Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors - Full Text View

Sponsor:	Memorial Sloan-Kettering Cancer Center
Collaborators:	University of Wisconsin, Madison Duke University Keryx / AOI Pharmaceuticals, Inc.
Information provided by (Responsible Party):	Memorial Sloan-Kettering Cancer Center
ClinicalTrials.gov Identifier:	NCT00776867

Purpose

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.

Condition	Intervention	Phase
Solid Tumors	Drug: perifosine	Phase I

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Phase I Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

Resource links provided by NLM:

MedlinePlus related topics: Cancer

Drug Information available for: Perifosine

U.S. FDA Resources

Further study details as provided by Memorial Sloan-Kettering Cancer Center:

Primary Outcome Measures:

Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer [ Time Frame: conclusion of the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

To determine whether pharmacokinetic serum levels correlate with toxicity [ Time Frame: conclusion of the study ] [ Designated as safety issue: Yes ]
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 [ Time Frame: conclusion of the study ] [ Designated as safety issue: No ]

Estimated Enrollment:	36
Study Start Date:	October 2008
Estimated Study Completion Date:	October 2012
Estimated Primary Completion Date:	October 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: perifosine 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.	Drug: perifosine 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).

Arms

Assigned Interventions

Experimental: perifosine

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.

Drug: perifosine

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).

Eligibility

Ages Eligible for Study:	up to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Any solid tumor that failed standard therapy.
Patient must have evidence for tumor by CT, MRI, PET scan, MIBG scan, serum markers, or tissue sampling.
Age ≤ 21 years.
Karnofsky/Lansky performance status ≥ 50% (Karnofsky score for age> 16 years and Lansky score for age ≤ 16 years)
ANC≥ 1000 at least 24 hours off GCSF
Platelets ≥ 75k at least one week off platelet transfusions
Hg≥ 8g/dL at least one week off PRBC transfusion
AST ≤ 3 x the upper limit of normal
ALT ≤ 3 x the upper limit of normal
Total bilirubin ≤ 2.0 mg/dl
serum creatinine ≤ 1.5 x the upper limit of normal for age, or calculated creatinine clearance or nuclear GFR ≥ 70 ml/min/1.73 m2.
≥ 3 weeks since last non-nitrourea chemotherapy
≥ 6 weeks since last nitroureas
≥ 4 weeks since last RT
Patients must agree to practice adequate contraception. Females of childbearing potential must have a negative B-HCG pregnancy test documented within 7 days prior to registration. Females must not be breast feeding.
Patients must be able to swallow tablets whole

Exclusion Criteria:

Pregnancy
Patients must not have active infection or serious intercurrent medical illness.
HIV-Positive patients receiving combination anti-retroviral therapy are excluded from the study due to possible retro-viral drug interactions. HIV testing not required.
Patients must not be taking EIAEDs. If patients were previously EIAEDs that have been discontinued, patients must have been off the agent for at least 2 weeks prior to registration.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00776867

Contacts

Contact: Ira Dunkel, MD	212-639-2153
Contact: Tanya Trippett, MD	212-639-8267

Locations

United States, New York
Memorial Sloan-Kettering Cancer Center	Recruiting
New York, New York, United States, 10065
Contact: Ira Dunkel, MD 212-639-2153
Contact: Tanya Trippett, MD 212-639-8267
Principal Investigator: Ira Dunkel, MD

Sponsors and Collaborators

Memorial Sloan-Kettering Cancer Center

University of Wisconsin, Madison

Duke University

Keryx / AOI Pharmaceuticals, Inc.

Investigators

Principal Investigator:

Ira Dunkel, MD

Memorial Sloan-Kettering Cancer Center

More Information

Additional Information:

Memorial Sloan-Kettering Cancer Center This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Memorial Sloan-Kettering Cancer Center
ClinicalTrials.gov Identifier:	NCT00776867 History of Changes
Other Study ID Numbers:	08-091
Study First Received:	October 20, 2008
Last Updated:	January 18, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Memorial Sloan-Kettering Cancer Center:

Pediatric
solid tumors
Pediatric solid tumors
08-091

Additional relevant MeSH terms:

Neoplasms

ClinicalTrials.gov processed this record on February 12, 2012