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Perifosine With Temsirolimus for Recurrent Pediatric Solid Tumors

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ClinicalTrials.gov Identifier: NCT01049841
Recruitment Status : Completed
First Posted : January 15, 2010
Last Update Posted : March 10, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:
The purpose of this study is to test the safety and effectiveness of 2 drugs, perifosine in combination with temsirolimus in children with solid tumors. Neither drug is currently part of the standard treatment of solid tumors in children. Both drugs have been tested alone to treat solid tumors in children with little success. There is now new insight that if given together, perifosine and temsirolimus may work together to stop the growth of solid tumors and may also make them shrink. The doctor wants to find out what effects; good and/or bad, perifosine in combination with temsirolimus has on the patient and the cancer. The doctors are testing four different dose schedules of perifosine with temsirolimus and the patient will be asked to partake in one of the dose schedules. The dose schedule will be lower for those enrolled early in the study.

Condition or disease Intervention/treatment Phase
Pediatric Solid Tumors Drug: perifosine + temsirolimus Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Perifosine With Temsirolimus for Recurrent Pediatric Solid Tumors
Actual Study Start Date : January 2010
Primary Completion Date : March 2017
Study Completion Date : March 2017

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: perifosine + temsirolimus
This is a single arm, phase I study. Eligible patients will receive a loading dose of oral perifosine on the first day, followed by a maintenance dose starting on the second day until progression. Each patient is assigned to a group according to their body surface area (BSA). Temsirolimus will be combined with perifosine at four dose levels to determine the MTD for the combination therapy. Temsirolimus dosing will start on the same day as the perifosine load.
Drug: perifosine + temsirolimus
The patient will take oral tablets of perifosine at a dose and frequency to be determined their height, weight and time when they enter the study as those entering early will be treated with lower dose levels (dosing frequency will vary from once a week to daily). The patient will receive an intravenous injection of temsirolimus once a week at a dose to be determined by their height, weight and time when they enter the study as those entering early will be treated with lower doses. In addition they will be asked to keep a medicine diary.

Outcome Measures

Primary Outcome Measures :
  1. Maximum Tolerated Dose (MTD) of perifosine + temsirolimus combination in children with cancer. [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. To determine whether pharmacokinetic serum levels of both perifosine and temsirolimus correlate with toxicity. [ Time Frame: 2 years ]
  2. To record preliminary data on the efficacy of the perifosine + temsirolimus combination. [ Time Frame: 2 years ]
  3. 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: 2 years ]

Eligibility Criteria

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Any solid tumor that has failed standard therapy
  • Patient must have evidence of tumor by CT, MRI, MIBG scan, serum markers, or tissue sampling.
  • Age ≤ 21 years (age ≤ 35 years for biopsy proven medulloblastoma or neuroblastoma)
  • 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 ≥ 100K at least one week off platelet transfusions
  • Hg≥ 8g/dL at least one week off PRBC transfusion
  • AST ≤ 2 x the upper limit of normal
  • ALT ≤ 2 x the upper limit of normal
  • Total bilirubin ≤ 2.0 mg/dl
  • Patients must have cholesterol level < 350 mg/dl and triglycerides level < 400 mg/dl because temsirolimus can induce hyperlipidemia.
  • 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 serum B-HCG pregnancy test documented within 14 days prior to registration. Females must not be breast feeding.
  • Patients must be able to swallow tablets whole
  • Patients that participated in the phase I single agent perifosine study for recurrent pediatric solid tumors and did not experience a DLT are eligible to participate in this study and can start ≥ 2 weeks since last dose of perifosine
  • Patients that have been previously treated with an mTOR inhibitor can still enroll in this trial as long as they did not experience a DLT in the single agent mTOR inhibitor trial

Exclusion Criteria:

  • Pregnancy
  • Patients must not have an uncontrolled active infection.
  • 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 on EIAEDs that have been discontinued, patients must have been off the agent for at least 2 weeks prior to registration.
  • History of or known pulmonary hypertension or history of or known pneumonitis.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01049841

United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10065
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
University of Wisconsin, Madison
Duke University
AEterna Zentaris
Principal Investigator: Ira Dunkel, MD Memorial Sloan Kettering Cancer Center
More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT01049841     History of Changes
Other Study ID Numbers: 09-124
First Posted: January 15, 2010    Key Record Dates
Last Update Posted: March 10, 2017
Last Verified: March 2017

Keywords provided by Memorial Sloan Kettering Cancer Center:

Additional relevant MeSH terms:
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents