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OpenTrials
Completed

NCT Number: NCT00776867

Study of Single Agent Perifosine for Recurrent Pediatric Solid Tumors

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.

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Key information

Conditions

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Memorial Sloan-Kettering Cancer Center

New York, 10065, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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).

Primary outcomes

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

    Time frame: conclusion of the study

Secondary outcomes

  1. To determine whether pharmacokinetic serum levels correlate with toxicity

    Time frame: conclusion of the study

  2. 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

Sponsors and collaborators

Lead sponsor

Memorial Sloan Kettering Cancer Center

Other

Collaborators

  • AEterna Zentaris
  • Duke University
  • University of Wisconsin, Madison

Registry information

Official study title

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

Important dates

Study start
2008
Primary completion
2017
Study completion
2017
First posted
Oct 21, 2008
Registry last updated
Aug 1, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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