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A Phase II Study of Perifosine in Soft Tissue Sarcoma

Phase 2
18 Years
Not Enrolling
Recurrent Adult Soft Tissue Sarcoma, Stage III Adult Soft Tissue Sarcoma, Stage IV Adult Soft Tissue Sarcoma

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Trial Information

A Phase II Study of Perifosine in Soft Tissue Sarcoma


I. To evaluate the 6-month progression-free rate in patients treated with perifosine and
having advanced soft tissue sarcoma.


I. To evaluate survival and time to progression. II. To evaluate objective tumor response
status and duration. III. To evaluate adverse event rates. IV. To evaluate patterns of
treatment failure. V. To evaluate pharmacokinetics.

OUTLINE: This is a multicenter study.

Patients receive a loading dose of oral perifosine every 6 hours for a total of 4 doses on
day 1 and once daily on days 2-28 of course 1 only. For all subsequent courses, patients
receive oral perifosine once daily on days 1-28. Courses repeat every 4 weeks in the absence
of disease progression or unacceptable toxicity.

Patients are followed every 3 months for 5 years.

PROJECTED ACCRUAL: A total of 17-46 patients will be accrued for this study within 9-12

Inclusion Criteria:

- Histologically confirmed advanced soft tissue sarcoma

- Measurable disease; measurable disease lesions that are being monitored for response
and have been previously irradiated must have progressed > 25% since completion of
radiation therapy

- Absolute neutrophil count (ANC) >= 1,500/uL

- PLT >= 100,000/uL

- Total bilirubin =< upper normal limit (UNL)

- AST =< 2.5 x UNL

- Creatinine =< UNL or calculated creatinine clearance >= 60 mL/min (i.e. using the
Cockcroft-Gault or Jeliffe methods)

- Life expectancy >= 12 weeks

- ECOG performance status (PS) 0 or 1

- Capable of understanding the investigational nature, potential risks and benefits of
the study and able to provide valid informed consent

Exclusion Criteria:

- Any of the following as this regimen may be harmful to a developing fetus or nursing

- Pregnant women

- Breastfeeding women

- Men or women of childbearing potential or their sexual partners who are
unwilling to employ adequate contraception (condoms, diaphragm, birth control
pills, injections, intrauterine device [IUD], surgical sterilization,
subcutaneous implants, or abstinence, etc.)

- NOTE: Pregnant women are excluded from this study because perifosine is an
alkylphospholipid with the potential for teratogenic or abortifacient effects;
because there is an unknown but potential risk for adverse events in nursing
infants secondary to treatment of the mother with perifosine, breastfeeding
should be discontinued if the mother is treated with perifosine

- Any of the following:

- >= 3 prior cytotoxic chemotherapy regimens for metastatic sarcoma

- Chemotherapy =< 4 weeks prior to study entry

- Nitrosoureas or mitomycin C =< 6 weeks prior to study entry

- Radiotherapy =< 4 weeks prior to study entry

- Immunotherapy =< 4 weeks prior to study entry

- Biologic therapy =< 4 weeks prior to study entry

- Failure to recover from acute, reversible effects of prior therapy regardless of
interval since last treatment

- Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy
considered investigational (utilized for a non-FDA-approved indication and in the
context of a research investigation)

- Uncontrolled brain metastases; NOTE: these patients are excluded because of the poor
prognosis and because they often develop progressive neurologic dysfunction that
would confound the evaluation of neurologic and other adverse events; however, if
brain metastasis are treated and controlled for > 8 weeks, the patient would be
eligible for this study

- History of allergic reactions attributed to compounds of similar chemical or biologic
composition to perifosine

- Uncontrolled intercurrent illness including, but not limited to ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris,
symptomatic cardiac arrhythmia despite appropriate therapy, or psychiatric
illness/social situations that would limit compliance with study requirements

- HIV-positive patients receiving combination anti-retroviral therapy; NOTE: these
patients are excluded from the study because of possible pharmacokinetic interactions
with perifosine; appropriate studies may be undertaken in patients receiving
combination anti-retroviral therapy when indicated

- Prior malignancy, except for the following:

- Adequately treated basal cell or squamous cell skin cancer

- Adequately treated noninvasive carcinoma

- Other invasive cancer from which the patient has been disease-free for 5 years

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Progression-free rate

Outcome Time Frame:

6 months

Safety Issue:


Principal Investigator

Howard Bailey

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic


United States: Food and Drug Administration

Study ID:




Start Date:

June 2003

Completion Date:

Related Keywords:

  • Recurrent Adult Soft Tissue Sarcoma
  • Stage III Adult Soft Tissue Sarcoma
  • Stage IV Adult Soft Tissue Sarcoma
  • Sarcoma



Mayo Clinic Rochester, Minnesota  55905