Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

NCT00776867 | Completed Phase 1

• 1 other identifier: 08-091
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

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.

Conditions

Solid Tumors

Keywords

Pediatric; solid tumors; Pediatric solid tumors; 08-091

Outcome Measures

Primary Outcomes (1)

• Maximum Tolerated Dose (MTD) of perifosine monotherapy in children with cancer

  conclusion of the study

Secondary Outcomes (2)

• 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

Study Arms (1)

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.

Drug: perifosine

Interventions

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).

perifosine

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• University of Wisconsin, Madison: collaborator
• Duke University: collaborator
• AEterna Zentaris: collaborator

Study Sites (1)

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Publications (2)

• 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.

  PMID: 27649927 DERIVED

• 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.

  PMID: 22419878 DERIVED

Related Links

• Memorial Sloan-Kettering Cancer Center

MeSH Terms

Interventions

perifosine

Study Officials

• Ira Dunkel, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2008
• First Posted: October 21, 2008
• Study Start: October 1, 2008
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2017
• Last Updated: August 1, 2017

Record last verified: 2017-07

Locations

US (1)