Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

Solid Tumors Clinical Trial

Official title:

Phase I Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00776867
• Other study ID #: 08-091
• Status: Completed
• Phase: Phase 1
• First received: October 20, 2008
• Last updated: July 31, 2017
• Start date: October 2008
• Est. completion date: July 2017

Study information

• Verified date: July 2017
• Source: Memorial Sloan Kettering Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: July 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

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.

Related Conditions & MeSH terms

• Neoplasms
• Solid Tumors

Intervention

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).

Locations

Country	Name	City	State
United States	Memorial Sloan-Kettering Cancer Center	New York	New York

Sponsors (4)

Lead Sponsor	Collaborator
Memorial Sloan Kettering Cancer Center	AEterna Zentaris, Duke University, University of Wisconsin, Madison

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer		conclusion of the study
Secondary	To determine whether pharmacokinetic serum levels correlate with toxicity		conclusion of the study
Secondary	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

External Links

•   Memorial Sloan-Kettering Cancer Center