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Phase II Study of Mifepristone (RU-486) in the Treatment of PR Positive Advanced/Recurrent Endometrioid Adenocarcinoma and Low Grade Endometrial Stromal Sarcoma (LGESS)

Phase 2
Not Enrolling
Endometrial Cancer

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Trial Information

Phase II Study of Mifepristone (RU-486) in the Treatment of PR Positive Advanced/Recurrent Endometrioid Adenocarcinoma and Low Grade Endometrial Stromal Sarcoma (LGESS)

Mifepristone is a drug that has been approved for use in the termination of pregnancy. It
has been studied in women with breast and ovarian cancer.

Before treatment starts, patients will have a complete checkup, blood tests, a CT scan, and
a chest x-ray. Women able to have children must have a negative urine pregnancy test. Blood
tests and a complete checkup will also be done within 3 days of starting each course of
therapy and a month after treatment ends. Tumors will be measured by CT scans once every 2-3
months and at the end of treatment.

Patients in this study will take Mifepristone in the form of a pill by mouth every day. Each
course of therapy is 4 weeks long. Patients will see their doctor for an exam and blood
tests before they begin each 4 week course of therapy. The dose of Mifepristone may be
lowered if the patient has side effects. Patients who have a complete response (there is no
evidence of cancer by physical exam or x-ray) will continue taking Mifepristone for 2 years
after this response is documented. Other patients will take Mifepristone for as long as it
benefits them.

This is an investigational study. At least 12 and as many as 37 patients will take part in
this study. Patients from MD Anderson Cancer Center and Gynecologic Oncology of Houston,
P.A. will be enrolled.

Inclusion Criteria:

1. Histologic diagnosis of advanced/recurrent endometrioid adenocarcinoma or low grade
endometrial stromal sarcoma (LGESS) not amenable to curative surgery or radiotherapy.

2. Patients should have previously undergone radical surgery (minimum of total abdominal
hysterectomy and bilateral salpingoophorectomy) definitive radiation therapy, or not
be candidates for such procedures.

3. The primary (including archived specimens) or recurrent tumor must be PR positive.
Hormone receptor positivity is defined as PR positivity in >/+10% cells by

4. Prior radiotherapy must have been completed at least 2 weeks prior to the initiation
of Mifepristone and patients must have recovered from the acute side effects of such

5. Performance status < Zubrod 2.

6. Estimated life expectancy of at least 12 weeks.

7. Prior chemotherapy for recurrent or metastatic endometrial cancer is permitted.

8. Patients must have measurable disease as defined by the presence of bidimensionally
measurable lesions with clearly defined margins on x-ray, scan (CT or MRI) or
physical exam.

9. Adequate bone marrow reserve: granulocyte count > 1.5 x 109/L, hemoglobin > 9 g/dL
(transfusion-independent) and platelets > 100,000 K/UL.

10. Adequate liver and renal function as defined as: total bilirubin value < 1.5 mg/dL;
SGPT < 2x the upper limit of normal or < 5x the upper limit of normal when liver
metastases are present; serum creatinine value of < 1.8 mg/dL. All qualifying
laboratory parameters must be determined within one week of first treatment.

11. Patient compliance and geographic proximity that allows adequate follow-up.

12. Signed informed consent.

Exclusion Criteria:

1. Patients with serous or clear cell carcinomas of the endometrium.

2. Patients whose tumor is known to be PR negative.

3. Uncontrolled hypercalcemia.

4. Patients taking phenytoin, phenobarbital or carbamazepine.

5. Known predisposition to thromboembolic disorder, which in the investigator's judgment
would put the patient at unacceptable risk for thromboembolic complications.

6. Patients who have received treatment for brain metastases may be enrolled provided
they have remained stable for at least 6 months after surgery or radiation.

7. Women taking estrogen, progestin or antiprogestins. Patients taking these drugs must
have discontinued their use at least 3 weeks prior to beginning treatment with

8. History of other malignancy (except adequately treated non-melanomatous carcinoma of
the skin or cervical carcinoma in situ) unless in complete remission and off all
therapy for that disease for a minimum of 5 years.

9. Use of any chemotherapy or investigational agent within 3 weeks prior to taking study

10. Concurrent serious infection.

11. Patients with serious intercurrent medical illness.

12. Serious concomitant systemic disorders incompatible with the study (at the discretion
of the investigator).

13. Patients whom absorption of drugs is likely to be impaired due to either concomitant
medications or prior surgery.

14. Overt psychosis or mental disability or otherwise incompetent to give informed

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Patients with Overall Response

Outcome Description:

Overall Response = Complete and Partial Responses

Outcome Time Frame:

With each 4 week cycle, follow up 2 years

Safety Issue:


Principal Investigator

Lois M. Ramondetta, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

September 2001

Completion Date:

May 2008

Related Keywords:

  • Endometrial Cancer
  • Endometrial Cancer
  • Low Grade Endometrial Stromal Sarcoma
  • RU-486
  • Mifepristone
  • Endometrial Neoplasms
  • Sarcoma, Endometrial Stromal
  • Carcinoma, Endometrioid
  • Adenoma
  • Endometrial Stromal Tumors



U.T.M.D. Anderson Cancer CenterHouston, Texas  77030