Compassionate Use of Mifepristone in Brain/Nervous System and Other Cancers

• Sponsor: New Mexico Cancer Care Alliance (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00832871
• Collaborator: The Feminist Majority Foundation (Other)
• Enrollment: 4
• Location: 1
• Arm: 1
• Duration (Months): 85
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cancer	Drug: Mifepristone	N/A

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

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: INST 0817: Compassionate Use of Mifepristone
• Study Start Date: Nov 1, 2008
• Actual Primary Completion Date: Dec 1, 2015
• Actual Study Completion Date: Dec 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Mifepristone 200 mg RU-486 (Mifepristone) daily	Drug: Mifepristone; Mifepristone 200 mg will be administered orally; Other Names: RU-486; Mifeprex

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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 Outcome Measures

1. Overall Survival [5 years]

   The time from patient entry into the protocol to death by any cause.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Universtiy of New Mexico - Cancer Center	Albuquerque	New Mexico	United States	87106

Sponsors and Collaborators

• New Mexico Cancer Care Alliance
• The Feminist Majority Foundation

Investigators

• Principal Investigator: Fa-Chyi Lee, M.D., University of New Mexico Cancer Center

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• University of New Mexico Cancer Center
• New Mexico Cancer Care Alliance

Publications

None provided.

• Responsible Party: New Mexico Cancer Care Alliance
• ClinicalTrials.gov Identifier: NCT00832871
• Other Study ID Numbers: INST 0817; NCI-2011-02682
• First Posted: Jan 30, 2009
• Last Update Posted: Jun 29, 2018
• Last Verified: May 1, 2018

• Keywords provided by New Mexico Cancer Care Alliance: Mifepristone; RU-486; Compassionate Use; Antiprogesterone
• Additional relevant MeSH terms: Mifepristone

Study Results

Participant Flow

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Period Title: Overall Study
STARTED	4
COMPLETED	4
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Overall Participants	4
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	45
Sex: Female, Male (Count of Participants)
Female	2 50%
Male	2 50%
Region of Enrollment (participants) [Number]
United States	4 100%

Outcome Measures

1. Primary Outcome

Title	Duration of Response
Description	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

Outcome Measure Data

Analysis Population Description

Only one patient achieved stable disease. This patient's duration of response could therefore be reported. The other three patients progressed on treatment.

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Measure Participants	1
Median (Full Range) [days]	44

2. Secondary Outcome

Title	Toxicity Associated With Adrenal Insufficiency
Description	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)

Outcome Measure Data

Analysis Population Description

All patients received at least one dose of study medication and are included in this analysis.

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Measure Participants	4
Nausea	0 0%
Vomiting	0 0%
Lethargy	0 0%
Dizziness	0 0%
Fatigue	0 0%
Anorexia	0 0%
Skin Rash	0 0%

3. Other Pre-specified Outcome

Title	Overall Survival
Description	The time from patient entry into the protocol to death by any cause.
Time Frame	5 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Measure Participants	4
Median (Standard Deviation) [Months]	24.2 (28.5)

Adverse Events

Arm/Group Title	Mifepristone
Arm/Group Description	200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
All Cause Mortality
	Mifepristone
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Mifepristone
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Other (Not Including Serious) Adverse Events
	Mifepristone
	Affected / at Risk (%)	# Events
Total	2/4 (50%)
Blood and lymphatic system disorders
Edema: limb	1/4 (25%)	1
Edema: trunk	1/4 (25%)	1
Eye disorders
Watery eye	1/4 (25%)	1
Diplopia	1/4 (25%)	1
Nervous system disorders
Headache	1/4 (25%)	1

Limitations/Caveats

This is a compassionate use protocol. No statistical analysis was specified in the protocol; it states that only descriptive data will be collected. Data from only 4 patients should not be considered reliable.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.