Cancer
Conditions
Keywords
Mifepristone, RU-486, Compassionate Use, Antiprogesterone
Brief summary
The purpose of this study is to see if mifepristone prevents worsening of your cancer. Mifepristone is an antiprogesterone agent, a drug which blocks female hormones, that is commonly used for the termination of pregnancies. It has not been approved by the Food and Drug Administration for use in the treatment of cancer. It is unlicensed in the United States for your condition. However, previous work has indicated that mifepristone may be useful due to how it works. It is being made available for use in the United States for compassionate use through the Feminist Majority Foundation.
Detailed description
This is a compassionate use of mifepristone treatment for patients with conditions that could respond to an antiprogesterone agent, including: * Meningioma. * Breast cancer * Colon Cancer * Endometrial Stromal Sarcoma * Bilateral Chronic Central Serous Retinopathy * Cushing's Syndrome * Metastatic Adrenocortical Cancer * Ovarian Cancer * Other conditions as determined by the attending physicians
Interventions
DRUGMifepristone
Mifepristone 200 mg will be administered orally
Sponsors
The Feminist Majority Foundation
New Mexico Cancer Research Alliance
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* All patients 18 years of age or older. * Patients must sign an informed consent. * Patients should be in such a health condition in the opinion of the attending physician that with the administration of mifepristone benefits may outweigh risks.
Exclusion criteria
* Pregnant women or nursing mothers are not eligible for this trial. Patients of child bearing potential must use adequate contraception.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Duration of Response | 5 years | The time from the date of response (not the beginning of treatment unless there is stable disease) to disease progression. Response and progression are evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) (version 1.0). Target lesions are assessed by computerized tomography (CT) or magnetic resonance imaging (MRI): Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), neither sufficient decrease in the sum of the longest diameter of target lesions to qualify for PR nor sufficient increase in the sum of the longest diameter of target lesions to qualify for Progressive Disease; Progressive Disease (PD), 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Toxicity Associated With Adrenal Insufficiency | Up to 8 weeks after the end of study treatment or until any adverse events are resolved (whichever is longest) | Toxicity will be evaluated per National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 3.0. Frequency and severity of adverse events will be tabulated using counts the following events of interest, which are related to possible adrenal insufficiency: nausea, vomiting, lethargy, dizziness, fatigue, anorexia, and skin rash. Any grade of these events that are self-reported by patients as well as events identified by physician assessment (e.g. physical exam) will be included. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival | 5 years | The time from patient entry into the protocol to death by any cause. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Mifepristone 200 mg RU-486 (Mifepristone) daily
Mifepristone: Mifepristone 200 mg will be administered orally | 4 |
| Total | 4 |
Baseline characteristics
| Characteristic | Mifepristone |
|---|---|
| Age, Continuous | 45 years |
| Region of Enrollment United States | 4 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 2 / 4 |
| serious Total, serious adverse events | 0 / 4 |
Outcome results
Primary
Duration of Response
The time from the date of response (not the beginning of treatment unless there is stable disease) to disease progression. Response and progression are evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) (version 1.0). Target lesions are assessed by computerized tomography (CT) or magnetic resonance imaging (MRI): Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), neither sufficient decrease in the sum of the longest diameter of target lesions to qualify for PR nor sufficient increase in the sum of the longest diameter of target lesions to qualify for Progressive Disease; Progressive Disease (PD), 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Time frame: 5 years
Population: Only one patient achieved stable disease. This patient's duration of response could therefore be reported. The other three patients progressed on treatment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Mifepristone | Duration of Response | 44 days |
Secondary
Toxicity Associated With Adrenal Insufficiency
Toxicity will be evaluated per National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 3.0. Frequency and severity of adverse events will be tabulated using counts the following events of interest, which are related to possible adrenal insufficiency: nausea, vomiting, lethargy, dizziness, fatigue, anorexia, and skin rash. Any grade of these events that are self-reported by patients as well as events identified by physician assessment (e.g. physical exam) will be included.
Time frame: Up to 8 weeks after the end of study treatment or until any adverse events are resolved (whichever is longest)
Population: All patients received at least one dose of study medication and are included in this analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Dizziness | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Fatigue | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Nausea | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Vomiting | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Lethargy | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Anorexia | 0 percentage of participants |
| Mifepristone | Toxicity Associated With Adrenal Insufficiency | Skin Rash | 0 percentage of participants |
Other Pre-specified
Overall Survival
The time from patient entry into the protocol to death by any cause.
Time frame: 5 years
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Mifepristone | Overall Survival | 24.2 Months | Standard Deviation 28.5 |