Kettering Cancer Center | View source |
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| ID | Term |
|---|---|
| C105905 | perifosine |
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| conclusion of the study |
| Sun W, Modak S. Emerging treatment options for the treatment of neuroblastoma: potential role of perifosine. Onco Targets Ther. 2012;5:21-9. doi: 10.2147/OTT.S14578. Epub 2012 Mar 2. |