Overview

This trial is active, not recruiting.

Condition solid tumors
Treatment perifosine
Phase phase 1
Target JNK1
Sponsor Memorial Sloan Kettering Cancer Center
Collaborator University of Wisconsin, Madison
Start date October 2008
End date October 2016
Trial size 36 participants
Trial identifier NCT00776867, 08-091

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(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.
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).

Primary Outcomes

Measure
Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer
time frame: conclusion of the study

Secondary Outcomes

Measure
To determine whether pharmacokinetic serum levels correlate with toxicity
time frame: 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
time frame: conclusion of the study

Eligibility Criteria

Male or female participants up to 21 years old.

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-nitrosourea chemotherapy - ≥ 6 weeks since last nitrosoureas - ≥ 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 14 days prior to drug initiation. 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.

Additional Information

Official title Phase I Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors
Principal investigator Ira Dunkel, MD
Trial information was received from ClinicalTrials.gov and was last updated in September 2015.
Information provided to ClinicalTrials.gov by Memorial Sloan Kettering Cancer Center.