Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

Phase 1

Completed

• Conditions: Solid Tumors
• Interventions: Drug: perifosine

• Registration Number: NCT00776867
• Lead Sponsor: Memorial Sloan Kettering Cancer Center

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-10
• Study First Submitted: 2008-10-20
• Study First Submitted QC: 2008-10-20
• Study First Posted: 2008-10-21
• Status Verified: 2017-07
• Primary Completion: 2017-07
• Study Completion: 2017-07
• Last Update Submitted: 2017-07-31
• Last Update Posted: 2017-08-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 38

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
perifosine	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.

Primary Outcome Measures

Name	Time	Method
Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer	conclusion of the study

Secondary Outcome Measures

Name	Time	Method
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	conclusion of the study

Trial Locations

Locations (1)

Memorial Sloan-Kettering Cancer Center; 🇺🇸 New York, New York, United States